1. Preclinical data provide quantitative evidence of TSHA-102's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions on a cell-by-cell basis

    Significant survival benefit demonstrated in 4-5 week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease, a more translatable model of the disorder in humans

    IND/CTA filing expected in second half of 2021, with Phase 1/2 trial initiation anticipated by year-end

    Estimated 25,000 patients in U.S. and Europe represent multi-billion-dollar commercial opportunity

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the…

    Preclinical data provide quantitative evidence of TSHA-102's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions on a cell-by-cell basis

    Significant survival benefit demonstrated in 4-5 week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease, a more translatable model of the disorder in humans

    IND/CTA filing expected in second half of 2021, with Phase 1/2 trial initiation anticipated by year-end

    Estimated 25,000 patients in U.S. and Europe represent multi-billion-dollar commercial opportunity

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been granted orphan drug designation from the European Commission for TSHA-102, an AAV9-based gene replacement therapy in development for Rett syndrome.

    "The receipt of orphan drug designation from the European Commission represents an important regulatory milestone that has the potential to expedite the global clinical development of TSHA-102, a one-time gene therapy with disease modifying potential," said RA Session II, President, Founder and Chief Executive Officer of Taysha. "Promising preclinical data demonstrate that TSHA-102's novel self-regulatory feedback mechanism, miRARE, has the ability to regulate MECP2 expression in a genotype-dependent manner on a cell-by-cell basis. We are highly encouraged that this novel treatment approach can help effectively address a disease that has historically been difficult to treat, and we look forward to submitting an IND/CTA in the second half of this year and initiating a Phase 1/2 clinical trial by year-end."

    Rett syndrome is a severe genetic neurodevelopmental disorder caused by a mutation in the X-linked MECP2 gene essential for neuronal and synaptic function in the brain. Primarily occurring in females, Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 in the United States and European Union. Patients have normal early development, with symptom onset typically beginning between six to 18 months of age. Rett syndrome is characterized by rapid developmental regression that leads to intellectual disabilities, loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments and breathing issues. Currently, there are no approved therapies.

    TSHA-102 is a self-complementary intrathecally delivered AAV9 gene replacement therapy under development for the treatment of Rett syndrome. TSHA-102 delivers the MECP2 gene, which includes the novel miRNA-Responsive Auto-Regulatory Element (miRARE), to regulate MECP2 expression in a genotypic-dependent manner on a cell-by-cell basis. The miRARE technology is designed to prevent toxicity associated with overexpression of MECP2.

    Positive preclinical data for TSHA-102 in Rett syndrome recently published in Brain provide quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression on a cell-by-cell basis across different regions of the brain in both wild type and knockout mouse models of Rett syndrome. TSHA-102 has previously received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA). Submission of an IND/CTA filing for TSHA-102 is expected in the second half of 2021, with initiation of a Phase 1/2 clinical trial by year-end 2021.

    The European Commission grants orphan drug designation for medicines being developed for the diagnosis, prevention or treatment of treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-102, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-102's eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  2. TSHA-104 restored to normal levels elevation of blood lactate on exhaustive exercise in dose-dependent manner in SURF1 knockout mice

    TSHA-104 increased COX1 activity in brain and muscle in dose-dependent manner in SURF1 knockout mice

    Combination of intrathecal and intravenous delivery did not prove benefit over intrathecal alone

    No associated toxicity risks or severe tissue damage were seen

    There are no approved treatments for the underlying cause of the disease and the burden of disease and risk of mortality are high

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous…

    TSHA-104 restored to normal levels elevation of blood lactate on exhaustive exercise in dose-dependent manner in SURF1 knockout mice

    TSHA-104 increased COX1 activity in brain and muscle in dose-dependent manner in SURF1 knockout mice

    Combination of intrathecal and intravenous delivery did not prove benefit over intrathecal alone

    No associated toxicity risks or severe tissue damage were seen

    There are no approved treatments for the underlying cause of the disease and the burden of disease and risk of mortality are high

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced publication of new preclinical data for TSHA-104 in SURF1-associated Leigh syndrome in Molecular Therapy: Methods & Clinical Development.

    SURF1 deficiency is a monogenic mitochondrial disorder that is the most common cause of cytochrome c oxidase deficient Leigh syndrome, a rapidly progressive neurological disorder that leads to degeneration of the CNS. Leigh syndrome typically presents in the first year of life and is characterized by progressive loss of mental and movement abilities that can result in death in early childhood. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation, and the estimated prevalence of SURF1 deficiency is 300 to 400 patients in the United States and European Union. There are currently no approved therapies to treat SURF1-associated Leigh syndrome.

    "SURF1 is part of cytochrome c oxidase, a mitochondrial enzyme known as COX involved in the metabolic production of ATP. Children with SURF1 deficiency have severely impaired COX activity and cannot generate ATP by aerobic respiration appropriately. This disruption in overall energy metabolism increases anaerobic respiration, leading to elevated levels of lactate and the clinical phenotype of Leigh syndrome," said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "We are pleased that the published preclinical data for TSHA-104 demonstrate that the gene replacement therapy can address the hallmark characteristics of mitochondrial dysfunction by delivering a functional SURF1 gene, ultimately restoring COX activity and mitochondrial function and correcting elevated lactate levels in the body."

    RA Session II, President, Founder and CEO of Taysha, added, "In the mouse model, we are seeing expression of SURF1 in the brain and spinal cord at levels sufficient to effectively restore COX activity in the brain, liver and muscle with one intrathecal administration of TSHA-104. We are encouraged by the ability of TSHA-104 to diminish exhaustive exercise-induced lactic acidosis, supporting intact mitochondrial function under states of stress in these preclinical models. This proof-of-concept study indicates TSHA-104 is safe and effective in rescuing metabolic dysfunction in preclinical models of SURF1 deficiency."

    A one-time intrathecal injection of TSHA-104 resulted in codon-optimized human SURF1 expression in multiple relevant brain and spinal cord regions in SURF1 knockout mice. A combination of intrathecal and intravenous delivery did not provide any benefit over intrathecal delivery alone, supporting continued use of intrathecal administration. TSHA-104 increased mitochondrially encoded cytochrome c oxidase 1 (MT-CO1) abundance four weeks post-injection in the CNS and peripheral tissues and increased COX1 activity in brain and muscle in a dose-dependent manner. Biochemical COX activity correlated with histological COX content level, supporting regional COX activity. Following exhaustive exercise-induced lactic acidosis TSHA-104 was able to rescue elevated lactate levels in SURF1 knockout mice dose-dependently. The safety profile assessed by non-GLP (Good Laboratory Practice) evaluation in wild-type mice using a similar dosing regimen determined no associated toxicity risks or severe tissue damage detected up to one year post-injection. Collectively this proof-of-concept study demonstrates TSHA-104 can safely and sufficiently rescue mitochondrial dysfunction in SURF1 knockout mice, supporting further translational development for the treatment of SURF1-associated Leigh syndrome.

    TSHA-104 is an AAV9-based gene replacement therapy encoding the human SURF1 protein that is administered intrathecally for the treatment of SURF1-associated Leigh syndrome. TSHA-104 has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-104, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-104's eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  3. Virtual event on Wednesday, September 22, 2021, at 10:00 a.m. ET will provide an overview of Rett syndrome, discuss natural history, and the TSHA-102 program and clinical development strategy

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced it will host a virtual key opinion leader (KOL) webinar on TSHA-102 for the treatment of Rett syndrome on Wednesday, September 22, 2021, from 10:00 a.m. to 12:30 p.m. ET.

    The event will feature a presentation from key opinion leader Jeffrey Neul, M.D., Ph.D…

    Virtual event on Wednesday, September 22, 2021, at 10:00 a.m. ET will provide an overview of Rett syndrome, discuss natural history, and the TSHA-102 program and clinical development strategy

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced it will host a virtual key opinion leader (KOL) webinar on TSHA-102 for the treatment of Rett syndrome on Wednesday, September 22, 2021, from 10:00 a.m. to 12:30 p.m. ET.

    The event will feature a presentation from key opinion leader Jeffrey Neul, M.D., Ph.D., Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor of Pediatrics, Pharmacology, and Special Education, Pediatric Neurology at the Vanderbilt University Medical Center, who will provide an overview of Rett syndrome and the underlying pathology of this genetic neurodevelopmental disorder, as well as discuss the natural history of the disease.

    The event will also feature presentations from:

    • Monica Coenraads, Chief Executive Officer of Rett Syndrome Research Trust (RSRT), who will discuss the burden of disease and provide a patient and caregiver perspective
    • Steven Gray, Ph.D., Associate Professor in the Department of Pediatrics at UT Southwestern and Chief Scientific Advisor at Taysha, who will review the miRARE platform and preclinical data generated to date for TSHA-102 in Rett syndrome
    • Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of R&D of Taysha, who will discuss the clinical development strategy for TSHA-102 in Rett syndrome and provide a regulatory update

    To register for the investor day, please click here. A live video webcast will be available in the "Events & Media" section of the Taysha corporate website. An archived version of the event will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  4. Citi's 16th Annual BioPharma Conference on September 8, 2021

    Morgan Stanley 19th Annual Global Healthcare Conference on September 9, 2021 at 5:00 pm ET

    2021 Wells Fargo Virtual Healthcare Conference on September 10,2021 at 1:20 pm ET

    HC Wainwright 23rd Annual Global Investment Conference on September 13, 2021 at 7:00 am ET

    SVB Leerink CybeRx Series: Neuromuscular, Rare Diseases & Genetic Medicines on September 23,2021

    2021 Cantor Virtual Global Healthcare Conference on September 27, 2021 at 8:00 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS…

    Citi's 16th Annual BioPharma Conference on September 8, 2021

    Morgan Stanley 19th Annual Global Healthcare Conference on September 9, 2021 at 5:00 pm ET

    2021 Wells Fargo Virtual Healthcare Conference on September 10, 2021 at 1:20 pm ET

    HC Wainwright 23rd Annual Global Investment Conference on September 13, 2021 at 7:00 am ET

    SVB Leerink CybeRx Series: Neuromuscular, Rare Diseases & Genetic Medicines on September 23, 2021

    2021 Cantor Virtual Global Healthcare Conference on September 27, 2021 at 8:00 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in the following investor healthcare conferences: Citi's 16th Annual BioPharma Conference, Morgan Stanley 19th Annual Global Healthcare Conference, 2021 Wells Fargo Virtual Healthcare Conference, HC Wainwright 23rd Annual Global Investment Conference and the 2021 Cantor Virtual Global Healthcare Conference and the SVB Leerink CybeRx Series.

    Conferences Details:

    Event:

    Citi's 16th Annual BioPharma Conference

    Date:

    Wednesday September 8, 2021

    Format:

    1x1 meetings

    Participants:

    RA Session II, President, Founder and CEO

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

    Kamran Alam, Chief Financial Officer

    Kimberly Lee, SVP, Corporate Communications and Investor Relations

    Event:

    Morgan Stanley 19th Annual Global Healthcare Conference

    Date:

    Thursday September 9, 2021

    Time:

    5:00 pm ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

    Kamran Alam, Chief Financial Officer

    Event:

    2021 Wells Fargo Virtual Healthcare Conference

    Date:

    Friday September 10, 2021

    Time:

    1:20 pm ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

    Kamran Alam, Chief Financial Officer

    Event:

    HC Wainwright 23rd Annual Global Investment Conference

    Date:

    Monday September 13, 2021

    Time:

    7:00 am ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

    Kamran Alam, Chief Financial Officer

    Event:

    SVB Leerink CybeRx Series: Neuromuscular, Rare Diseases & Genetic Medicine

    Date:

    Thursday September 23, 2021

    Format:

    1x1 meetings

    Participants:

    RA Session II, President, Founder and CEO

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

    Kamran Alam, Chief Financial Officer

    Kimberly Lee, SVP, Corporate Communications and Investor Relations

    Event:

    2021 Cantor Virtual Global Healthcare Conference

    Date:

    Monday September 27, 2021

    Time:

    8:00 am ET

    Format:

    Fireside Chat

    Participants:

    RA Session II, President, Founder and CEO

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

    Kamran Alam, Chief Financial Officer

    Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  5. Designation supports unmet need for treatment options for patients with rare form of genetic epilepsy

    TSHA-105 is first program in Taysha's pipeline to receive designation from European Commission

    No approved treatments for the underlying cause of the disease

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been granted orphan drug designation from the European Commission for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

    "SLC13A5 deficiency leads…

    Designation supports unmet need for treatment options for patients with rare form of genetic epilepsy

    TSHA-105 is first program in Taysha's pipeline to receive designation from European Commission

    No approved treatments for the underlying cause of the disease

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been granted orphan drug designation from the European Commission for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

    "SLC13A5 deficiency leads to a debilitating form of genetic epilepsy in children that results in persistent seizures and developmental delays, requiring constant supervision and care. With no available disease modifying treatments for this disease, we are pleased that TSHA-105 has been granted orphan drug designation from both the FDA and European Commission, highlighting the global need and important potential of TSHA-105 in helping treat this form of epilepsy," said RA Session II, President, Founder and CEO of Taysha. "We look forward to working with regulatory agencies to advance this promising gene replacement strategy as expeditiously as possible."

    SLC13A5 deficiency is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. As an autosomal recessive disorder, two copies of the mutated gene must be inherited for an infant to be affected. This type of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life. SLC13A5 deficiency is a rare disorder, with an estimated prevalence of 1,900 patients in the United States and in Europe. Current standards of care are anti-seizure medications which only target the symptoms and do not address the underlying cause of the disease.

    The European Commission grants orphan drug designation for medicines being developed for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect fewer than 5 in 10,000 people in the European Union. Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-105, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-105's eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  6. Virtual event on Monday, August 30, 2021, at 10:00 a.m. ET will provide an overview of CLN1 disease, discuss natural history, the TSHA-118 program and clinical development strategy

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced it will host a virtual key opinion leader (KOL) webinar on TSHA-118 for the treatment of CLN1 disease on Monday, August 30, 2021, from 10:00 a.m. to 12:30 p.m. ET.

    The event will feature a presentation from key opinion leader Angela Schulz, M.D., Ph.D., University Medical…

    Virtual event on Monday, August 30, 2021, at 10:00 a.m. ET will provide an overview of CLN1 disease, discuss natural history, the TSHA-118 program and clinical development strategy

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced it will host a virtual key opinion leader (KOL) webinar on TSHA-118 for the treatment of CLN1 disease on Monday, August 30, 2021, from 10:00 a.m. to 12:30 p.m. ET.

    The event will feature a presentation from key opinion leader Angela Schulz, M.D., Ph.D., University Medical Center Hamburg-Eppendorf, who will provide an overview of CLN1 disease, a severe, neurodegenerative lysosomal storage disease with no approved treatment, and discuss the natural history of the disease.

    Topics of discussion will also include the patient and caregiver perspective on the burden of disease, preclinical pharmacology and toxicology data for TSHA-118, and the clinical development strategy for the TSHA-118 program. A question and answer session will follow each formal presentation.

    The event will feature presentations from:

    • Sharon King, President of Taylor's Tale, who will discuss the burden of disease and provide a patient and caregiver perspective
    • Steven Gray, Ph.D., Associate Professor in the Department of Pediatrics at UT Southwestern and Chief Scientific Advisor at Taysha, who will review the preclinical and pharmacology data for TSHA-118 for the treatment of CLN1 disease
    • Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, CMO and Head of R&D of Taysha, who will discuss the clinical development strategy for TSHA-118

    Registration for this event is available through LifeSci Events. A live video webcast will be available in the "Events & Media" section of the Taysha corporate website. An archived version of the event will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  7. Positive pre-IND/CTA feedback obtained from several key regulatory agencies for TSHA-102 in Rett syndrome, TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1 disease, paving the way for multiple anticipated IND/CTA filings in the second half of 2021

    Entered into a non-dilutive term loan agreement with Silicon Valley Bank that provides up to $100 million in tranches at an attractive interest rate that lowers overall cost of capital, strengthens the balance sheet and offers additional financial and operational flexibility; assuming full drawdown, cash runway expected to be sufficient to achieve potential regulatory approval for TSHA-120 in GAN without the need for additional financing

    Reiterate guidance for clinical, regulatory and preclinical

    Positive pre-IND/CTA feedback obtained from several key regulatory agencies for TSHA-102 in Rett syndrome, TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1 disease, paving the way for multiple anticipated IND/CTA filings in the second half of 2021

    Entered into a non-dilutive term loan agreement with Silicon Valley Bank that provides up to $100 million in tranches at an attractive interest rate that lowers overall cost of capital, strengthens the balance sheet and offers additional financial and operational flexibility; assuming full drawdown, cash runway expected to be sufficient to achieve potential regulatory approval for TSHA-120 in GAN without the need for additional financing

    Reiterate guidance for clinical, regulatory and preclinical milestones in second half of 2021, including Phase 1/2 data from the highest dose cohort of TSHA-120 study in GAN, regulatory update for GAN program, preliminary Phase 1/2 safety and biomarker data for TSHA-101 in GM2 gangliosidosis, and initiation of Phase 1/2 trials in CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome

    Numerous successful GMP runs provided sufficient drug products to support five planned Phase 1/2 trials and multiple programs in IND/CTA-enabling studies in the second half of 2021

    Recent publications of GAN natural history data for TSHA-120 and positive preclinical data for TSHA-102 in Rett syndrome in the journal Brain provide further support of clinical development strategies

    Completed first R&D and manufacturing investor days highlighting preclinical and clinical data from multiple programs, the company's unique three-pillar approach to manufacturing and its capabilities, the regulatory environment for gene therapy manufacturing, and the immunology of gene therapy

    Upcoming virtual investor mini-series for CLN1, Rett and Angelman syndrome programs to feature Key Opinion Leaders and highlight progress across these programs

    Conference call and live webcast today at 8:00 a.m. Eastern Time

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported financial results for the second quarter ended June 30, 2021 and provided a corporate update.

    "We continue to make significant progress on several key clinical, manufacturing and strategic corporate initiatives which were highlighted in our R&D and Manufacturing investor days and in our recent press release announcing our non-dilutive financing with SVB," noted RA Session II, President, Founder and CEO of Taysha. "We have received positive pre-IND/CTA feedback from several key regulatory agencies for our Rett syndrome, GM2 gangliosidosis and CLN1 disease programs, which pave the way for multiple expected IND/CTA filings and clinical trial initiations in the second half of the year. Our success relies on robust, sustainable and high-quality manufacturing to support our portfolio, and we have completed numerous successful GMP runs to support five planned Phase 1/2 trials and several programs in IND/CTA-enabling studies. With ambitious corporate objectives planned over the next 12 to 18 months, we are pleased to have recently entered into a loan agreement for up to $100 million at an attractive interest rate which lowers overall cost of capital and contains no financial covenants or warrants. The initial draw bolsters our cash position and provides financial and operational flexibility. We believe full drawdown of this non-dilutive financing will extend our cash runway to support key value-creating milestones, including the release of Phase 1/2 data from the highest dose cohort in GAN, and Phase 1/2 data in GM2 gangliosidosis, Rett syndrome, CLN1 disease and SURF1-associated Leigh syndrome and, importantly, a potential regulatory approval for TSHA-120 in GAN without the need for additional financing. We look forward to updating you on our continued progress throughout the year."

    Recent Corporate Highlights

    • Positive pre-IND/CTA feedback obtained from multiple key regulatory agencies for TSHA-102 in Rett syndrome, TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1 disease, paving the way for multiple expected IND/CTA filings in the second half of 2021
    • Secured up to $100 million non-dilutive term loan financing, with $40 million available at closing from Silicon Valley Bank (SVB), of which Taysha has drawn $30 million. The company has the option to draw down the remaining tranches, subject to certain conditions. The interest rate is the greater of 7.0% or the WSJ Prime Rate plus 3.75% and there are no financial covenants or warrants
    • Multiple successful GMP runs provided drug products to support five planned Phase 1/2 trials and multiple programs in IND/CTA-enabling studies in the second half of 2021
    • Publication of GAN natural history data for TSHA-120 in peer-reviewed journal, Brain, that included robust assessment across clinical outcomes for GAN, including motor, sensory, respiratory, neurophysiologic, MRI and biopsy data. All GAN natural history data was generated and supported by the Intramural Program of the National Institute of Neurological Disorders and Stroke (NINDS), U.S. National Institutes of Health (NIH) under umbrella protocol NCT01568658 and under the leadership of the Principal Investigator, Carsten Bönneman
    • Publication of positive preclinical data for TSHA-102 in Rett syndrome in Brain that provided quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression on a cell-by-cell basis across different regions of the brain in both wild type and knockout mouse models of Rett syndrome
    • Announcement of positive data from multiple programs at R&D Day on June 28-29
      • Phase 1/2 visual acuity data for TSHA-120 in patients with GAN demonstrated a dose-dependent trend towards stabilization of visual acuity, one of the most challenging symptoms to treat. Recently obtained preclinical data demonstrated no impact on dorsal root ganglia in non-human primates when TSHA-120 was delivered intrathecally
      • Preclinical data for TSHA-102 in Rett syndrome demonstrated that miRARE reduced overall expression of MECP2 transgene expression compared to unregulated MECP2 in wild type mice. miRARE regulated genotype-dependent MECP2 expression across different brain regions in wild type and Rett knockout mouse models on a cell-by-cell basis
      • An in-depth look into natural history provided incremental data on disease progression and motor development delays in patients with GM2 gangliosidosis
      • Preclinical data for TSHA-118 demonstrated sustained preservation of motor function and rescue of survival with higher doses of TSHA-118 and earlier intervention in CLN1 knockout mice
      • Positive findings indicated a small increase in COX1 activity can significantly improve the clinical phenotype of patients with SURF1-associated Leigh syndrome; similarly, reduction in COX activity correlated with disease worsening in patient fibroblast data
      • In SLC13A5 knockout mice, TSHA-105 decreased plasma citrate levels and reduced seizures and associated deaths
      • In SLC6A1 knockout and heterozygous mouse models, CNS administration of TSHA-103 rescued abnormal seizure activity. Recently obtained data demonstrated rescue of functional measures such as nesting, open field activity, hind limb clasping and latency to fall from the rotarod
      • TSHA-112 reduced GYS1 expression in the APBD knockout model which resulted in decreased polyglucosan body formation in mice brain
      • TSHA-111-LAFORIN and TSHA-111-MALIN reduced GYS1 expression in the laforin and malin knockout models which resulted in decreased Lafora body formation in mice brain
      • Preclinical data for TSHA-113 demonstrated significant reduction of tau mRNA and protein levels, validating the potential of using AAV-mediated gene silencing to achieve life-long reduction of tau protein levels and warranting further preclinical development
      • Two novel approaches to treat Angelman syndrome disclosed, including knockdown of UBE3A-ATS to unsilenced the paternal allele and a gene replacement strategy on UBE3A to mimic maternal UBE3A allele expression
    • Manufacturing Day on July 27th highlighted the company's unique three-pillar approach to the manufacturing process, its manufacturing capabilities, the regulatory environment for gene therapy manufacturing, and the immunology of gene therapy with a focus on the practical aspects of initiating and monitoring immunosuppressive regimes
    • Appointment of Chief Development Officer, Mary Newman, who previously served as Senior Vice President of Regulatory Affairs at Astellas Gene Therapies, formerly Audentes, and brings over 30 years of experience in translational development, program management and regulatory affairs in the biotech industry
    • Founding member of the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve
    • Continued progress on internal manufacturing facility with completion of the demolition phase
    • Opened the Dallas headquarters in May 2021
    • Expansion of employee base to 150 across all areas of the organization

    Anticipated Milestones by Program

    TSHA-120 for giant axonal neuropathy (GAN): an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN, a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin

    • Report clinical data for TSHA-120 from the high dose cohort of 3.5x1014 total vg in second half of 2021
    • Engage with key regulatory agencies to discuss the approval pathway and provide a regulatory update by year-end 2021

    TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes – HEXA and HEXB, comprising the alpha and beta sub-units of Beta Hexoseaminidase A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

    • Report preliminary Phase 1/2 safety and biomarker data (Queen's University trial) in second half of 2021
    • Submit an Investigational New Drug (IND) application in the U.S. in second half of 2021
    • Initiate Phase 1/2 clinical trial in the U.S. in second half of 2021

    TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments

    • CLN1 Investor Day on August 30, 2021
    • Maintain current open IND
    • Initiate a Phase 1/2 clinical trial in second half of 2021
    • Report biomarker data in first half of 2022

    TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for a severe neurodevelopmental disorder, designed to deliver MECP2, as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis

    • Rett syndrome Investor Day in September 2021
    • Submit IND/CTA filing in second half of 2021
    • Initiate Phase 1/2 clinical trial by year-end 2021
    • Report clinical data by year-end 2022

    TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments

    • Submit IND/CTA filing in second half of 2021
    • Initiate Phase 1/2 trial by year-end 2021
    • Report biomarker data in first half of 2022

    Pipeline programs in IND/CTA-enabling studies

    • Submit an IND/CTA filing for one of six programs in 2021: TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN and TSHA-111-MALIN in two forms of Lafora disease, TSHA-112 in APBD, TSHA-119 in GM2 AB variant and TSHA-103 in SLC6A1 haploinsufficiency disorder

    Discovery programs

    • Advance four new undisclosed programs focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases into preclinical development in 2021

    Next-generation technology platform

    • Continue development efforts focused on regulated transgene expression with expansion of miRARE platform into additional CNS diseases
    • Initiate confirmatory preclinical studies for the vagus nerve redosing platform in canines
    • Advance mini-gene discovery program in genetic forms of epilepsy and neurodevelopmental disorders
    • Continue discovery and development efforts around next-generation capsids

    Second Quarter 2021 Financial Highlights

    Research and Development (R&D) Expenses: Research and development expenses were $30.6 million for the three months ended June 30, 2021, compared to $3.1 million for the three months ended June 30, 2020. The $27.5 million increase was primarily attributable to an increase of $10.3 million of expenses incurred in research and development manufacturing and other raw material purchases, which included cGMP batches produced by Catalent and UT Southwestern. We incurred an increase in employee compensation expenses of $8.5 million, which included $2.2 million of non-cash stock-based compensation, and $8.7 million in third-party research and development expenses, which includes clinical trial CRO activities, GLP toxicology studies, and consulting for regulatory and clinical studies.

    General and Administrative (G&A) Expenses: General and administrative expenses were $10.1 million for the three months ended June 30, 2021, compared to $0.9 million for the three months ended June 30, 2020. The increase was primarily attributable to incremental compensation expense, which included non-cash stock-based compensation, and additional consulting and professional fees.

    Net loss: Net loss for the second quarter ended June 30, 2021 was $40.9 million or $1.09 per share, as compared to a net loss of $21.2 million, or $1.95 per share, for the second quarter ended June 30, 2020.

    Cash and cash equivalents: As of June 30, 2021, Taysha had $197.4 million in cash and cash equivalents.

    Conference Call and Webcast Information

    Taysha management will hold a conference call and webcast today at 8:00 a.m. ET / 7:00 a.m. CT to review its financial and operating results and to provide a corporate update. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13722197. The live webcast and replay may be accessed by visiting Taysha's website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," "plans," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply and our anticipated cash runway assuming full access to the $100 million potentially available under the loan and security agreement. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

     

    Taysha Gene Therapies, Inc.

    Consolidated Statements of Operations

    (in thousands, except share and per share data)

    (Unaudited)

      For the Three Months

    Ended June 30,
      For the Six Months

    Ended June 30,
     

    2021

     

    2020

     

    2021

     

    2020

    Operating expenses:        
    Research and development  

    $

    30,643

     

     

    $

    3,062

     

     

    $

    54,497

     

     

    $

    8,576

     

    General and administrative  

     

    10,129

     

     

     

    948

     

     

     

    18,365

     

     

     

    1,018

     

    Total operating expenses  

     

    40,772

     

     

     

    4,010

     

     

     

    72,862

     

     

     

    9,594

     

    Loss from operations  

     

    (40,772

    )

     

     

    (4,010

    )

     

     

    (72,862

    )

     

     

    (9,594

    )

    Other income (expense):        
    Change in fair value of preferred stock tranche liability  

     

    -

     

     

     

    (17,210

    )

     

     

    -

     

     

     

    (17,030

    )

    Interest income  

     

    40

     

     

     

    -

     

     

     

    106

     

     

     

    -

     

    Interest expense  

     

    (194

    )

     

     

    -

     

     

     

    (194

    )

     

     

    (27

    )

    Total other expense, net  

     

    (154

    )

     

     

    (17,210

    )

     

     

    (88

    )

     

     

    (17,057

    )

    Net loss  

    $

    (40,926

    )

     

    $

    (21,220

    )

     

    $

    (72,950

    )

     

    $

    (26,651

    )

    Net loss per common share, basic and diluted  

    $

    (1.09

    )

     

    $

    (1.95

    )

     

    $

    (1.96

    )

     

    $

    (2.45

    )

    Weighted average common shares outstanding, basic and diluted  

     

    37,479,164

     

     

     

    10,894,999

     

     

     

    37,237,115

     

     

     

    10,894,999

     

     

    Taysha Gene Therapies, Inc.

    Consolidated Balance Sheet Data

    (in thousands, except share and per share data)

    (Unaudited)

      June 30,

    2021
      December 31,

    2020
    ASSETS    
    Current assets:    
    Cash and cash equivalents  

    $

    197,370

     

     

    $

    251,253

     

    Prepaid expenses and other current assets  

     

    9,932

     

     

     

    6,626

     

    Total current assets  

     

    207,302

     

     

     

    257,879

     

    Deferred lease asset  

     

    691

     

     

     

    715

     

    Property, plant and equipment, net  

     

    33,867

     

     

     

    287

     

    Total assets  

    $

    241,860

     

     

    $

    258,881

     

         
    LIABILITIES AND STOCKHOLDERS' EQUITY    
    Current liabilities    
    Accounts payable  

    $

    6,355

     

     

    $

    1,994

     

    Accrued expenses and other current liabilities  

     

    22,036

     

     

     

    5,135

     

    Total current liabilities  

     

    28,391

     

     

     

    7,129

     

    Build-to-suit lease liability  

     

    26,209

     

     

     

    -

     

    Other non-current liabilities  

     

    765

     

     

     

    450

     

    Total liabilities  

     

    55,365

     

     

     

    7,579

     

         
    Stockholders' equity    
    Preferred stock, $0.00001 par value per share; 10,000,000 shares

    authorized and no shares issued and outstanding as of June 30, 2021

    and December 31, 2020
     

     

    -

     

     

     

    -

     

    Common stock, $0.00001 par value per share; 200,000,000 shares

    authorized and 38,391,165 and 37,761,435 issued and outstanding as of

    June 30, 2021 and December 31, 2020, respectively
     

     

    -

     

     

     

    -

     

    Additional paid-in capital  

     

    320,571

     

     

     

    312,428

     

    Accumulated deficit  

     

    (134,076

    )

     

     

    (61,126

    )

    Total stockholders' equity  

     

    186,495

     

     

     

    251,302

     

    Total liabilities and stockholders' equity  

    $

    241,860

     

     

    $

    258,881

     

     

    View Full Article Hide Full Article
  8. Financing strengthens balance sheet, furthers financial and operational flexibility and lowers overall cost of capital

    No financial covenants and no warrants issued in connection with non-dilutive financing

    Full drawdown expected to extend cash runway to support key value-creating milestones including availability of Phase 1/2 GAN data from the highest dose cohort as well as regulatory guidance on registration pathway for GAN, data readouts in GM2 gangliosidosis, Rett syndrome, CLN1 disease, and SURF1-associated Leigh syndrome and, importantly, a potential regulatory approval for TSHA-120 in GAN without the need for additional financing

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing…

    Financing strengthens balance sheet, furthers financial and operational flexibility and lowers overall cost of capital

    No financial covenants and no warrants issued in connection with non-dilutive financing

    Full drawdown expected to extend cash runway to support key value-creating milestones including availability of Phase 1/2 GAN data from the highest dose cohort as well as regulatory guidance on registration pathway for GAN, data readouts in GM2 gangliosidosis, Rett syndrome, CLN1 disease, and SURF1-associated Leigh syndrome and, importantly, a potential regulatory approval for TSHA-120 in GAN without the need for additional financing

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has entered into a loan and security agreement with Silicon Valley Bank (SVB) that provides Taysha with up to $100 million of borrowing capacity.

    "Access to this non-dilutive financing at an attractive cost of capital, along with the current cash on hand, will provide Taysha with operational and financial flexibility to achieve numerous value-generating milestones including a potential regulatory approval for TSHA-120 in giant axonal neuropathy, or GAN," said RA Session II, Chief Executive Officer of Taysha. "Additional milestones include the release of Phase 1/2 data in the highest dose cohort in GAN, and Phase 1/2 data in GM2 gangliosidosis, Rett syndrome, CLN1 disease and SURF1-associated Leigh syndrome. We are pleased to partner with SVB as we continue to execute on our ambitious business plan."

    This non-dilutive financing provides Taysha with up to $100 million, with $40 million available at closing of which Taysha has drawn $30.0 million. The Company has the option to draw down the remaining tranches, subject to certain conditions. The interest rate is the greater of 7.0% or the WSJ Prime Rate plus 3.75%. There are no financial covenants and no warrants associated with the term loan.

    "Our financial commitment to Taysha speaks to our confidence in its core strategies and is consistent with our support of innovative life sciences businesses," said Michael White, Head of Business Development, Life Science & Healthcare, Silicon Valley Bank. "We are delighted to provide additional capital for the Company to further advance its robust development pipeline and achieve key value-generating milestones in the years to come."

    "In the last 12 months, we have quickly made the transition from a private to public company and from preclinical to clinical to pivotal-stage," said Kamran Alam, Chief Financial Officer of Taysha. "Building upon this momentum, we expect this non-dilutive financing to enable us to be well positioned to maximize long-term stockholder value."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    About Silicon Valley Bank

    For nearly 40 years, Silicon Valley Bank (SVB) has helped innovative companies and their investors move bold ideas forward, fast. SVB provides targeted financial services and expertise through its offices in innovation centers around the world. With commercial, international and private banking services, SVB helps address the unique needs of innovators. Learn more at svb.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the anticipated use of proceeds from borrowings under the loan and security agreement, our ability to access the full $100 million potentially available under the loan and security agreement and our ability to fund operations into the second half of 2023. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the year ended December 31, 2020 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  9. Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the second quarter ended June 30, 2021, and host a corporate update conference call and webcast on Monday, August 16, 2021, at 8:00 AM Eastern Time.

    Conference Call Details

    Monday, August 16, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

     

    877-407-0792

    International:

     

    201-689-8263

    Conference ID:

     

    13722197

    Webcast:

     

    https://ir.tayshagtx.com/news-events/events-presentations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the second quarter ended June 30, 2021, and host a corporate update conference call and webcast on Monday, August 16, 2021, at 8:00 AM Eastern Time.

    Conference Call Details

    Monday, August 16, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

     

    877-407-0792

    International:

     

    201-689-8263

    Conference ID:

     

    13722197

    Webcast:

     

    https://ir.tayshagtx.com/news-events/events-presentations

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  10. BTIG Virtual Biotechnology Conference on August 9, 2021 at 11:30 am ET

    2021 Wedbush Pacgrow Healthcare Virtual Conference on August 11, 2021 at 8:35 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivitol-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a fireside chat for the BTIG Virtual Biotechnology Conference and a panel for the 2021 Wedbush Pacgrow Healthcare Virtual Conference.

    Conferences Details:

    Event:

    BTIG Virtual Biotechnology Conference

    Date:

    Monday August 9, 2021

    Time:

    11:30 am ET

    Format…

    BTIG Virtual Biotechnology Conference on August 9, 2021 at 11:30 am ET

    2021 Wedbush Pacgrow Healthcare Virtual Conference on August 11, 2021 at 8:35 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivitol-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a fireside chat for the BTIG Virtual Biotechnology Conference and a panel for the 2021 Wedbush Pacgrow Healthcare Virtual Conference.

    Conferences Details:

    Event:

    BTIG Virtual Biotechnology Conference

    Date:

    Monday August 9, 2021

    Time:

    11:30 am ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

     

     

    Event:

    2021 Wedbush Pacgrow Healthcare Virtual Conference

    Date:

    Wednesday August 11, 2021

    Time:

    8:35 am ET

    Format:

    Panel discussion

    Participants:

    RA Session II, President, Founder and CEO

    Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  11. Virtual event on Tuesday, July 27, 2021, at 10:00 a.m. ET will highlight the company's unique three-pillar approach to manufacturing, its strategic capabilities and other key considerations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host a virtual manufacturing day for analysts and investors. The event will be webcast live on Tuesday, July 27, 2021, from 10:00 a.m. to 1:00 p.m. ET.

    Topics of discussion will include the company's unique three-pillar approach to the manufacturing…

    Virtual event on Tuesday, July 27, 2021, at 10:00 a.m. ET will highlight the company's unique three-pillar approach to manufacturing, its strategic capabilities and other key considerations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host a virtual manufacturing day for analysts and investors. The event will be webcast live on Tuesday, July 27, 2021, from 10:00 a.m. to 1:00 p.m. ET.

    Topics of discussion will include the company's unique three-pillar approach to the manufacturing process, its manufacturing capabilities, the regulatory environment for gene therapy manufacturing, and the immunology of gene therapy. A question and answer session will follow each formal presentation.

    The event will feature presentations from Taysha senior leaders:

    • Frederick Porter, Ph.D.

      Chief Technical Officer, Taysha Gene Therapies
    • Greg Gara

      Senior Vice President, Manufacturing, Taysha Gene Therapies
    • Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM

      Chief Medical Officer and Head of Research and Development, Taysha Gene Therapies

    Registration for this event is available through LifeSci Events. A live video webcast will be available in the "Events & Media" section of the Taysha corporate website. An archived version of the event will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  12. William Blair Biotech Focus Conference on July 15, 2021 at 11:00 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat for the William Blair Biotech Focus Conference 2021.

    Conference's Details:

     

    Event:

    William Blair Biotech Focus Conference 2021

    Date:

    Thursday, July 15, 2021

    Time:

    11:00 am ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer…

    William Blair Biotech Focus Conference on July 15, 2021 at 11:00 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat for the William Blair Biotech Focus Conference 2021.

    Conference's Details:

     

    Event:

    William Blair Biotech Focus Conference 2021

    Date:

    Thursday, July 15, 2021

    Time:

    11:00 am ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

    A live webcast of this conference will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  13. Largest cohort of genetically confirmed patients with GAN, including patients with the classic (early-onset) and milder (late-onset) forms of GAN

    Largest cross-sectional analysis highlighted clinical differences in patients with early-onset GAN versus late-onset GAN based on MFM32 performance as well as other functional motor scales and disease markers

    Robust assessment across clinical outcomes for GAN, including motor, sensory, respiratory, neurophysiologic, MRI and biopsy data

    First clinical study to evaluate a cohort of individuals with GAN for autonomic impairment

    On track to report clinical data for TSHA-120 from the 3.5x1014 total vg dose cohort in the second half of 2021

    Planning to engage with major regulatory agencies to discuss

    Largest cohort of genetically confirmed patients with GAN, including patients with the classic (early-onset) and milder (late-onset) forms of GAN

    Largest cross-sectional analysis highlighted clinical differences in patients with early-onset GAN versus late-onset GAN based on MFM32 performance as well as other functional motor scales and disease markers

    Robust assessment across clinical outcomes for GAN, including motor, sensory, respiratory, neurophysiologic, MRI and biopsy data

    First clinical study to evaluate a cohort of individuals with GAN for autonomic impairment

    On track to report clinical data for TSHA-120 from the 3.5x1014 total vg dose cohort in the second half of 2021

    Planning to engage with major regulatory agencies to discuss the approval pathway and expect to provide a regulatory update by year-end 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new analyses of natural history data for TSHA-120 in giant axonal neuropathy, or GAN. The data were published online and will be included in the June edition of Brain, a highly esteemed neurological science peer-reviewed journal.

    GAN is a progressive neurodegenerative disease that affects both the central and peripheral nervous systems. The disease is caused by loss-of-function mutations in the gene coding for gigaxonin, which results in dysregulation of intermediate filament turnover, an important structural component of the cell. Although no symptoms are present in the first few months of life, many children with GAN do show early symptoms and features before the age of five, including unsteady gait, frequent falls, and motor weakness. Symptoms worsen over time and children develop scoliosis, contractures, atrophy of the spinal cord and abnormalities of the white matter in the brain. Currently, there are no approved treatments for GAN, which results in death for patients in their late teens or early twenties.

    In this natural history study, 45 patients, age 3 years to 21 years old, with genetically confirmed GAN were enrolled at NIH and evaluated at their first enrollment visit. The objective of the cross-sectional analysis was to identify genetic variants, explore correlations between genotype and phenotype, identify reliable markers of disease severity and assess how these markers correlate with ambulatory function and the impact of the early- and late-onset phenotypes on these markers.

    The two sub cohorts of GAN patients in the study included thirty-five patients with early-onset GAN and 10 patients with late-onset GAN. In the early-onset cohort, the mean age of onset of gait or motor impairment was 2.3 years old whereas the mean age of onset of symptoms in the late-onset cohort was 5.4 years old. Motor Function Measure 32 (MFM32), a validated and well-known scale to measure strength and motor function had the strongest correlation across outcome measures and age in patients with GAN. Patients with late-onset GAN had better functional performance compared to similarly aged patients with early-onset GAN. Ambulatory ability between the two phenotypes also differed. Disease progression in early-onset GAN patients occurred in a uniform and homogenous manner. Autonomic manifestations of the disease did not correlate with age or motor function.

    "The recent publication in Brain serves as the baseline data for a longitudinal natural history assessment and adds important context to results from three dose cohorts in the ongoing clinical GAN trial," said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "These results also confirm our findings that there is a clinical difference between early-onset GAN, a relentlessly progressive and fatal neuropathy, and late-onset GAN, which has significant disease morbidity, and underscores the importance for GAN to be included in genetic screens for hereditary neuropathies. The estimated prevalence for GAN is 2,400 patients but the GAN population may be larger than previously appreciated. We view genetic testing as an important aspect of patient identification and look forward to leveraging our key collaborations with companies to increase patient diagnostic efforts and allow for earlier intervention. Of note, data from today's publication provide further support and confidence in the overall clinical program design for TSHA-120 which include this ongoing, prospective, natural history and outcomes assessment study, and an interventional dose selection safety and efficacy study. Building on the previously presented and favorable interventional study data, we remain on track to report clinical data from the highest dose cohort in the interventional clinical trial for TSHA-120 in the second half of this year. We look forward to engaging with major regulatory agencies to discuss the approval pathway for TSHA-120 and to provide a regulatory update by year-end."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-120, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-120's eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  14. Two-day R&D day on June 28 and 29, 2021 at 10:00 am ET will highlight progress across its pipeline and will feature presentations from key opinion leaders

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host its first two-day virtual research and development (R&D) day for analysts and investors. The event will be webcast live on June 28 and June 29, 2021 from 10:00 a.m. to 1:00 p.m. ET each day.

    The event will highlight the company's R&D progress, focused on advancement of its early…

    Two-day R&D day on June 28 and 29, 2021 at 10:00 am ET will highlight progress across its pipeline and will feature presentations from key opinion leaders

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host its first two-day virtual research and development (R&D) day for analysts and investors. The event will be webcast live on June 28 and June 29, 2021 from 10:00 a.m. to 1:00 p.m. ET each day.

    The event will highlight the company's R&D progress, focused on advancement of its early- and late-stage investigational programs. Topics of discussion will include:

    Day 1 – June 28, 2021

    • TSHA-120 (GAN): Pivotal-stage AAV9 gene replacement therapy program for the treatment of giant axonal neuropathy (GAN), a rare autosomal recessive disease of the central and peripheral nervous systems caused by loss-of-function gigaxonin gene mutations.
    • TSHA-101 (GM2 gangliosidosis): The first bicistronic AAV9 gene therapy in clinical development designed to deliver two genes, HEXA and HEXB, comprising the alpha and beta sub-units of beta hexosaminidase A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease. TSHA-101 is currently in Phase 1/2 development.
    • TSHA-118 (CLN1 disease): AAV9-based gene therapy designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1 disease, a rapidly progressing rare lysosomal storage disease with no currently approved treatments. This gene replacement therapy program is currently under an open IND, with initiation of a Phase 1/2 trial expected in the second half of 2021.
    • TSHA-102 (Rett syndrome): AAV9-based gene therapy in development for Rett syndrome, a severe neurodevelopmental disorder, designed to deliver MECP2, as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis. This regulated gene replacement therapy is currently in IND/CTA-enabling studies, with an IND/CTA filing expected in the second half of 2021.

    Day 2 – June 29, 2021

    • TSHA-104 (SURF1-associated Leigh syndrome): AAV9-based gene therapy with a transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no currently approved treatments. This gene replacement therapy program is currently in IND/CTA-enabling studies, with an IND/CTA filing expected in the second half of 2021.
    • TSHA-105 (SLC13A5 deficiency): AAV9-based gene therapy expressing human SLC13A5 protein to potentially treat SLC13A5 deficiency, a rare autosomal recessive epileptic encephalopathy characterized by the onset of seizures within the first few days of life. This gene replacement therapy program is currently in IND/CTA-enabling studies.
    • TSHA-103 (SLC6A1 haploinsufficiency disorder): Gene replacement therapy constructed from a codon-optimized version of the human SLC6A1 gene packaged within an AAV9 viral vector for the treatment of SLC6A1 haploinsufficiency disorder. This program is currently in IND/CTA-enabling studies.
    • TSHA-111-LAFORIN and TSHA-111-MALIN (Lafora disease): Recombinant AAV9 viral vectors with miRNA targeting the knockdown of GYS1 for the treatment of Lafora disease. These programs are currently in IND/CTA-enabling studies.
    • TSHA-112 (APBD): Recombinant AAV9 viral vector with miRNA targeting the knockdown of GYS1 for the treatment of Adult Polyglucosan Body Disease (APBD). This program is currently in IND/CTA-enabling studies.
    • Additional preclinical programs, including tauopathies (TSHA-113, an AAV9 viral vector that utilizes AAV-mediated gene silencing to potentially deliver life-long reduction of tau protein levels in neurons following a single dose) and Angelman syndrome (TSHA-106, an AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS).

    The event will feature presentations from Taysha senior leaders and the following key opinion leaders:

    • Steven Gray, Ph.D.

      Chief Scientific Advisor, Taysha Gene Therapies

      Associate Professor, Department of Pediatrics at UT Southwestern
    • Berge Minassian, M.D.

      Chief Medical Advisor, Taysha Gene Therapies

      Division Chief, Pediatric Neurology at UT Southwestern
    • Rachel Bailey, Ph.D.

      ssistant Professor, Department of Pediatrics at UT Southwestern
    • Kimberly Goodspeed, M.D.

      Assistant Professor, Department of Pediatrics, Neurology and Psychiatry at UT Southwestern

    Registration for this event is available through LifeSci Events. A live video webcast will be available in the "Events & Media" section of the Taysha corporate website. An archived version of the event will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  15. LifeSci Genetics Medicine Conference on June 22, 2021 at 10:00 am ET

    LSX Biotech Growth CEO Forum on June 22, 2021 at 1:10 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats at the LifeSci Genetics Medicine Conference and LSX Biotech Growth CEO Forum.

    Conferences Details:

    Event:

     

    LifeSci Genetics Medicine Conference

    Date:

     

    Tuesday, June 22, 2021

    Time:

     

    10:00 am ET

    Format:

     

    Fireside chat

    Participants:

     

    RA Session…

    LifeSci Genetics Medicine Conference on June 22, 2021 at 10:00 am ET

    LSX Biotech Growth CEO Forum on June 22, 2021 at 1:10 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats at the LifeSci Genetics Medicine Conference and LSX Biotech Growth CEO Forum.

    Conferences Details:

    Event:

     

    LifeSci Genetics Medicine Conference

    Date:

     

    Tuesday, June 22, 2021

    Time:

     

    10:00 am ET

    Format:

     

    Fireside chat

    Participants:

     

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

     

     

    Event:

     

    LSX Biotech Growth CEO Forum

    Topic:

     

    "Too Much of a Good Thing Can be Wonderful: Optimizing Value From Broad Discovery Platforms Through Strategy and Strategic Partnering"

    Date:

     

    Tuesday, June 22, 2021

    Time:

     

    1:10 pm ET

    Format:

     

    Fireside chat

    Participants:

     

    RA Session II, President, Founder and CEO

    Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  16. The Annual Stockholder Meeting on June 17, 2021 at 10 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its first annual stockholder meeting will be held June 17, 2021 at 10 am ET via webcast.

    The webcast for this meeting will be available in the "Events & Presentations" section of the Taysha corporate website. A copy of Taysha's 2020 annual report is available here and in the "Financial Information" section of the Taysha Corporate Website.

    About Taysha Gene Therapies

    Taysha Gene Therapies…

    The Annual Stockholder Meeting on June 17, 2021 at 10 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its first annual stockholder meeting will be held June 17, 2021 at 10 am ET via webcast.

    The webcast for this meeting will be available in the "Events & Presentations" section of the Taysha corporate website. A copy of Taysha's 2020 annual report is available here and in the "Financial Information" section of the Taysha Corporate Website.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  17. Jefferies Virtual Healthcare Conference on June 2, 2021 at 10:30 am ET

    Goldman Sachs 42nd Annual Global Healthcare Conference on June 10, 2021 at 5:30 pm ET

    JMP Securities Life Science Conference on June 16,2021 at 12:30 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivitol-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat for the Jefferies Virtual Healthcare Conference, Goldman Sachs 42nd Annual Global Healthcare Conference, and the JMP Securities Life Science Conference.

    Conferences Details:

    Jefferies Virtual Healthcare Conference on June 2, 2021 at 10:30 am ET

    Goldman Sachs 42nd Annual Global Healthcare Conference on June 10, 2021 at 5:30 pm ET

    JMP Securities Life Science Conference on June 16, 2021 at 12:30 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivitol-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat for the Jefferies Virtual Healthcare Conference, Goldman Sachs 42nd Annual Global Healthcare Conference, and the JMP Securities Life Science Conference.

    Conferences Details:

    Event:

    Jefferies Virtual Healthcare Conference

    Date:

    Wednesday, June 2, 2021

    Time:

    10:30 am ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

     

     

    Event:

    Goldman Sachs 42nd Annual Global Healthcare Conference

    Date:

    Thursday, June 10, 2021

    Time:

    5:30 pm ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

     

    Event:

    JMP Securities Life Science Conference

    Date:

    Wednesday, June 16, 2021

    Time:

    12:30 pm ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

    Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  18. Joined an alliance of life science companies dedicated to rare disease therapy development to support advocacy for policies that will improve processes for timely and cost-effective delivery of treatments

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has joined the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve.

    "Taysha…

    Joined an alliance of life science companies dedicated to rare disease therapy development to support advocacy for policies that will improve processes for timely and cost-effective delivery of treatments

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has joined the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve.

    "Taysha is honored to be a founding member of the Rare Disease Company Coalition," said RA Session II, President, Founder and Chief Executive Officer of Taysha. "As we grow to become a leader in the development of gene therapies for the treatment of rare diseases, we have a responsibility to our community, especially to our patients and their families, to advocate for more effective policies and regulations that will encourage innovation around potentially life-saving therapies. We look forward to working with this impressive group of companies to drive awareness about the specific challenges of rare disease research and development and promote solutions that will support timely delivery of valuable treatments to patients."

    As an education and advocacy-focused coalition of companies, the shared goal of the Coalition is to inform policymakers of the unique circumstances of the rare disease company business model, specifically focusing on the unique circumstances involved in developing and bringing to market therapies for small and differentiated patient population sizes. The Coalition will advocate for policy and regulatory frameworks that account for and recognize the distinct considerations of life science companies operating in the rare disease space and the impact it has on delivering innovative therapies to the patients they serve.

    Founding members of the Coalition include:

    • Acceleron Pharma
    • Aeglea BioTherapeutics
    • Agios Pharmaceuticals
    • Alnylam Pharmaceuticals
    • Harmony Biosciences
    • Orchard Therapeutics
    • Orphazyme US, Inc.
    • Sarepta Therapeutics
    • Taysha Gene Therapies
    • Ultragenyx Pharmaceutical

    In the coming month, the Coalition will engage with policy stakeholders on potentially impactful drug and healthcare policies and regulations currently under discussion, including prescription drug pricing, to highlight the consequences that blanket legislation can have on continued innovation for rare disease treatments.

    About the Rare Disease Company Coalition

    The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit www.rarecoalition.com.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  19. Mary Newman, former SVP of Regulatory Affairs at Astellas Gene Therapies, joins Taysha with over 30 years of experience in regulatory affairs and research and development in the biotech industry

    Extensive experience deeply rooted in gene therapy at Astellas Gene Therapies, Audentes Therapeutics and BioMarin

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer. Ms. Newman…

    Mary Newman, former SVP of Regulatory Affairs at Astellas Gene Therapies, joins Taysha with over 30 years of experience in regulatory affairs and research and development in the biotech industry

    Extensive experience deeply rooted in gene therapy at Astellas Gene Therapies, Audentes Therapeutics and BioMarin

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer. Ms. Newman will oversee program and portfolio management, as well as translational sciences, and will report to Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Taysha's Chief Medical Officer and Head of Research and Development.

    "We are pleased to welcome Mary to the Taysha leadership team," said RA Session II, President, Founder and CEO of Taysha. "In the past few months, we have significantly expanded our extremely talented R&D team, and Mary is an important addition to support the achievement of our ambitious pipeline advancement goals. Her decades of experience in all key aspects of drug development, particularly in gene therapy and rare disease, will be invaluable as we continue to grow our business."

    Ms. Newman joins Taysha with more than 30 years of experience in regulatory affairs and research and development within the biotechnology industry, focusing on rare diseases. Most recently, she served as Senior Vice President of Regulatory Affairs at Astellas Gene Therapies (formerly Audentes Therapeutics), where she oversaw global regulatory strategic development, all primary regulatory agency interactions, and regulatory compliance for Audentes' development candidates. Prior to joining Audentes, Ms. Newman served as the Senior Vice President, Regulatory Affairs and Quality Assurance at SARcode Bioscience Inc., and was responsible for the development of Xiidra® for the treatment of dry eye disease. The company was acquired by Shire Ltd. She previously held various management positions, with increasing responsibility, in Regulatory Affairs at BioMarin Pharmaceutical, Inc., Berlex Inc. (now Bayer HealthCare Pharmaceuticals Inc.), and Sequus Pharmaceuticals, Inc. (now Johnson & Johnson). While at BioMarin, Ms. Newman oversaw the development and approval of Kuvan® for the treatment of phenylketonuria (PKU), Naglazyme® for mucopolysaccharidosis (MPS) VI, and supported the final approval of Aldurazyme® for MPS I. She has also held various research and development leadership roles in oncology, neurology, and antifungal therapeutic areas. Ms. Newman serves as a member of the board of directors of Vedere Bio and is an advisor to the board of directors of Chameleon Biosciences. Ms. Newman holds a B.S. in Physiology from Oregon State University.

    "I am excited to join Taysha at such a transformative time in the company's corporate lifecycle," said Ms. Newman. "Taysha is well-positioned to be a leader in developing disease-modifying gene therapies for patients with monogenic CNS diseases. I look forward to contributing to the further progression of the company's robust pipeline of promising drug candidates."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  20.  2021 RBC Capital Markets Global Healthcare Conference on May 19, 2021 at 3:05 pm ET

    Oppenheimer Rare & Orphan Disease Summit on Friday May 21, 2021 at 4:35 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference and a virtual presentation at the Oppenheimer Rare & Orphan Disease Summit.

    Conference Details:

    Event:

    2021 RBC Capital Markets Global Healthcare Conference

    Date:

     2021 RBC Capital Markets Global Healthcare Conference on May 19, 2021 at 3:05 pm ET

    Oppenheimer Rare & Orphan Disease Summit on Friday May 21, 2021 at 4:35 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference and a virtual presentation at the Oppenheimer Rare & Orphan Disease Summit.

    Conference Details:

    Event:

    2021 RBC Capital Markets Global Healthcare Conference

    Date:

    Wednesday, May 19, 2021

    Time:

    3:05 pm ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of Research and Development

     

    Kamran Alam, Chief Financial Officer

     

     

    Event:

    Oppenheimer Rare & Orphan Disease Summit

    Date:

    Friday, May 21, 2021

    Time:

    4:35 pm ET

    Format:

    Presentation

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of Research and Development

     

    Kamran Alam, Chief Financial Officer

    Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  21. TSHA-120 program demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy (GAN); Expects clinical data from highest dose cohort in second half of 2021 and regulatory feedback from agencies by year-end 2021

    Expects to have five programs in Phase 1/2 trials in the second half of 2021, including GAN, GM2 gangliosidosis, CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome

    Positive preclinical data for TSHA-102 in Rett syndrome provided quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome; Results published

    TSHA-120 program demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy (GAN); Expects clinical data from highest dose cohort in second half of 2021 and regulatory feedback from agencies by year-end 2021

    Expects to have five programs in Phase 1/2 trials in the second half of 2021, including GAN, GM2 gangliosidosis, CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome

    Positive preclinical data for TSHA-102 in Rett syndrome provided quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome; Results published in Brain

    Treatment with TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5 week-old knockout Rett mice with meaningful accumulated disease, a more translatable model of the disorder in humans

    New data for multiple preclinical programs, including tauopathies, SLC13A5 deficiency, SLC6A1 haploinsufficiency, Angelman disease, Adult Polyglucosan Body Disease (APBD), Lafora disease, and GM2 AB variant, highlighted Taysha's next wave of novel gene therapies that have the potential to impact meaningful patient populations

    Plans for IND/CTA submission from one of the following programs by year-end 2021: SLC13A5 deficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

    Advancing development of multiple preclinical programs, including tauopathies and Angelman syndrome

    Virtual Research and Development Day on June 28-29, 2021 to feature Key Opinion Leaders and highlight progress across pipeline

    Conference call and webcast today at 8:00 AM Eastern Time

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported financial results for the first quarter ended March 31, 2021 and provided a corporate update.

    "Our team has ushered in the new year with a continued focus on achieving our corporate objectives and creating value for patients and shareholders," said RA Session II, President, Founder and CEO of Taysha. "Our recent acquisition of TSHA-120 for GAN immediately transformed Taysha into a pivotal-stage gene therapy company. Based on the compelling clinical and preclinical data package generated to date for this promising product candidate, we intend to engage with regulatory agencies to discuss a pathway to approval and look forward to providing clinical and regulatory updates in the second half of 2021 and by year-end 2021, respectively. We are also extremely pleased to share newly published preclinical data for TSHA-102 in Rett syndrome that provided, for the first time, quantitative evidence of miRARE's ability to show genotype-dependent regulation of MECP2 gene expression across different regions of the brain in wild type and knockout mouse models. Rett syndrome is an incredibly difficult disease to treat with gene replacement therapy given the challenges of safely regulating the degree of MECP2 expression from the MECP2 gene and we are encouraged that miRARE has achieved this regulation on a cell-by-cell basis without associated toxicities. The built-in self-regulatory feedback loop mechanism is a culmination of approximately 14 years of research and holds great potential for the treatment of what we now consider a reversible disease. Importantly, TSHA-102-treated knockout Rett mice with meaningful disease accumulation experienced a statistically significant survival extension by 56%, which we believe is a more translatable model of the disorder in humans. We believe these data validate our novel approach to treating Rett syndrome, help de-risk the clinical program and support advancement of TSHA-102 into a Phase 1/2 trial by year-end. With these and other recent value-creating achievements, we are even more confident in our outlook for the full year."

    Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha, said, "We expect a steady flow of near-term clinical, regulatory and preclinical catalysts for the remainder of 2021. We anticipate having five programs in Phase 1/2 trials and an additional six programs in IND/CTA-enabling studies by year-end 2021. For the remainder of 2021, we expect data from the highest dose cohort from the ongoing TSHA-120 trial for GAN, a regulatory update on the GAN program, first-in-human Phase 1/2 clinical data from the Queen's University trial of TSHA-101 in GM2 gangliosidosis and the initiation of Phase 1/2 trials in GM2 gangliosidosis in the U.S., CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome. For our preclinical programs, we expect to submit an IND/CTA for one of six programs in IND/CTA-enabling studies by year-end 2021. Moreover, we will continue to make advancements on payload design and on the miRARE, mini-gene and vagus nerve redosing platforms, which are expected to drive further innovation. We look forward to providing additional updates at our two-day R&D Day event in June and throughout the year."

    Recent Corporate Highlights

    • Published new preclinical data for TSHA-102 in Rett syndrome in Brain journal
      • Preclinical data provided quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome
      • TSHA-102 resulted in a statistically significant survival extension by 56%, whereas unregulated constructs did not extend survival significantly in the validated MECP2 knockout Rett mouse model
      • Benefit in 4-5 week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease should be a more translatable model to humans
      • In the pons and midbrain, miRARE inhibited mean expression in a genotype-dependent manner, as indicated by significantly fewer myc(+) cells observed in wild type mice than knockout mice (p<0.05), thereby demonstrating MECP2 levels within normal physiological parameters
      • These quantitative data, for the first time, demonstrated miRARE's ability to regulate gene expression on a cell-by-cell basis, highlighting its potential application in numerous diseases that require controlled gene expression
    • Acquired exclusive worldwide rights to TSHA-120, a clinical-stage AAV9 gene therapy program for the treatment of GAN
      • Human proof-of-concept data for TSHA-120 demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with GAN
      • To date, 14 patients have been dosed with one of four dose levels of TSHA-120. TSHA-120 has demonstrated a dose-response relationship with arrest of disease progression at the second-highest dose level (1.8x1014 total vector genomes [vg]) at one-year post-treatment, affecting a statistically significant 8-point improvement on the MFM32 score
      • Six of these patients treated at therapeutic dose levels have shown sustained dose-dependent improvements in MFM32 scores for more than three years
      • Bayesian analyses confirmed nearly 100% probability of clinically meaningful slowing of disease in patients dosed at 1.8x1014 total vg compared to natural history
      • Long-term results demonstrated that treatment with TSHA-120 at multiple dose levels was well-tolerated with no severe drug-related adverse events
    • Reported new preclinical data for TSHA-113 for tauopathies, TSHA-105 for SLC13A5 deficiency, TSHA-103 for SLC6A1 haploinsufficiency, TSHA-106 for Angelman syndrome, TSHA-112 for APBD, TSHA-111-LAFORIN and TSHA-111-MALIN for Lafora disease, and TSHA-119 for GM2 AB variant that support advancement into clinical testing
      • TSHA-113 significantly reduced tau mRNA and protein levels in mouse models of human tauopathies via cerebral spinal fluid (CSF) delivery
      • TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice
      • TSHA-103 improved nesting and EEG activity in the SLC6A1 knockout mouse model and reduced spike train activity in SLC6A1 knockout and heterozygous mouse models
      • TSHA-106 increased UBE3A expression through shRNA-mediated knockdown of UBE3A-ATS in in vitro cell lines across 26 distinct shRNA candidates for the treatment of Angelman disease
      • TSHA-112 generated significant reductions in GYS1 protein, abnormal glycogen accumulation and polyglucosan bodies in the APBD knockout mouse model
      • TSHA-111-LAFORIN and TSHA-111-MALIN achieved effective knockdown of GYS1 expression and insoluble glycogen and decreased Lafora body formation in laforin and malin mouse models
      • TSHA-119 caused a dose-dependent reduction of GM2 accumulation at 20 weeks in GM2A knockout mice
    • Announced presentations of preclinical data for TSHA-104 in SURF1-associated Leigh syndrome and TSHA-105 in SLC13A5 deficiency at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
      • TSHA-104 increased COX1 activity in brain and muscle and restored elevation of blood lactate on exhaustive exercise in a dose-dependent manner in SURF1 knockout mice
      • TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice
    • Initiated construction of its internal 187,000-square-foot current Good Manufacturing Practices (cGMP) manufacturing facility in Durham, North Carolina, that will include multiple production suites designed to have a total capacity of 2,000 liters for preclinical, clinical, and commercial production of Taysha's gene therapy pipeline; facility will include development, analytical, manufacturing and quality control testing capability for its broad portfolio of gene therapies
    • Established collaboration with Yale University, a key addition to partnerships with Cleveland Clinic and UTSW, to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of genetic epilepsies and neurodevelopmental disorders
    • Grew company from 80 to approximately 120 employees between February and April 2021
    • Announced a two-day virtual R&D Day on June 28th and June 29th to feature Key Opinion Leaders and highlight progress across the pipeline

    First Quarter 2021 Financial Highlights

    Research and Development (R&D) Expenses: R&D expenses were $23.9 million for the first quarter ended March 31, 2021, compared to $5.5 million for the first quarter ended March 31, 2020. The increase was primarily related to the company's development programs, as a result of increased manufacturing-related spend, clinical and preclinical activities, and headcount.

    General and Administrative (G&A) Expenses: G&A expenses were $8.2 million for the first quarter ended March 31, 2021, compared to less than $0.1 million for the first quarter ended March 31, 2020. The increase was primarily due to an increase in personnel costs resulting from increased headcount, professional services fees, and other corporate-related expenses.

    Net loss: Net loss for the first quarter ended March 31, 2021 was $32.0 million, or $0.87 per share, as compared to a net loss of $5.4 million, or $0.50 per share, for the first quarter ended March 31, 2020.

    Cash and cash equivalents: As of March 31, 2021, Taysha had $228.7 million in cash and cash equivalents, which is expected to support planned operations into 2023.

    Anticipated Milestones by Program

    TSHA-120 for giant axonal neuropathy (GAN): an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN, a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin

    • Report clinical data for TSHA-120 from the 3.5x1014 total vg dose cohort in the second half of 2021
    • Engage with major regulatory agencies to discuss the approval pathway and provide a regulatory update by year-end 2021

    TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes – HEXA and HEXB, comprising the alpha and beta sub-units of Beta Hexoseaminidase A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

    • Report preliminary Phase 1/2 safety and biomarker data (Queen's University trial) in the second half of 2021
    • Submit an Investigational New Drug (IND) application in the U.S. in the second half of 2021
    • Initiate Phase 1/2 clinical trial in the U.S. in the second half of 2021

    TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments

    • Maintain current open IND
    • Initiate a Phase 1/2 clinical trial in the second half of 2021
    • Report biomarker data in the first half of 2022

    TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for a severe neurodevelopmental disorder, designed to deliver MECP2, as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis

    • Submit IND/CTA filing in the second half of 2021
    • Initiate Phase 1/2 clinical trial by year-end 2021
    • Report clinical data by year-end 2022

    TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments

    • Submit IND/CTA filing in the second half of 2021
    • Initiate Phase 1/2 trial by year-end 2021
    • Report biomarker data in the first half of 2022

    Pipeline programs in IND/CTA-enabling studies

    • Submit an IND/CTA filing for one of six programs in 2021: TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN and TSHA-111-MALIN in two forms of Lafora disease, TSHA-112 in APBD, TSHA-119 in GM2 AB variant and TSHA-103 in SLC6A1 haploinsufficiency disorder

    Discovery programs

    • Advance four new undisclosed programs focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases into preclinical development in 2021

    Next-generation technology platform

    • Continue development efforts focused on regulated transgene expression with expansion of miRARE platform into additional CNS diseases
    • Initiate confirmatory preclinical studies for the vagus nerve redosing platform in canines
    • Advance mini-gene discovery program in genetic forms of epilepsy and neurodevelopmental disorders
    • Continue discovery and development efforts around next-generation capsids

    Anticipated Corporate Milestones in 2021

    • Continue construction of internal cGMP facility in 2021
    • Complete buildout of Dallas headquarters in Q2 2021
    • Expand employee base from approximately 120 (as of April 30, 2021) to approximately 150 by year-end 2021

    Conference Call and Webcast Information

    Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a corporate update. The dial-in number for the conference call is 855-327-6837 (U.S./Canada) or 631-891-4304 (international). The conference ID for all callers is 10014460. The live webcast and replay may be accessed by visiting Taysha's website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    Taysha Gene Therapies, Inc.

    Consolidated Statements of Operations

    (in thousands, except share and per share data)

    (Unaudited)

     

     

     

     

     

     

     

    For the Three Months

    Ended March 31, 2021

     

    For the Three Months

    Ended March 31, 2020

    Operating expenses:
    Research and development

    $

    23,854

     

    $

    5,514

     

    General and administrative

     

    8,236

     

     

    70

     

    Total operating expenses

     

    32,090

     

     

    5,584

     

    Loss from operations

     

    (32,090

    )

     

    (5,584

    )

    Other income (expense):
    Change in fair value of preferred stock tranche liability

     

    -

     

     

    180

     

    Interest income

     

    66

     

     

    -

     

    Interest expense

     

    -

     

     

    (27

    )

    Total other income, net

     

    66

     

     

    153

     

    Net loss

    $

    (32,024

    )

    $

    (5,431

    )

    Net loss per common share, basic and diluted

    $

    (0.87

    )

    $

    (0.50

    )

    Weighted average common shares outstanding, basic and diluted

     

    36,992,377

     

     

    10,894,999

     

     

    Taysha Gene Therapies, Inc.

    Consolidated Balance Sheet Data

    (in thousands)

    (Unaudited)

     

     

     

     

     

     

     

    March 31,

     

    December 31,

     

     

    2021

     

    2020

    Cash and cash equivalents

    $

    228,684

    $

    251,253

    Total assets

    $

    242,829

    $

    258,881

    Total liabilities

    $

    19,957

    $

    7,579

    Total stockholders' equity

    $

    222,872

    $

    251,302

     

    View Full Article Hide Full Article
  22. Preclinical data provide quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome

    TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5-week-old knockout Rett mice with meaningful accumulated disease whereas unregulated constructs did not extend survival significantly in the validated MECP2 knockout Rett mouse model

    Significant survival benefit in 4-5-week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease a more translatable model of the disorder in humans

    These quantitative data, for the first time, demonstrated miRARE's ability to regulate gene

    Preclinical data provide quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome

    TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5-week-old knockout Rett mice with meaningful accumulated disease whereas unregulated constructs did not extend survival significantly in the validated MECP2 knockout Rett mouse model

    Significant survival benefit in 4-5-week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease a more translatable model of the disorder in humans

    These quantitative data, for the first time, demonstrated miRARE's ability to regulate gene expression on a cell-by-cell basis, highlighting its potential application in numerous dose-sensitive diseases

    Data support an IND/CTA filing for TSHA-102 in Rett syndrome in the second half of 2021, with initiation of a Phase 1/2 trial anticipated by year-end

    Estimated 25,000 patients in U.S. and Europe represent multi-billion-dollar commercial opportunity

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new preclinical data for TSHA-102 in Rett syndrome. The data were published online and will be included in the May edition of Brain, a highly esteemed neurological science peer-reviewed journal.

    "Effective gene therapy targeting MECP2 for the treatment of Rett syndrome has been elusive due to the inability to properly regulate transgene expression," said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. "The built-in self-regulatory feedback loop mechanism in TSHA-102, work that was initiated in my laboratory in 2007, is a completely novel approach that allows for regulated expression of MECP2 on a cell-by-cell basis. These results published today are highly encouraging and allow us to conceive additional novel approaches using miRARE in conditions with dose-sensitive genes."

    "The complexities of developing an efficacious and well-tolerated gene therapy for the treatment of Rett syndrome are highlighted by phenotypic variability, mosaicism and the need to regulate MECP2 such that it does not cause overexpression-related toxicity. Today's data give us confidence that we can achieve appropriate MECP2 expression in all cells in a genotype-dependent manner, which we believe significantly de-risks the developmental program in its translation to humans," said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "Historically, unregulated gene replacement of MECP2 resulted in overt adverse events, including death in wild type mice due to overexpression of the MECP2 protein. With the built-in regulatory element, miRARE, TSHA-102 provided a statistically significant survival extension by 56% in 4- to 5-week-old knockout Rett mice. We see the potential for broadening the miRARE platform to other CNS diseases requiring regulated gene expression. These positive data support our intent to file an IND/CTA for TSHA-102 in the second half of this year, followed by initiation of a Phase 1/2 trial by year-end 2021. TSHA-102 has the potential to address a significant unmet need for an estimated 25,000 patient with Rett syndrome across the United States and in Europe."

    The preclinical study was conducted by the UT Southwestern Medical Center (UT Southwestern) laboratory of Sarah Sinnett, Ph.D., and evaluated the safety and efficacy of regulated miniMECP2 gene transfer, TSHA-102 (AAV9/miniMECP2-miRARE), via intrathecal (IT) administration in adolescent mice between four and five weeks of age. TSHA-102 was compared to unregulated full length MECP2 (AAV9/MECP2) and unregulated miniMECP2 (AAV9/miniMECP2).

    TSHA-102 extended knockout survival by 56% via IT delivery. In contrast, the unregulated miniMECP2 gene transfer failed to significantly extend knockout survival at either dose tested. Additionally, the unregulated full-length MECP2 construct did not demonstrate a significant extension in survival and was associated with an unacceptable toxicity profile in wild type mice.

    In addition to survival, behavioral side effects were explored. Mice were subjected to phenotypic scoring and a battery of tests including gait, hindlimb clasping, tremor and others to comprise an aggregate behavioral score. miRARE attenuated MECP2-mediated aggravation in wild type aggregate phenotype severity scores. Mice were scored on an aggregate severity scale using an established protocol. AAV9/MECP2- and AAV9/miniMECP2-treated wild type mice had a significantly higher mean (worse) aggregate behavioral severity score versus that observed for saline-treated mice (p <0.05; at 6–30 and 7–27 weeks of age, respectively). TSHA-102-treated wild type mice had a significantly lower (better) mean aggregate severity score versus those of AAV9/MECP2- and AAV9/miniMECP2-treated mice at most timepoints from 11–19 and 9–20 weeks of age, respectively. No significant difference was observed between saline- and TSHA-102-treated wild type mice.

    Of note, miRARE-mediated genotype-dependent gene regulation was demonstrated by analyzing tissue sections from wild type and knockout mice treated with AAV9 vectors given intrathecally. TSHA-102 demonstrated regulated MECP2 expression in different regions of the brain. In the pons and midbrain, miRARE inhibited mean MECP2 gene expression in a genotype-dependent manner as indicated by significantly fewer myc(+) cells observed in wild type mice compared to knockout mice (p<0.05), thereby demonstrating that TSHA-102 achieved MECP2 expression levels similar to normal physiological parameters.

    "It has been a challenge finding an approach that can appropriately regulate MECP2 expression in Rett syndrome but the preclinical data for TSHA-102 published today support the miRARE approach," said Sarah Sinnett, Ph.D., Assistant Professor in the Department of Pediatrics at UT Southwestern. "It is clear that the disease is reversible, and I am encouraged that this novel strategy may enable us to make a difference in the management of this disease."

    The publication is available by clicking on the following link: https://academic.oup.com/brain/advance-article-abstract/doi/10.1093/brain/awab182/6265600.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Drs. Gray and Sinnett have intellectual property interest in Taysha and UTSW has a financial interest in the company.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-102, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  23. TSHA-104 increased COX1 activity in brain and muscle and restored elevation of blood lactate on exhaustive exercise in dose-dependent manner in SURF1 knockout mice

    TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice

    On track to file IND/CTA for TSHA-104 in SURF1-associated Leigh syndrome in second half of 2021

    IND/CTA-enabling studies for TSHA-105 in SLC13A5 deficiency are ongoing

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in…

    TSHA-104 increased COX1 activity in brain and muscle and restored elevation of blood lactate on exhaustive exercise in dose-dependent manner in SURF1 knockout mice

    TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice

    On track to file IND/CTA for TSHA-104 in SURF1-associated Leigh syndrome in second half of 2021

    IND/CTA-enabling studies for TSHA-105 in SLC13A5 deficiency are ongoing

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that preclinical data from its investigational gene therapy programs will be presented at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), which will be held virtually May 11-14, 2021.

    "Presentations at this year's ASGCT will highlight the positive preclinical results for TSHA-104 in SURF1-associated Leigh syndrome and TSHA-105 in SLC13A5 deficiency that support our advancement of these programs," said RA Session II, President, Founder and Chief Executive Officer of Taysha. "Among the compelling data, we have shown that TSHA-104 as a single intrathecally administered gene replacement therapy was effective in improving SURF1 deficiency-related dysfunctions, such as diminished COX1 activity in brain and muscle and blood lactate on exhaustive exercise in a dose-dependent manner in SURF1 knockout mice. In SLC13A5 deficiency, CSF-delivered TSHA-105 resulted in improved EEG activity and reduced seizure susceptibility in SLC13A5 knockout mice. We remain on track to file an IND/CTA in SURF1-associated Leigh syndrome in the second half of this year and continue to advance TSHA-105 in SLC13A5 deficiency towards the clinic."

    Summary of Abstracts and Posters (all times in Eastern Time)

    Thursday, May 13, 2021 at 7:00 – 7:15 pm

    • Abstract Presentation title: Gene Replacement Therapy for SURF1-Related Leigh Syndrome Using AAV9
    • Session title: Clinical Trials and Advanced Preclinical Studies for Neurologic Diseases
    • Abstract number: 165
    • Authors: Qinglan Ling, Matthew Rioux, Steven Gray
    • Presenter: Qinglan Ling, Ph.D., of UT Southwestern Medical Center
      • COX activity was partially and significantly rescued in all tested tissues of AAV9/hSURF1-treated mice via intrathecal (IT) delivery
      • AAV9/hSURF1-treated mice demonstrated a dose-dependent increase in hSURF1 mRNA expression, restoration of MT-CO1 protein expression in the brain
      • Gene replacement treatment also mitigated the lactic acidosis upon exhaustive exercise at mid-age

    Thursday, May 13, 2021 at 7:00 – 7:15 pm

    • Abstract Presentation title: scAAV9 Gene Replacement Therapy for Epileptic SLC13A5 Deficiency
    • Session title: AAV Therapies for Neurological and Sensory Diseases
    • Abstract number: 137
    • Authors: Rachel Marion Bailey, Lauren Bailey, Morgan Schackmuth, Irvin Garza
    • Presenter: Rachel Bailey, Ph.D., of UT Southwestern Medical Center
      • TSHA-105-treated knockout mice demonstrated significantly decreased plasma citrate levels compared to knockout mice treated with vehicle
      • TSHA-105-treated knockout mice had reduced spike train activity and seizure frequency on electroencephalogram (EEG)

    Tuesday, May 11, 2021 at 8:00 – 10:00 am

    • Poster title: Novel AAV Capsids for Enhanced Gene Transfer to the Cerebellum, Spinal Cord, and Schwann Cells
    • Session: AAV Vectors – Virology and Vectorology
    • Abstract number: 314
    • Authors: Xin Chen, Thomas Dong, Widler Casy, Yuhui Hu, Daphne Chen, Thomas McCown, Steven Gray
      • A capsid DNA shuffling and directed evolution process was pursued to generate new AAV variants for nervous system gene transfer. In the IT cohort, more than 15 variants had biodistribution values at least ten times greater than AAV9 with reduced biodistribution to the liver
      • Results demonstrated that some variants could be used to treat cerebellar diseases with Purkinje cell involvement and other variants could be used to treat peripheral demyelinating neuropathies with Schwann cell involvement

    The ASGCT abstracts are now available at https://www.cell.com/molecular-therapy-family/molecular-therapy/issue?pii=S1525-0016(21)X0002-0.

    ASGCT-Sponsored Pre-Meeting Workshops (all times in Eastern Time)

    Monday, May 10, 2021 at 12:55 – 1:05 pm

    • Workshop title: Transitioning From Academics to Industry
    • Session 2: Learning From Experience: Case Studies of Transitions from Academia to Industry
    • Topic: Moving from Academic Vector Production to Commercial Scale Manufacturing
    • Speaker: Frederick Porter, Ph.D., Chief Technical Officer of Taysha Gene Therapies

    Monday, May 10, 2021 at 3:00 – 3:15 pm

    • Workshop title: Emerging Issues in Market Access
    • Session 1: Gene Therapy Investment and Capital
    • Keynote: Attracting Capital and Building a Company in the Gene Therapy Space
    • Speaker: RA Session II, President, Founder and Chief Executive Officer of Taysha Gene Therapies

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  24. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) ("Taysha"), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the first quarter ended March 31, 2021, and host a corporate update conference call and webcast on Tuesday, May 11, 2021, at 8:00 AM Eastern Time.

    Conference Call Details
    Tuesday, May 11, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

     

    855-327-6837

    International:

     

    631-891-4304

    Conference ID:

     

    10014460

    Webcast:

     

    https://ir.tayshagtx.com/news-events/events-presentations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA) ("Taysha"), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for the first quarter ended March 31, 2021, and host a corporate update conference call and webcast on Tuesday, May 11, 2021, at 8:00 AM Eastern Time.

    Conference Call Details
    Tuesday, May 11, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

     

    855-327-6837

    International:

     

    631-891-4304

    Conference ID:

     

    10014460

    Webcast:

     

    https://ir.tayshagtx.com/news-events/events-presentations

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  25. TSHA-113 significantly reduced tau mRNA and protein levels in mouse models of human tauopathies via cerebral spinal fluid (CSF) delivery supporting further preclinical development

    TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice

    TSHA-106 increased UBE3A expression through shRNA-mediated knockdown of UBE3A-ATS in in vitro cell lines across 26 distinct shRNA candidates for the treatment of Angelman disease

    TSHA-112 generated significant reductions in GYS1 protein, abnormal glycogen accumulation and polyglucosan bodies in the APBD knockout mouse model

    TSHA-111-LAFORIN and TSHA-111-MALIN achieved effective knockdown of

    TSHA-113 significantly reduced tau mRNA and protein levels in mouse models of human tauopathies via cerebral spinal fluid (CSF) delivery supporting further preclinical development

    TSHA-105 significantly reduced plasma citrate levels, normalized EEG brain activity, and reduced the number of seizures and seizure susceptibility in SLC13A5 knockout mice

    TSHA-106 increased UBE3A expression through shRNA-mediated knockdown of UBE3A-ATS in in vitro cell lines across 26 distinct shRNA candidates for the treatment of Angelman disease

    TSHA-112 generated significant reductions in GYS1 protein, abnormal glycogen accumulation and polyglucosan bodies in the APBD knockout mouse model

    TSHA-111-LAFORIN and TSHA-111-MALIN achieved effective knockdown of GYS1 expression and insoluble glycogen and decreased Lafora body formation in laforin and malin mouse models

    TSHA-110 caused a dose-dependent reduction of GM2 accumulation at 20 weeks in GM2A knockout mice

    Positive proof-of-concept data for gene therapy candidates in SCL13A5 deficiency, APBD, Lafora disease and GM2 AB variant support advancement into clinical testing

    Expect to submit IND/CTA for one of the following programs by the end of 2021: SLC13A5 deficiency, APBD, Lafora disease or GM2 AB variant

    Taysha's virtual Research and Development Day in June 2021 will highlight progress across R&D pipeline

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced new data for multiple preclinical programs and a planned R&D Day, which will be held in June 2021.

    "Collectively, these new preclinical data highlight Taysha's next wave of novel gene therapies that have the potential to impact meaningful patient populations. The promising data underscore our ability to rapidly and reproducibly investigate disease biology, design innovative gene therapies and efficiently advance the development of these drug candidates," said RA Session II, President, Founder and Chief Executive Officer of Taysha. "Among the compelling new data, for the first time, we have shown that TSHA-113, an AAV9 gene therapy that utilizes AAV-mediated gene silencing, reduced tau expression in mouse models of human tauopathies. The potential implications of these data are far reaching, and we intend to further evaluate TSHA-113 in additional preclinical studies. The totality of the preclinical data presented today support the fundamental elements of our scientific approach of coupling validated technology with novel targeted payload design while utilizing a proven HEK293 suspension manufacturing process. We believe our deep pipeline and innovative scientific engine hold tremendous potential, and we are poised to continue delivering meaningful value to patients with monogenic CNS diseases."

    "Today's data demonstrate the breadth, depth and velocity of our development engine as a sustainable pivotal-stage gene therapy company. There are no approved disease modifying therapies for any of the programs in our portfolio and we are encouraged by the results of our gene therapy approach of vectorized RNA and gene replacement therapies across our portfolio," said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "We are very excited to further develop TSHA-113 in tauopathies, including Alzheimer's disease, MAPT-associated frontotemporal dementia and progressive supranuclear palsy, based on the significant reduction in tau expression demonstrated in transgenic mouse models of human tauopathies. In addition, to date, we have advanced five programs into IND/CTA-enabling studies, including TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN in Lafora disease, TSHA-111-MALIN in Lafora disease, TSHA-112 in APBD and TSHA-119 in GM2 AB variant. We intend to file an IND/CTA for one of these five named programs by the end of 2021. By mid-year, we intend to select a development candidate for Angelman syndrome and obtain interim expression and safety data from confirmatory non-human primate studies by year-end. We remain on track to report Phase 1/2 biomarker data for TSHA-101 in GM2 gangliosidosis in the second half of this year and to provide a clinical and regulatory update for TSHA-120 in giant axonal neuropathy by year-end. Finally, in the second half of the year, we continue to expect dosing of the first patient with CLN1 disease in a Phase 1/2 trial for TSHA-118 under an already open IND, filing an IND/CTA for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome, and filing an IND for TSHA-101 in GM2 gangliosidosis in the U.S. These anticipated clinical and regulatory milestones are expected to be followed by the initiation of Phase 1/2 clinical trials for each of these indications. We look forward to providing additional updates at our R&D Day in June."

    TSHA-113 for Tauopathies

    Taysha is developing tau-specific microRNA (miRNA) shuttles designed to target tau mRNA for all six isoforms found in the human brain and/or mouse brain. TSHA-113 is an AAV9 capsid that packages these miRNA shuttles and is delivered in the CSF for the treatment of tauopathies.

    • In transgenic mouse models carrying human tau, TSHA-113 significantly reduced tau mRNA and protein levels, while demonstrating widespread expression in neurons and glia
    • Together with previous in vitro findings, these data further validate selective reduction of tau mRNA and protein levels and warrant further preclinical development
    • An estimated 6.2 million Americans and 7.8 million Europeans are living with Alzheimer's disease
    • There are an estimated 13,000 patients in the U.S. and Europe affected by MAPT-associated frontotemporal dementia, progressive supranuclear palsy and corticobasal degeneration, which represent a significant commercial opportunity

    TSHA-105 for SLC13A5 deficiency

    TSHA-105 is a recombinant self-complementary AAV9 vector that expresses the human SLC13A5 protein under the control of a ubiquitous promoter. The drug candidate is being developed for the treatment of SLC13A5 deficiency.

    • In SLC13A5 knockout mice, treatment with TSHA-105 resulted in a significant, sustainable decrease of plasma citrate levels up to three months post-injection compared to age-matched, wildtype controls
    • TSHA-105 normalized electroencephalogram (EEG) brain activity, reduced the number of seizures, and reduced seizure susceptibility compared to vehicle-treated controls
    • The company has advanced TSHA-105 into IND/CTA-enabling studies
    • There are an estimated 1,900 patients with SLC13A5 deficiency in the United States and in Europe

    TSHA-106 for Angelman syndrome

    TSHA-106 is an intrathecally delivered AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS, the antisense transcript governing the expression of UBE3A through the paternal allele.

    • In vitro testing in a neuroblast cell line demonstrated consistent knockdown of UBE3A-ATS and a subsequent increase in UBE3A expression across 26 distinct shRNA candidates
    • Selection of development candidate expected by mid-year
    • Interim expression and safety data from confirmatory non-human primate (NHP) studies expected by the end of 2021
    • There are an estimated 55,000 patients with Angelman syndrome in the United States and in Europe

    TSHA-112 for Adult Polyglucosan Body Disease (APBD)

    TSHA-112 is an intrathecally delivered AAV9 viral vector designed for miRNA-mediated knockdown of the GYS1 gene to treat APBD.

    • In preclinical studies, miRNA knockdown of GYS1 induced significant reductions in GYS1 mRNA, GYS1 protein, abnormal glycogen accumulation, and polyglucosan bodies throughout the brain in an APBD knockout mouse model
    • TSHA-112 decreased neuroinflammatory markers across three distinct mouse models
    • The company has advanced TSHA-112 into IND/CTA-enabling studies
    • There are an estimated 10,000 patients with APBD in the United States and in Europe

    TSHA-111-LAFORIN for EPM2A and TSHA-111-MALIN for EPM2B for Lafora disease

    TSHA-111-LAFORIN and TSHA-111-MALIN are intrathecally delivered AAV9 viral vectors designed for miRNA-mediated knockdown of the GYS1 gene to treat Lafora disease.

    • In preclinical studies, TSHA-111-LAFORIN and TSHA-111-MALIN achieved effective knockdown of GYS1 expression and insoluble glycogen in the Lafora disease laforin and malin mouse models, respectively
    • Both product candidates decreased Lafora body formation within the brain in their respective mouse models
    • The company has advanced TSHA-111-LAFORIN and TSHA-111-MALIN into IND/CTA-enabling studies
    • There are an estimated 700 patients with Lafora disease in the United States and in Europe

    TSHA-119 for GM2 AB variant

    TSHA-119 is a self-complementary AAV9 vector designed to deliver a functional copy of the GM2A gene to treat GM2 AB variant.

    • In preclinical studies, TSHA-110 caused a significant, dose-dependent reduction of GM2 accumulation at 20 weeks in mice that were dosed intrathecally at postnatal day 1 or at 6 weeks of age
    • Long-term follow up studies, which include bi-monthly behavioral, as well as biochemical and histological analyses, are currently ongoing
    • The company has advanced TSHA-112 into IND/CTA-enabling studies
    • There are approximately 200 patients with GM2 AB variant in the United States and in Europe

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  26. Program invented in the lab of Dr. Steven Gray, Taysha's Chief Scientific Advisor, immediately transforms Taysha into a sustainable pivotal-stage gene therapy company

    Clinical and preclinical data package validates the scientific approach of Dr. Steven Gray, UT Southwestern, and Taysha, with readthrough to existing portfolio

    Groundbreaking clinical trial run by the NIH is the first intrathecally dosed gene therapy program in history

    Human proof-of-concept data for TSHA-120 demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy

    Plans to engage with regulatory agencies in the United States, Europe and Japan as soon as possible

    Estimated 2,400 patients

    Program invented in the lab of Dr. Steven Gray, Taysha's Chief Scientific Advisor, immediately transforms Taysha into a sustainable pivotal-stage gene therapy company

    Clinical and preclinical data package validates the scientific approach of Dr. Steven Gray, UT Southwestern, and Taysha, with readthrough to existing portfolio

    Groundbreaking clinical trial run by the NIH is the first intrathecally dosed gene therapy program in history

    Human proof-of-concept data for TSHA-120 demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy

    Plans to engage with regulatory agencies in the United States, Europe and Japan as soon as possible

    Estimated 2,400 patients in U.S. and Europe represent potentially greater than $2 billion near-term commercial opportunity

    Program provides basis for accelerating build-out of commercial infrastructure to support patient identification, payor engagement and product distribution

    Conference call and webcast today at 8:00 AM Eastern Time

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the acquisition of exclusive worldwide rights to a clinical-stage AAV9 gene therapy program, now known as TSHA-120, for the treatment of giant axonal neuropathy (GAN). TSHA-120 is an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN. The trial is being conducted by the National Institutes of Health (NIH) in close collaboration with a leading patient advocacy group focused on finding treatments and cures for GAN. TSHA-120 has received rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of GAN.

    GAN is a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin. Many children with GAN show symptoms and features before the age of five, including progressive scoliosis, contractures, atrophy of the spinal cord, giant axons – also known as nerve fibers – and abnormalities of the white matter in the brain. Currently, there are no approved treatments for GAN, which often results in death for patients in their late teens or early twenties. TSHA-120 was originally developed in the laboratory of Taysha's Chief Scientific Advisor, Dr. Steven Gray, and advanced in a historic ongoing clinical trial by the NIH as the first intrathecally dosed gene therapy study.

    "Dr. Steven Gray's work on the GAN program was the catalyst for all the other translational research initiatives in his lab and we are very pleased to continue this important and meaningful work that has had a significant impact across the entire gene therapy landscape. As the program that laid the foundation for our robust pipeline, we believe that TSHA-120 is a seamless strategic fit and will be immediately value-accretive for Taysha. TSHA-120 clinical data generated to date is a clear validation of our scientific approach with read-through to our existing product development pipeline. Consistent with all of our gene therapy candidates, TSHA-120 targets a monogenic CNS disease, is delivered intrathecally using a proven AAV9 vector, and utilizes a highly scalable HEK293 suspension manufacturing process. Collectively, these key parallels enable Taysha to leverage synergies across its core competencies to efficiently develop and commercialize TSHA-120," said RA Session II, President, Founder and Chief Executive Officer of Taysha. "The efficacy data in preclinical studies with the GAN knockout rodent model were extremely compelling, and TSHA-120 demonstrated significant improvement in pathology across a range of tissues, and notably improved the pathological appearance of the dorsal root ganglia, which is a key component of disease progression. We look forward to quickly working with the regulatory agencies on a path forward to approval of TSHA-120, and in parallel, accelerating the build-out of our commercial infrastructure to support patient identification, payor engagement and product distribution. TSHA-120 has the potential to address a significant unmet need for an estimated 2,400 patients with GAN across the United States and in Europe, potentially representing a near-term commercial opportunity of greater than $2 billion."

    The National Institute of Neurological Disorders and Stroke (NINDS) division of the NIH is conducting the ongoing open-label, non-randomized, dose-escalation clinical trial of TSHA-120 for the treatment of GAN. The primary endpoint is safety, with secondary endpoints measuring efficacy using pathologic, physiologic, functional, and clinical markers. A primary measure of clinical efficacy is the Motor Function Measure 32 (MFM32) score, a quantitative scale designed to assess the severity and progression of motor function abilities. There is precedent for its use in multiple clinical studies for neuromuscular diseases, including spinal muscular atrophy amongst others. To date, 14 patients have been dosed with one of four dose levels of TSHA-120. TSHA-120 has demonstrated a dose-response relationship with arrest of disease progression at the second-highest dose level (1.8x1014 total vector genomes [vg]) at one-year post-treatment, affecting a statistically significant 8-point improvement on the MFM32 score. A 4-point change on the MFM32 score is considered clinically meaningful. Six of these patients treated at therapeutic dose levels have shown sustained dose-dependent improvements in MFM32 scores for more than three years. Long-term results demonstrated that treatment with TSHA-120 at multiple dose levels was well-tolerated with no severe drug-related adverse events. Additional data are expected later this year, including results from the highest dose cohort (3.5x1014 total vg).

    "TSHA-120 is the first successful in-human intrathecal gene transfer in the history of gene therapy and, as such, has had a significant impact across the field. This program further supports our approach to treating monogenic diseases of the CNS and may enable us to pursue proof-of-concept for our redosing platform," said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "We are very encouraged by TSHA-120's halting effect on disease progression at therapeutic dose levels and long-term durability of effect in patients living with GAN, and we look forward to highlighting the initial clinical data in an R&D Day in June 2021. In the meantime, we plan to engage regulatory agencies in the United States, Europe and Japan as soon as possible."

    Before the end of the year, Taysha intends to request an End-of-Phase meeting with the FDA and engage with the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan to discuss the regulatory pathway for TSHA-120. Taysha expects to provide a regulatory and clinical update on TSHA-120, including data from the 3.5x1014 total vg cohort by year-end.

    Under the terms of the agreement, in exchange for granting Taysha the exclusive worldwide rights to TSHA-120, the leading GAN patient advocacy group will receive an upfront payment of $5.5 million and will be eligible to receive clinical, regulatory and commercial milestones totaling up to $19.3 million, as well as a low, single-digit royalty on net sales upon commercialization of the product.

    Conference Call and Webcast Information

    Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to review its acquisition of the GAN program. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13718632. The live webcast and replay may be accessed by visiting Taysha's website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-120, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-120's eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  27. 20th Annual Needham Virtual Healthcare Conference on Monday, April 12, 2021 at 10:15 am ET

    Chardan 5th Annual Manufacturing Summit on Monday, April 26, 2021 at 1:00 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats for the 20th Annual Needham Virtual Healthcare Conference and the Chardan 5th Annual Manufacturing Summit.

    Conferences Details:

    Event:

     

    20th Annual Needham Virtual Healthcare Conference

    Date:

     

    Monday, April 12, 2021

    20th Annual Needham Virtual Healthcare Conference on Monday, April 12, 2021 at 10:15 am ET

    Chardan 5th Annual Manufacturing Summit on Monday, April 26, 2021 at 1:00 pm ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats for the 20th Annual Needham Virtual Healthcare Conference and the Chardan 5th Annual Manufacturing Summit.

    Conferences Details:

    Event:

     

    20th Annual Needham Virtual Healthcare Conference

    Date:

     

    Monday, April 12, 2021

    Time:

     

    10:15 am ET

    Format:

     

    Fireside chat

    Participants:

     

    RA Session II, President, Founder and CEO

     

     

     

    Event:

     

    Chardan 5th Annual Manufacturing Summit

    Date:

     

    Monday, April 26, 2021

    Time:

     

    1:00 pm ET

    Format:

     

    Fireside chat

    Participants:

     

    RA Session II, President, Founder and CEO

     

     

    Fred Porter, Ph.D., Chief Technical Officer

     

     

    Greg Gara, Senior Vice President of Manufacturing

    Webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  28. Oppenheimer 31st Annual Healthcare Conference on Tuesday, March 16, 2021 at 1:50 pm ET

    Stifel's 3rd Annual CNS Day on Wednesday, March 31, 2021 at 10:30 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats for the Oppenheimer 31st Annual Healthcare Conference and Stifel's 3rd Annual CNS Day.

    Conferences Details:

    Event: Oppenheimer 31st Annual Healthcare Conference (Virtual)
    Date: Tuesday, March 16, 2021
    Time: 1:50 pm ET
    Format: Fireside chat
    Participants…

    Oppenheimer 31st Annual Healthcare Conference on Tuesday, March 16, 2021 at 1:50 pm ET

    Stifel's 3rd Annual CNS Day on Wednesday, March 31, 2021 at 10:30 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats for the Oppenheimer 31st Annual Healthcare Conference and Stifel's 3rd Annual CNS Day.

    Conferences Details:

    Event: Oppenheimer 31st Annual Healthcare Conference (Virtual)

    Date: Tuesday, March 16, 2021

    Time: 1:50 pm ET

    Format: Fireside chat

    Participants: RA Session II, President, Founder and CEO

    Event: Stifel's 3rd Annual CNS Day (Virtual)

    Date: Wednesday, March 31, 2021

    Time: 10:30 am ET

    Format: Fireside chat

    Participants: RA Session II, President, Founder and CEO

    Audio webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  29. Taysha to have an exclusive option on new payloads, constructs, and intellectual property arising from research conducted under the agreement

    Agreement leverages Taysha's existing partnership with UT Southwestern Gene Therapy Program to develop new constructs incorporating novel mini-gene payloads

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced a multi-year collaboration to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of neurodevelopmental disorders. Taysha…

    Taysha to have an exclusive option on new payloads, constructs, and intellectual property arising from research conducted under the agreement

    Agreement leverages Taysha's existing partnership with UT Southwestern Gene Therapy Program to develop new constructs incorporating novel mini-gene payloads

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced a multi-year collaboration to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of neurodevelopmental disorders. Taysha will have an exclusive option on new payloads, constructs, and intellectual property associated with, and arising from, the research conducted under this agreement.

    The collaboration with Yong-Hui Jiang, MD, Ph.D., Professor and Chief of Medical Genetics at Yale University, follows the previously announced collaborations with Cleveland Clinic and UT Southwestern Gene Therapy Program (UTSW) to support the creation of a novel next-generation mini-gene platform designed to overcome key challenges in gene therapy. Under the terms of this most recent agreement, a team of researchers from Yale University will create mini-gene payloads designed to treat neurodevelopmental disorders including intellectual disability. UTSW will produce viral vector constructs that incorporate the mini-gene payloads and evaluate the constructs in in vivo and in vitro efficacy models.

    "Our collaboration with Yale is a key addition to our established partnerships with Cleveland Clinic and UTSW, designed to advance our breakthrough next-generation mini-gene platform and to potentially expand the range of genetic diseases that can be treated by AAV-based gene therapy," said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "We look forward to harnessing each partner's unique capabilities and expertise to better address the challenge of vector capacity and believe our collective efforts may allow significant advancement in the field of gene therapy."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our collaborations with Yale University, the Cleveland Clinic and UTSW, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  30. Expects preliminary Phase 1/2 safety and biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis from the Queen's University study in second half of 2021 and by year-end 2021, respectively

    Plans to initiate Phase 1/2 clinical trial for TSHA-118 in CLN1 disease in the second half of 2021 and Phase 1/2 clinical trials for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end

    Plans to initiate a U.S. Phase 1/2 clinical trial for TSHA-101 in GM2 gangliosidosis in second half of 2021

    Expects IND/CTA submission from one of the following programs: SLC13A5 deficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

    Conference call and webcast today at 8:00 AM Eastern Time

    Taysha…

    Expects preliminary Phase 1/2 safety and biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis from the Queen's University study in second half of 2021 and by year-end 2021, respectively

    Plans to initiate Phase 1/2 clinical trial for TSHA-118 in CLN1 disease in the second half of 2021 and Phase 1/2 clinical trials for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end

    Plans to initiate a U.S. Phase 1/2 clinical trial for TSHA-101 in GM2 gangliosidosis in second half of 2021

    Expects IND/CTA submission from one of the following programs: SLC13A5 deficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

    Conference call and webcast today at 8:00 AM Eastern Time

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported financial results for the full-year ended December 31, 2020 and provided a corporate update.

    "In 2020, we successfully completed one of the fastest seed to IPOs in biotech history and made significant strides in advancing our pipeline initiatives, next-generation technology platforms and manufacturing strategy," said RA Session II, President, Founder and CEO of Taysha. "We expect 2021 to be a transformational year for Taysha, as we anticipate reporting first-in-human clinical data for TSHA-101 in GM2 gangliosidosis and initiating Phase 1/2 trials in CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome. We will be submitting multiple IND/CTAs across three CNS franchises, and advancing four gene therapies into IND-/CTA-enabling studies and initiating work on additional targets. We continue to enhance our platform of next-generation technologies to optimize key components of our approach with AAV-based gene therapies. With recent additions to our team and Board of Directors and the formation of our Scientific Advisory Board, we believe we have the resources needed to achieve our corporate initiatives this year. Lastly, we will continue to evaluate other opportunities to maximize the value of our pipeline and technology platforms. We look forward to providing updates on our progress throughout the year and at an R&D day later this year."

    2020 Corporate Highlights and Updates

    Since inception a year ago, the company has achieved its strategic priorities, as highlighted by:

    • Health Canada's approval of Queen's University's clinical trial application (CTA) for the Phase 1/2 clinical trial of TSHA-101 for the treatment of GM2 gangliosidosis
    • Expanded portfolio of CNS gene therapy programs across three distinct franchises from 18 to 25 product candidates
    • A portfolio of rare pediatric disease and orphan drug designations obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, TSHA-104 for SURF1-associated Leigh syndrome, TSHA-105 for epilepsy caused by SLC13A5 deficiency and TSHA-118 for CLN1 disease
    • Continued progress on next-generation technology platform, including achievement of animal proof-of-concept for vagus nerve redosing
    • Expanded partnership with UT Southwestern (UTSW) through launch of an innovation fund to accelerate advancement of AAV gene therapies for the treatment of monogenic diseases of the CNS
    • Collaboration established with Cleveland Clinic and UTSW to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of genetic epilepsies and additional CNS disorders
    • Collaborations established with Invitae and AllStripes to support access to genetic testing and earlier diagnosis of patients with CNS diseases and to inform the understanding of the natural history and disease burden of and patients' diagnostic journeys with SURF1-associated Leigh syndrome, respectively
    • Manufacturing partnerships established with UTSW and Catalent and entered into a lease agreement to occupy and configure an approximately 187,000-square-foot commercial-scale current Good Manufacturing Practices (cGMP) manufacturing facility in Durham, North Carolina to support preclinical, clinical and commercial production for Taysha's broad pipeline of gene therapies
    • Appointment of industry-leading gene therapy executives, Kathy Reape, M.D., former Chief Medical Officer of Spark Therapeutics, and Laura Sepp-Lorenzino, Ph.D., Chief Scientific Officer of Intellia Therapeutics, to the Board of Directors
    • Formation of independent Scientific Advisory Board consisting of preeminent international scientific and clinical thought leaders in gene therapy, CNS diseases and drug discovery and development
    • Expansion of leadership team to add significant gene therapy expertise and to deepen manufacturing, human resources, legal and corporate communications capabilities
    • The company has grown from a handful of employees at inception to approximately 80 as of the end of February
    • Gross proceeds of approximately $307 million raised since the company's formation, including $181 million in gross proceeds from IPO

    Full-Year 2020 Financial Highlights

    Research and Development (R&D) Expenses: R&D expenses were $31.9 million for the year ended December 31, 2020 compared to $1.0 million for the period from September 20, 2019 (the date of company inception) to December 31, 2019. The increase was primarily related to the company's development programs, as a result of increased manufacturing-related spend, clinical and preclinical activities, and headcount.

    General and Administrative (G&A) Expenses: G&A expenses were $11.1 million for the year ended December 31, 2020 compared to $0.1 million for the period from September 20, 2019 (the date of company inception) to December 31, 2019. The increase was primarily due to an increase in personnel costs resulting from increased headcount, professional services fees, and other corporate-related expenses.

    Other Expenses: Other expenses were $17.0 million for the year ended December 31, 2020 which were non-cash in nature and represented the change in fair value of the preferred stock tranche liability associated with the Series A convertible preferred stock.

    Net loss: Net loss for the year ended December 31, 2020 was $60.0 million, or $3.40 per share, as compared to a net loss of $1.1 million, or $0.12 per share, for the period from September 20, 2019 (the date of company inception) to December 31, 2019.

    Cash and cash equivalents: As of December 31, 2020, Taysha had $251.3 million in cash and cash equivalents, which included $165.9 million in net proceeds from the company's IPO completed in September 2020.

    Anticipated Milestones by Program

    TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes – HEXA and HEXB – intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

    • Report preliminary Phase 1/2 safety and biomarker data (Queen's University trial) in the second half of 2021
    • Submit an Investigational New Drug (IND) application in the U.S. in the second half of 2021
    • Initiate Phase 1/2 clinical trial in the U.S. in the second half of 2021
    • Report preliminary Phase 1/2 clinical data (Queen's University trial) by year-end 2021

    TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments

    • Maintain current open IND
    • Initiate a Phase 1/2 clinical trial in the second half of 2021 with commercial-grade GMP material

    TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for a severe neurodevelopmental disorder, designed to deliver MECP2 as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis

    • Submit IND/CTA filing in the second half of 2021
    • Initiate Phase 1/2 clinical trial by year-end 2021 with commercial-grade GMP material

    TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a codon optimized transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments

    • Submit IND/CTA filing in the second half of 2021
    • Initiate Phase 1/2 trial by year-end 2021 with commercial-grade GMP material

    Pipeline programs advancing into IND-/CTA-enabling studies

    • Advance four programs into IND-/CTA-enabling studies including TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN and TSHA-111-MALIN in Lafora disease, TSHA-112 in APBD and TSHA-119 in GM2 AB variant, and continue IND-/CTA-enabling work on TSHA-103 in SLC6A1 haploinsufficiency disorder in 2021
    • Submit an IND/CTA filing for one of the five above-named programs in 2021

    Discovery programs

    • Advance four new undisclosed programs focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases into preclinical development in 2021

    Next-generation technology platform

    • Continue development efforts on regulated transgene expression with expansion of miRARE platform into additional CNS diseases
    • Explore vagus nerve redosing platform in large animal models
    • Advance mini-gene discovery program in genetic forms of epilepsy and neurodevelopmental disorders
    • Continue discovery and development efforts on next-generation capsids

    Anticipated Corporate Milestones in 2021

    • Initiate construction on internal cGMP facility in 2021
    • Complete buildout of Dallas headquarters by mid-year
    • Expand employee base from nearly 40 (as of December 31, 2020) to approximately 150 by year-end 2021

    Conference Call and Webcast Information

    Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a corporate update. The dial-in number for the conference call is 833-614-1477 (U.S./Canada) or 914-987-7215 (international). The conference ID for all callers is 3183829. The live webcast and replay may be accessed by visiting Taysha's website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates and early-stage programs, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the year ended December 31, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    Taysha Gene Therapies, Inc.

    Consolidated Statement of Operations

    (in thousands, except share and per share data)

    For the Year Ended

    December 31, 2020

     

    For the Period from

    September 20, 2019

    (date of inception)

    to

    December 31, 2019

    Operating expenses:
    Research and development

    $

    31,893

     

    $

    987

     

    General and administrative

     

    11,109

     

     

    128

     

    Total operating expenses

     

    43,002

     

     

    1,115

     

    Loss from operations

     

    (43,002

    )

     

    (1,115

    )

    Other (expense) income:
    Change in fair value of preferred stock tranche liability

     

    (17,030

    )

     

     

    Interest income

     

    49

     

     

     

    Interest expense

     

    (28

    )

     

     

    Total other (expense) income

     

    (17,009

    )

     

     

    Net loss

    $

    (60,011

    )

    $

    (1,115

    )

    Net loss per common share, basic and diluted

    $

    (3.40

    )

    $

    (0.12

    )

    Weighted average common shares outstanding, basic and diluted

     

    17,665,683

     

     

    9,625,679

     

    Taysha Gene Therapies, Inc.

    Consolidated Balance Sheet Data

    (in thousands)

    December 31,

     

    December 31,

    2020

     

    2019

    Cash and cash equivalents

    $

    251,253

    $

     

    Total assets

    $

    258,881

    $

    15

     

    Total liabilities

    $

    7,579

    $

    150

     

    Total stockholders' equity (deficit)

    $

    251,302

    $

    (135

    )

     

    View Full Article Hide Full Article
  31. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) ("Taysha"), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for full-year ended December 31, 2020, and host a corporate update conference call and webcast on Wednesday, March 3, 2021, at 8:00 AM Eastern Time.

    Conference Call Details
    Wednesday, March 3, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

    833-614-1477

    International:

    914-987-7215

    Conference ID:

    3183829

    Webcast:

    https://ir.tayshagtx.com/news-events/events-presentations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA) ("Taysha"), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will report its financial results for full-year ended December 31, 2020, and host a corporate update conference call and webcast on Wednesday, March 3, 2021, at 8:00 AM Eastern Time.

    Conference Call Details
    Wednesday, March 3, at 8:00 AM Eastern Time / 7:00 AM Central Time

    Toll Free:

    833-614-1477

    International:

    914-987-7215

    Conference ID:

    3183829

    Webcast:

    https://ir.tayshagtx.com/news-events/events-presentations

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  32. Launches #RareAlly initiative to recognize those in the rare disease community who inspire and motivate us each day

    Furthers efforts by patient advocacy groups to educate and support the rare disease community

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations, today announced its support of Rare Disease Day and the launch of its #RareAlly initiative to recognize those in the rare disease community who inspire and motivate each other.

    #RareAlly is intended to recognize those who inspire us to be allies in this journey, including patients…

    Launches #RareAlly initiative to recognize those in the rare disease community who inspire and motivate us each day

    Furthers efforts by patient advocacy groups to educate and support the rare disease community

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations, today announced its support of Rare Disease Day and the launch of its #RareAlly initiative to recognize those in the rare disease community who inspire and motivate each other.

    #RareAlly is intended to recognize those who inspire us to be allies in this journey, including patients, caregivers, advocacy leaders, researchers, physicians, coworkers or the many other partners that fuel the Company's work. For Rare Disease Day and every day, Taysha celebrates those allies in the rare disease community who inspire us, who challenge us to give and be our best, and who we lean on for support. A collection of inspiring individuals and their allies is reflected at www.tayshagtx.com/rare-ally.

    "#RareAlly is Taysha's way of championing the individuals and groups who inspire us each and every day," said Emily McGinnis, Chief Patient Officer at Taysha. "We know it takes a collective, coordinated and bold effort to bring new medicines to patients with rare disease, many conditions for which there are no approved treatments. #RareAlly is our daily commitment to those we lean on to reach new heights and make our mission a reality."

    As part of Rare Disease Day activities, Taysha is raising funds through its #RareAlly photo submission campaign to support the rare disease community, and will host an all-employee event featuring a guest speaker from the UT Southwestern Gene Therapy Program and a screening of the "One Shot to Live" documentary series created by the Rare Village Foundation. Moments captured throughout the month of February can be found on Taysha's Twitter and LinkedIn pages.

    Approximately 300 million people around the world live with a rare disease and approximately 70% of rare genetic diseases start in childhood. In recognition of the significant amount of people living with rare conditions, the European Organization for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders in the U.S. (NORD) organized Rare Disease Day. It is held annually on the last day of February, a rare month with 28 days or 29 days during a leap year. More information about Rare Disease Day is available at www.rarediseaseday.org.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  33. Members include preeminent international scientific and clinical thought leaders in gene therapy, diseases of the central nervous system, and drug discovery and development

    Collective experience will provide invaluable research, clinical insights and input on development of broad portfolio focused on monogenic diseases of the central nervous system (CNS) in both rare and large patient populations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations, today announced the formation of an independent Scientific Advisory Board (SAB) that will work closely with…

    Members include preeminent international scientific and clinical thought leaders in gene therapy, diseases of the central nervous system, and drug discovery and development

    Collective experience will provide invaluable research, clinical insights and input on development of broad portfolio focused on monogenic diseases of the central nervous system (CNS) in both rare and large patient populations

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations, today announced the formation of an independent Scientific Advisory Board (SAB) that will work closely with senior management to advance the company's clinical development and commercialization efforts.

    "We are excited and privileged to have the opportunity to work with this cross-functional group of esteemed scientific and clinical thought leaders on initiatives from discovery, through pre-clinical and clinical development and commercialization," said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "They bring a wealth of knowledge in the development of gene therapy products and diseases of the CNS that will be invaluable as we advance our extensive pipeline of AAV-based gene therapies for the treatment of monogenic diseases of the CNS. Formalizing the SAB is an important accomplishment that will help position Taysha for sustained success as we further our R&D initiatives."

    The SAB brings together the expertise of esteemed independent scientists and clinicians covering Taysha's key areas of research in monogenic diseases and gene therapy products. Members of the SAB will provide scientific review and guidance to the company around its R&D and related business activities.

    Members of Taysha's SAB include:

    Deborah Bilder, M.D., is an Associate Professor at the University of Utah in Educational Psychology, General Pediatrics, and Child Psychiatry. Her research interests include clinical trials, medications, and biologics that target rare genetic conditions and has authored over 45 peer-reviewed articles. She is the Principal Investigator for the Utah Registry of Autism and Developmental Disabilities and Co-Principal Investigator for the Utah site of the Centers for Disease Control and Prevention's Autism and Developmental Disabilities Monitoring Network. Dr. Bilder is Co-Chair of the DAC Committee in psychiatry at the University of Utah and a consultant for the Utah Regional Education in Neurodevelopmental and Related Disabilities program. She has been awarded the Triple Board Program Teaching Award from the University of Utah Division of Child and Adolescent Psychiatry. She is a steering committee member for BioMarin Pharmaceutical Phase 3 Clinical Trial and also serves as a medical advisor for the Utah chapter of Make-a-Wish Foundation. Dr. Bilder earned her medical degree from Vanderbilt University.

    Alan Boyd, B.Sc., M.B., Ch.B., FRSB, FFLM, FRCP, FFPM, is the CEO and Founder of Boyd Consultants and a fellow and Immediate Past-President of the Faculty of Pharmaceutical Medicine, Royal Colleges of Physicians, UK. Professor Boyd is also a Council Member and the Independent Clinician Trustee on the Board of the Academy of Medical Royal Colleges, UK. He is also an honorary professor at the University of Birmingham Medical School, in recognition of his expertise in medicine development. He has significant pharmaceutical industry experience and was the Head of Medical Research at AstraZeneca and the Research and Development Director at Ark Therapeutics Ltd, specializing in the development of gene therapy products. He is a graduate in biochemistry and medicine from the University of Birmingham, UK.

    Wendy K. Chung, M.D., Ph.D., is a Kennedy Family Professor of Pediatrics in Medicine, Attending Physician in the Division of Molecular Genetics, Department of Pediatrics and Medicine, and the Director of Clinical Genetics, Clinical Cancer Genetics, and Precision Medicine Resource at the Irving Institute for Translational Research, all at Columbia University. Her research interests include spinal muscular atrophy, autism, and neurogenetics. Dr Chung has authored over 500 peer-reviewed articles and 75 textbook chapters and serves on the Editorial Board of Molecular Case Studies and The American Journal of Human Genetics. Dr Chung is the Director of Clinical Research at the Simons Foundation Autism Research Initiative (SFARI) and a member of the National Academy of Medicine. Dr. Chung earned her medical degree from Cornell University Medical College and her doctorate from Rockefeller University.

    David P. Dimmock, M.D., is the Senior Medical Director of Rady Children's Institute for Genomic Medicine. Dr. Dimmock is an expert in the field of clinical genomic medicine, the Principal Investigator on multiple clinical trials of novel therapeutics in rare metabolic diseases and an author of over 100 peer-reviewed articles, publications, chapters, books and reviews. He has been an invited advisor to the U.S. Food and Drug Administration in the Office of Orphan Diseases and has overseen regulatory submissions for whole genome sequencing devices. At the Center for Disease Control, he was a member of the Planning and Organizing Committee of NeXT-StoC to develop guidance to ensure analytic quality of next-generation sequencing tests. In addition, he was a member of the National Genomics Board UK and CLIAC NGS Guidelines Forum. He is a Scientific Advisory Board member for BioMarin Pharmaceuticals. Dr. Dimmock is a graduate from St. George's, University of London.

    Michael W. Lawlor, M.D., Ph.D., is a Professor of Pathology, Biomedical Engineering, Physiology, Cell Biology, Neurobiology, and Anatomy and the Associate Director of the Neuroscience Research Center at the Medical College of Wisconsin. He is a Board-Certified Anatomic Pathologist and Neuropathologist, and his research interests include pediatric muscle disease and gene therapy. Dr. Lawlor is an Editorial Board member of Muscle and Nerve and Journal of Neuropathology and Experimental Neurology. He is currently serving as an SAB member for Solid Biosciences in support of its gene therapy programs. Dr. Lawlor earned his medical degree and doctorate from Loyola University School of Medicine and his residency, fellowship, and postdoctoral training was completed at Massachusetts General Hospital and Boston Children's Hospital in association with Harvard Medical School.

    Gerald S. Lipshutz, M.D., M.S., is a Professor-in-Residence in the Departments of Surgery and Molecular and Medical Pharmacology, Surgical Director of the Pancreas/Auto-islet Transplant Program and Chairman of the Academic Medicine College at the David Geffen School of Medicine at University of California, Los Angeles. His clinical specialties and interests include liver and pancreas transplantation and gene and cell therapies for single-gene metabolic disorders of the liver. Dr. Lipshutz is a grant reviewer for the Wellcome Trust and the US National Institutes of Health where he is a standing member of the Gene and Drug Delivery (GDD) study section. He is a Principal Investigator at the UCLA Lipschutz Hepatic Regenerative Medical Laboratory and for several NIH-funded and industry-sponsored studies for gene therapies. He is author of over 70 peer-reviewed articles and is an Editorial Board member of Molecular Therapy - Methods and Clinical Development and Gene Therapy. Dr. Lipshutz earned his medical degree from the University of California, Los Angeles.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  34. Truist Virtual Fireside Chat on February 11, 2021 at 1:00 pm ET

    10th Annual SVB Leerink Global Healthcare Conference on Friday, February 26, 2021 at 8:40 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat for the Truist Fireside Chat series as well as the 10th Annual SVB Leerink Global Healthcare Conference.

    Conferences Details:

    Event:

    Truist Virtual Fireside Chat with Joon Lee, MD, PhD, Director and Senior Biotech Equity Research Analyst…

    Truist Virtual Fireside Chat on February 11, 2021 at 1:00 pm ET

    10th Annual SVB Leerink Global Healthcare Conference on Friday, February 26, 2021 at 8:40 am ET

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in a virtual fireside chat for the Truist Fireside Chat series as well as the 10th Annual SVB Leerink Global Healthcare Conference.

    Conferences Details:

    Event:

    Truist Virtual Fireside Chat with Joon Lee, MD, PhD, Director and Senior Biotech Equity Research Analyst

    Date:

    Thursday, February 11, 2021

    Time:

    1:00 pm ET

    Format:

    Fireside chat

    Participants:

    RA Session II, President, Founder and CEO

     

    Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

     

    Kamran Alam, Chief Financial Officer

     

    Dr. Kimberly Lee, SVP, Corporate Communications and Investor Relations

     

     

    Event:

    10th Annual SVB Leerink Global Healthcare Conference (Virtual)

    Date:

    Friday, February 26, 2021

    Time:

    8:40 am ET

    Format:

    Presentation

    Participants:

    RA Session II, President, Founder and CEO

    Audio webcasts for these conferences will be available in the "Events & Media" section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  35. Collaboration with Cleveland Clinic to support creation of novel next-generation mini-gene platform to pursue monogenic diseases not addressable by conventional AAV gene therapy technologies

    Collaboration with UT Southwestern Gene Therapy Program to develop new constructs incorporating novel mini-gene payloads, aligned with previously established partnership with Taysha

    Taysha to have an exclusive option on new payloads, constructs and intellectual property arising from research conducted under the agreements

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system…

    Collaboration with Cleveland Clinic to support creation of novel next-generation mini-gene platform to pursue monogenic diseases not addressable by conventional AAV gene therapy technologies

    Collaboration with UT Southwestern Gene Therapy Program to develop new constructs incorporating novel mini-gene payloads, aligned with previously established partnership with Taysha

    Taysha to have an exclusive option on new payloads, constructs and intellectual property arising from research conducted under the agreements

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced multi-year collaborations with Cleveland Clinic and UT Southwestern Gene Therapy Program (UTSW) to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of genetic epilepsies and additional CNS disorders. Taysha will have an exclusive option on new payloads, constructs and intellectual property associated with, and arising from, the research conducted under this agreement.

    A team of researchers from Cleveland Clinic Lerner Research Institute will create mini-gene payloads designed to address some of the long-standing limitations in AAV gene therapy. UTSW will create and evaluate vector constructs in in vivo and in vitro efficacy models of genetic epilepsies and additional CNS disorders.

    "By pushing the boundaries of AAV vector engineering, we may be able to overcome some of the challenges inherent with gene therapy and potentially expand the range of treatable genetic CNS diseases with gene therapies. We appreciate the support from Taysha and UTSW in this work," said Dennis Lal, Ph.D., Assistant Staff at Cleveland Clinic Genomic Medicine Institute and Neurological Institute. "We believe that our proprietary approach to overcoming current limitations of packaging capacity and our access to data on thousands of protein structures associated with a whole host of monogenic CNS disorders has the potential to enable a deep pipeline of functioning mini-genes."

    "Cleveland Clinic and UTSW are two of the world's preeminent leaders in gene therapy innovation, and this collaboration is designed to leverage our capabilities and synergies with these institutions to pioneer novel approaches to address vector capacity, which is a common limitation when treating genetic disorders associated with large proteins," said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "We look forward to a productive collaboration with the goal of developing treatments with promising benefits to patients with debilitating genetic epilepsies."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our collaboration with the Cleveland Clinic and UTSW, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  36. Expects Phase 1/2 biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis in second half of 2021 and by year-end 2021, respectively

    Plans to initiate a U.S. Phase 1/2 trial for TSHA-101 in GM2 gangliosidosis in second half of 2021 as well as Phase 1/2 trials for TSHA-118 in CLN1, TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end 2021

    Anticipates advancement of four programs into IND/CTA-enabling studies: SLC13A5 haploinsufficiency, Adult Polyglycosan Body Deficiency (APBD), Lafora disease and GM2 AB variant

    Expects IND/CTA submission from one of the following programs: SLC13A5 haploinsufficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

    Anticipates advancement

    Expects Phase 1/2 biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis in second half of 2021 and by year-end 2021, respectively

    Plans to initiate a U.S. Phase 1/2 trial for TSHA-101 in GM2 gangliosidosis in second half of 2021 as well as Phase 1/2 trials for TSHA-118 in CLN1, TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end 2021

    Anticipates advancement of four programs into IND/CTA-enabling studies: SLC13A5 haploinsufficiency, Adult Polyglycosan Body Deficiency (APBD), Lafora disease and GM2 AB variant

    Expects IND/CTA submission from one of the following programs: SLC13A5 haploinsufficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

    Anticipates advancement of four new undisclosed programs into preclinical development focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases

    Intends to advance the development of next-generation technologies including miRARE platform, redosing strategy, mini-gene payloads and novel capsids, to optimize key components of the company's AAV-based gene therapies

    Continues to make progress on internal 187,000 square-foot, 2,000-liter capacity, multi-product cGMP facility located in Durham, NC

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA) ("Taysha"), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today highlighted its strategic priorities and provided a business outlook for 2021.

    "We enter 2021 having built a strong foundation on which to execute our corporate and pipeline objectives. Notably, we expanded our seasoned leadership team and esteemed board of directors steeped in gene therapy development and commercialization expertise, successfully raised funds in our initial public offering, transitioned from a preclinical- to a clinical-stage company, and achieved important progress on R&D initiatives and our three-pillar manufacturing strategy," said RA Session II, President, Founder and CEO of Taysha. "2021 will be a transformational year as we intend to rapidly advance multiple drug candidates to clinical proof-of-concept, further expand our platform-enabled pipeline and advance next-generation technologies. Specifically, we expect to report clinical data for our GM2 gangliosidosis program in the second half of this year and have multiple ongoing clinical studies by year end. We also anticipate several IND/CTA submissions across three CNS franchises and have multiple therapies in IND/CTA-enabling studies while advancing four new programs into preclinical development. In addition, we are excited to advance our next-generation platform technologies and further our efforts in redosing, transgene regulation and capsid development. We believe that our platform will drive future sustained innovation and value creation and look forward to highlighting the productivity of our platform in an R&D day later this year. Lastly, we continue to make progress on cGMP facility and process development capabilities with the completion of the design phase and initiation of procurement of long lead equipment."

    Anticipated Milestones by Program

    TSHA-101 for infantile GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes – HEXA and HEXB – intrathecally for the treatment of infantile GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

    • Report Phase 1/2 safety and biomarker data (Queen's University trial) in the second half of 2021
    • Submit an Investigational New Drug (IND) application in the U.S. in the second half of 2021
    • Initiate Phase 1/2 clinical trial in the U.S. in the second half of 2021
    • Report preliminary Phase 1/2 clinical data (Queen's University trial) by year-end 2021

    TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments

    • Maintain current open IND
    • Initiate a Phase 1/2 clinical trial in the second half of 2021 with commercial-grade GMP material

    TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for one of the most common genetic causes of severe intellectual disability, designed to deliver MECP2 as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis

    • Submit IND/Clinical Trial Application (CTA) filing in the second half of 2021
    • Initiate Phase 1/2 clinical trial by year-end 2021 with commercial-grade GMP material

    TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a codon optimized transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments

    • Submit IND/CTA filing in the second half of 2021
    • Initiate Phase 1/2 trial by year-end 2021 with commercial-grade GMP material

    Pipeline programs advancing into IND/CTA-enabling studies

    • Advance four programs into IND/CTA-enabling studies including TSHA-105 in SLC13A5 haploinsufficiency, TSHA-111 in Lafora, TSHA-112 in APBD and TSHA-119 in GM2 AB variant, and continue IND/CTA-enabling work on TSHA-103 in SLC6A1 haploinsufficiency in 2021
    • Submit an IND/CTA filing for one of the five above-named programs in 2021

    Discovery programs

    • Advance four new undisclosed programs focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases into preclinical development in 2021

    Next-generation technology platform

    • Continue development efforts on regulated transgene expression with expansion of miRARE platform into additional CNS diseases
    • Explore vagus nerve redosing platform in large animal models
    • Advance mini-gene discovery program in genetic forms of epilepsy and neurodevelopmental disorders
    • Continue discovery and development efforts on next-generation capsids

    Anticipated Corporate Milestones in 2021

    • Complete technical development of, and initiate construction on, internal cGMP facility in 2021
    • Complete buildout of Dallas headquarters by mid-year
    • Expand employee base from nearly 40 to approximately 150 by year-end 2021

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates and early-stage programs, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  37. Timothy J. Douros, J.D., former General Counsel at Bluebird Bio, joins as Chief Legal Officer and Corporate Secretary, brings over 25 years of legal experience in the biotech industry

    Tracy M. Porter, M.Ed., SPHR, former Head of Human Resources at Audentes Therapeutics, joins as Chief People Officer, brings nearly 30 years of human resources experience

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced new additions to its leadership team with the appointments of Timothy J. Douros, J.D., as Chief Legal Officer and…

    Timothy J. Douros, J.D., former General Counsel at Bluebird Bio, joins as Chief Legal Officer and Corporate Secretary, brings over 25 years of legal experience in the biotech industry

    Tracy M. Porter, M.Ed., SPHR, former Head of Human Resources at Audentes Therapeutics, joins as Chief People Officer, brings nearly 30 years of human resources experience

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced new additions to its leadership team with the appointments of Timothy J. Douros, J.D., as Chief Legal Officer and Corporate Secretary and Tracy M. Porter, M.Ed., SPHR, as Chief People Officer. Mr. Douros will lead all aspects of the company's legal organization. Ms. Porter will oversee all aspects of human resources, including operations, talent acquisition and employee development.

    "We are thrilled to welcome Tim and Tracy to the Taysha leadership team," said RA Session II, President, Founder and CEO of Taysha. "Tim brings significant gene therapy experience from Bluebird Bio where he led a global team, in cross functional collaboration with all corporate functions. His experiences include a wide range of legal and corporate matters which will be invaluable as we continue to advance our programs and evolve as a company. With her vast experience in strategic and operation roles in human resources, Tracy has successfully led and scaled global organizations quickly, most recently at Audentes where she helped grow the base of global gene therapy professionals by more than 50% in less than a year. Tracy will be instrumental in helping us continue to build a successful organization, scale thoughtfully and maintain our culture. Importantly, both Tim and Tracy's experiences working in companies at various stages of the biotech life cycle will be essential to our future growth strategy."

    Mr. Douros brings over 25 years of legal experience in biotech specializing in intellectual property, strategic licensing and contracting, litigation and dispute resolution, as well as international and healthcare compliance while building and managing high-performing teams. Prior to joining Taysha, he served as Senior Vice President, General Counsel at Bluebird Bio, Inc., where he led a global team of attorneys and staff and was a strategic partner to all internal clients, including business development, research and development, clinical operations and regulatory affairs. Prior to that, he was Vice President, Deputy General Counsel and Chief IP Counsel at Bluebird Bio where he provided counsel and managed all legal services related to the company's European infrastructure build to support product launches as well as provided counsel on all areas of compliance. Mr. Douros also led the corporate intellectual property function and managed all corporate dispute resolution matters. Before joining Bluebird, he spent nearly 13 years at Cubist Pharmaceuticals, Inc., holding positions of increasing responsibility, most recently serving as Vice President, Chief International Counsel & Chief IP Counsel. Mr. Douros received an A.B. in Chemistry from Dartmouth College and a J.D. from Boston College Law School.

    "I could not be more excited to join such an accomplished and energetic team of gene therapy trailblazers," said Mr. Douros. "With its potentially de-risked approach to gene therapy development, Taysha is well-positioned to be a leader in developing potentially disease-modifying gene therapies for patients with monogenic CNS diseases and I look forward to contributing to the company's growth."

    Ms. Porter joins Taysha with nearly 30 years as a human resources executive focused in entrepreneurial global business environments and specializing in pharmaceuticals and biotech. She has guided organizations from start-up and early-stage biotech to initial product launches and has provided human resources support through manufacturing expansion, acquisitions, divestitures, strategic business reorganizations and integrations. She most recently served as Vice President, Head of Human Resources at Audentes Therapeutics, where she developed, implemented and sustained its human capital strategy to rapidly scale and support the company's growth. Prior to joining Audentes, she spent time at Medivation Inc., and Bayer Healthcare LLC. Ms. Porter received a B.S. in Human Development and Counseling from the University of North Carolina at Greensboro and an M.Ed. in Global Human Resource Development from the University of Illinois at Urbana-Champaign.

    "Taysha's community of talented, diverse and passionate people is exceptional and I am honored to be a part of the team," said Ms. Porter. "Taysha has a remarkable vision to develop and commercialize innovative therapies to potentially transform patients' lives. I look forward to helping build the team and capabilities to realize that vision."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  38. Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy

    TSHA-105 is the second program from Taysha's genetic epilepsy franchise to receive dual designations

    TSHA-105 joins a portfolio of rare pediatric disease and orphan drug designations obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1, TSHA-102 for Rett syndrome, TSHA-104 for SURF1-associated Leigh syndrome and TSHA-103 for SLC6A1-related epilepsy

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient…

    Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy

    TSHA-105 is the second program from Taysha's genetic epilepsy franchise to receive dual designations

    TSHA-105 joins a portfolio of rare pediatric disease and orphan drug designations obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1, TSHA-102 for Rett syndrome, TSHA-104 for SURF1-associated Leigh syndrome and TSHA-103 for SLC6A1-related epilepsy

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

    "There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease," said RA Session II, President, Founder and CEO of Taysha. "We are encouraged by the early evidence of TSHA-105's disease-modifying approach and believe these designations will help us potentially accelerate the development of this exciting program. We look forward to working with the FDA to make TSHA-105 available to patients as expeditiously as possible."

    SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. This rare form of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life.

    "We are pleased that the FDA recognizes TSHA-105's potential as an innovative therapeutic option for SLC13A5 deficiency," said Rachel Bailey, Ph.D., Assistant Professor in Pediatric Neurology at UT Southwestern. "This disease is a debilitating form of genetic epilepsy in children that significantly impacts movement, motor control, cognition and quality of life, and there remains a need to alter the course of this disease early in life."

    "As a mother of two children with SLC13A5 deficiency, I have witnessed firsthand the devastating impact that numerous seizures and comorbidities accompanying the disease has on those affected by this disease," said Kim Nye, Founder of TESS Research Foundation. "Taysha's commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community."

    The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

    Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years post approval.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-105, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  39. Expanded collaboration to support discovery of novel gene therapy candidates and next-generation technologies in new disease areas

    Taysha to have an exclusive option on discovery programs and intellectual property arising from research conducted under the agreement

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, and UT Southwestern Medical Center (UTSW) today announced the launch of an innovation fund to discover and develop novel gene therapy candidates and next-generation technologies for monogenic diseases of the CNS…

    Expanded collaboration to support discovery of novel gene therapy candidates and next-generation technologies in new disease areas

    Taysha to have an exclusive option on discovery programs and intellectual property arising from research conducted under the agreement

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, and UT Southwestern Medical Center (UTSW) today announced the launch of an innovation fund to discover and develop novel gene therapy candidates and next-generation technologies for monogenic diseases of the CNS. This expanded partnership will support UTSW's discovery efforts to facilitate the translation of promising discoveries from bench to clinic. Taysha will have an exclusive option on new programs and intellectual property associated with, and arising from, the research conducted under this agreement.

    A team of researchers from the gene therapy program at UT Southwestern will explore novel gene therapy targets in new disease areas and create next-generation gene therapy technology platforms to address some of the current limitations of this modality.

    "We are excited to expand our alliance with UTSW to accelerate the discovery and development of novel gene therapy candidates and next-generation technologies for patients with monogenic CNS diseases," said RA Session II, President, Founder and CEO of Taysha. "We believe that the combination of UTSW's translational research expertise in gene therapy and strong track record of innovation and our experience in drug development and GMP manufacturing will create opportunities to reach more patients with unmet medical needs. Our relationship with the UTSW gene therapy program has produced over 18 novel product candidates, including TSHA-101 in GM2 gangliosidosis and TSHA-118 in CLN1, which are currently in clinical development. We are pleased by the significant progress our partnership has achieved and are excited to build on that foundation and momentum to bring additional compelling innovation to the clinic."

    About The University of Texas Southwestern Medical Center

    UT Southwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institution's faculty has received six Nobel Prizes and includes 23 members of the National Academy of Sciences, 17 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,500 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 105,000 hospitalized patients, nearly 370,000 emergency room cases, and oversee approximately 3 million outpatient visits a year.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  40. Taysha to leverage AllStripes' technology platform to support development of TSHA-104 for SURF1-associated Leigh syndrome

    Taysha Gene Therapies, Inc., (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced a multi-year collaboration with AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating research for patients with rare diseases, during which Taysha will leverage AllStripes' platform to inform its understanding of SURF1-associated Leigh syndrome natural history and burden of disease, as well as patients' diagnostic…

    Taysha to leverage AllStripes' technology platform to support development of TSHA-104 for SURF1-associated Leigh syndrome

    Taysha Gene Therapies, Inc., (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced a multi-year collaboration with AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating research for patients with rare diseases, during which Taysha will leverage AllStripes' platform to inform its understanding of SURF1-associated Leigh syndrome natural history and burden of disease, as well as patients' diagnostic journeys.

    The collaboration will focus on advancing the development of TSHA-104, an AAV9-based gene therapy product candidate in development for the treatment of SURF1-associated Leigh syndrome. Taysha will utilize AllStripes' clinical database to uncover new insights into disease progression and better inform selection of endpoints for clinical studies.

    "This collaboration will allow us to leverage the AllStripes technology platform to optimize our therapeutic strategy and to potentially accelerate the development of TSHA-104 in SURF1-associated Leigh syndrome," said RA Session, II, President, Founder and Chief Executive Officer of Taysha. "We remain committed to developing a safe and effective gene therapy for patients suffering with this devastating disease, and data generated from this unique collaboration could bring us one step closer to our goal."

    "Taysha has brought together accomplished and knowledgeable gene therapy and CNS disease experts to develop potentially transformative therapies," said Nancy Yu, Co-founder and Chief Executive Officer of AllStripes. "With no available treatment for SURF1-associated Leigh syndrome, we are very pleased to empower patients and their families with an avenue to participate in research that will support the development path of TSHA-104. We are hopeful that this novel gene therapy will bring meaningful benefit to children and their families, and give them more time together."

    TSHA-104 has been granted rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of SURF1-associated Leigh syndrome. An Investigational New Drug (IND) application for TSHA-104 in SURF1-associated Leigh syndrome is expected to be submitted to the FDA in 2021.

    About SURF1-Associated Leigh Syndrome

    SURF1 deficiency is a monogenic mitochondrial disorder and is the most common cause of cytochrome c oxidase deficient Leigh syndrome. Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation. There are currently no approved therapies to treat SURF1-associated Leigh syndrome.

    About TSHA-104

    TSHA-104 is an investigational AAV9-based gene therapy administered intrathecally for the treatment of SURF1-associated Leigh syndrome. The product candidate is a recombinant AAV9 vector with engineered transgene encoding the human SURF1 protein. TSHA-104 has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    About AllStripes

    AllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, Spark Capital, Maveron Capital, Village Global, Garuda Ventures and a number of angel investors. For more information, visit www.allstripes.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our collaboration with AllStripes, the potential of our product candidates, including TSHA-104, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  41. Taysha to Leverage AllStripes' Technology Platform to Support Development of TSHA-104, a Gene Therapy for SURF1-Associated Leigh Syndrome

    AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating research for patients with rare diseases, today announced a multiyear collaboration with Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations.

    The collaboration will focus on advancing the development of TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome, a deadly rare disease…

    Taysha to Leverage AllStripes' Technology Platform to Support Development of TSHA-104, a Gene Therapy for SURF1-Associated Leigh Syndrome

    AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating research for patients with rare diseases, today announced a multiyear collaboration with Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations.

    The collaboration will focus on advancing the development of TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome, a deadly rare disease that primarily affects infants. AllStripes will use its platform, which gives patients control over their health histories, to unify otherwise scattered and fragmented SURF1-associated clinical data, allowing researchers to uncover new insights into the natural history and burden of disease and better inform the development of clinical studies.

    "This collaboration will allow us to leverage the AllStripes technology platform to optimize our therapeutic strategy and to potentially accelerate the development of TSHA-104 in SURF1-associated Leigh syndrome," said RA Session, II, president, founder and chief executive officer of Taysha. "We remain committed to developing a safe and effective gene therapy for patients suffering with this devastating disease, and data generated from this unique collaboration could bring us one step closer to our goal."

    Mutations in the SURF1 gene prevent mitochondria from producing enough energy for cells in the body to function normally, leading to Leigh syndrome, a severe and rare neurological disorder characterized by progressive loss of mental and movement abilities. SURF1-associated Leigh syndrome typically presents during infancy or early childhood, and often results in death within a few years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation. There is currently no targeted treatment or cure for SURF1-associated Leigh syndrome.

    "Taysha has brought together accomplished and knowledgeable gene therapy and CNS disease experts to develop potentially transformative therapies," said Nancy Yu, co-founder and chief executive officer of AllStripes. "With no available treatment for SURF1-associated Leigh syndrome, we are very pleased to empower patients and their families with an avenue to participate in research that will support the development path of TSHA-104. We are hopeful that this novel gene therapy will bring meaningful benefit to children and their families, and give them more time together."

    TSHA-104 has been granted rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of SURF1-associated Leigh syndrome. An Investigational New Drug (IND) application for TSHA-104 in SURF1-associated Leigh syndrome is expected to be submitted to the FDA in 2021.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    About AllStripes

    AllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, Spark Capital, Maveron Capital, Village Global, Garuda Ventures and a number of angel investors. For more information, visit www.allstripes.com.

    View Full Article Hide Full Article
  42. Greg Gara joins as SVP of Manufacturing, previously led the construction and commission of the AveXis Libertyville, Research Triangle Park and Longmont manufacturing facilities

    Kimberly Lee, D.O., joins as SVP of Corporate Communications and Investor Relations, brings over 20 years of capital markets and strategic corporate finance experience

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced new additions to its leadership team with the appointments of Greg Gara as Senior Vice President of Manufacturing and Kimberly…

    Greg Gara joins as SVP of Manufacturing, previously led the construction and commission of the AveXis Libertyville, Research Triangle Park and Longmont manufacturing facilities

    Kimberly Lee, D.O., joins as SVP of Corporate Communications and Investor Relations, brings over 20 years of capital markets and strategic corporate finance experience

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced new additions to its leadership team with the appointments of Greg Gara as Senior Vice President of Manufacturing and Kimberly Lee, D.O., as Senior Vice President of Corporate Communications and Investor Relations.

    "We are excited to welcome Greg and Kim to Taysha's leadership team," said RA Session II, President, Founder and CEO of Taysha. "They each bring significant domain experience and their contributions will be invaluable as we continue our mission of eradicating monogenic CNS diseases. Greg's technical expertise in AAV gene therapy manufacturing along with his proven success in constructing several cGMP gene therapy facilities and Kim's deep experience across capital markets and corporate communications will add tremendous value to the team. Importantly, both share our unrelenting, patient-first focus and passion for bringing new cures to life."

    Mr. Gara has over 25 years of experience in designing, constructing, and starting up large- and small-scale manufacturing facilities for biotechnology companies globally. Prior to joining Taysha, he served as Vice President of Pharmaceutical Engineering at Sarepta, where he led and managed manufacturing operations for all gene therapy products. Before Sarepta, he served as Vice President of Technical Operations and Engineering at AveXis, a Novartis company, where he led the design, construction, and startup of the Libertyville facility and the new facilities in Research Triangle Park and Colorado. Mr. Gara also led the team for the facility expansion in North Carolina and the renovation of the Colorado site. Prior to AveXis, he led the facilities and engineering organization at Hospira prior to the company's acquisition by Pfizer. Before joining Hospira, he spent 15 years at Amgen, holding positions of increasing responsibility, and was part of the Cork, Ireland, construction project. Mr. Gara received a B.A. in Biology and Environmental Science from Augustana College.

    "Taysha's dedication to the development and commercialization of potentially transformative gene therapy treatments and its innovative and pioneering spirit is truly inspiring and I am excited to contribute in a meaningful way," said Mr. Gara. "I look forward to playing an instrumental role in the company's growth and expansion of its manufacturing capabilities."

    Dr. Lee joins Taysha with over 20 years of capital markets, strategic corporate finance, and communications experience from prior roles as a biotech equity research analyst on Wall Street and corporate strategy, communications, and investor relations professional. She most recently served as Head of Corporate Strategy and Investor Relations at Lexicon Pharmaceuticals and previously as Vice President of Corporate Strategy, Corporate Communications and Investor Relations at Raptor Pharmaceuticals until its acquisition by Horizon Pharma. Prior to joining Raptor, Dr. Lee was a biotechnology sell-side analyst at investment banks, including Jefferies and Wedbush Securities, covering biotechnology companies across all market capitalizations, multiple therapeutic areas, and modalities. Dr. Lee received a B.S. in Biological Sciences from Stanford University and a D.O. from Kirksville College of Osteopathic Medicine.

    "In less than one year, Taysha has made extraordinary progress in developing and funding its elegant platform and rapidly advancing its product candidates for the betterment of patients and I am thrilled and grateful to be a part of this journey," said Dr. Lee. "I am eager to learn from and work alongside this team of gene therapy experts at this exciting stage of our company's lifecycle and I look forward to making lasting contributions."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    View Full Article Hide Full Article
  43. TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both HEXA and HEXB transgenes within a single AAV9 vector construct

    TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118's open investigational new drug application for CLN1

    Interim data from Phase 1/2 trial anticipated in 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that Queen's University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health…

    TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both HEXA and HEXB transgenes within a single AAV9 vector construct

    TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118's open investigational new drug application for CLN1

    Interim data from Phase 1/2 trial anticipated in 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that Queen's University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its investigator-sponsored Phase 1/2 trial exploring TSHA-101, Taysha's investigational AAV9-based gene therapy, for the treatment of infantile GM2 gangliosidosis.

    "TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy," said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. "GM2 is a devastating lysosomal storage disease with no approved treatments and today's CTA approval marks a formative moment for children suffering from this rapidly progressive and fatal disease."

    The trial will be a single arm, open-label Phase 1/2 trial evaluating the use of TSHA-101 for the treatment of infants with GM2. The study will be sponsored by Queen's University and led by Jagdeep S. Walia, MBBS, FRCPC, FCCMG, Clinical Geneticist and Associate Professor Head, Division of Medical Genetics (Department of Pediatrics) at Queen's, and Director of Research (Department of Pediatrics), at the Kingston Health Sciences Centre.

    "Preclinical evidence to date supports our belief that TSHA-101, when given intrathecally as a bicistronic transgene packaged into a single AAV9 vector, has the potential to address the lysosomal enzyme deficiency, to change the disease trajectory and to improve patient survival," said Dr. Jagdeep S. Walia. "We are pleased to have the support of Health Canada as we continue to advance TSHA-101."

    "Today's CTA approval is a culmination of our team's and Dr. Walia's tireless efforts and a momentous occasion for children affected by GM2 along with their parents and caregivers," said RA Session II, Founder, President and CEO of Taysha. "We are grateful to our partners at Queen's University for their work to advance this gene therapy into the clinic."

    About GM2 Gangliosidosis

    GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the β-hexosaminidase A enzyme. These genetic defects result in progressive dysfunction of the central nervous system. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

    About TSHA-101

    TSHA-101 is an investigational gene therapy administered intrathecally for the treatment of infantile GM2 gangliosidosis. The gene therapy is designed to deliver two genes – HEXA and HEXB – driven by a single promoter within the same AAV9 construct, also known as a bicistronic vector. This approach allows the simultaneous expression of a 1:1 ratio of the two subunits of protein required to generate a functional enzyme. It is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA).

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-101, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  44. Approximately 187,000-square-foot facility located in Durham, NC designed to support preclinical through commercial cGMP manufacturing for Taysha's broad pipeline of gene therapies

    Multiple production suites with total capacity of 2,000 liters expected to be production-ready by 2023

    Taysha to invest $75 million and create approximately 200 jobs over two and a half years; Company to receive up to $9.4 million in state and local incentives

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has entered into…

    Approximately 187,000-square-foot facility located in Durham, NC designed to support preclinical through commercial cGMP manufacturing for Taysha's broad pipeline of gene therapies

    Multiple production suites with total capacity of 2,000 liters expected to be production-ready by 2023

    Taysha to invest $75 million and create approximately 200 jobs over two and a half years; Company to receive up to $9.4 million in state and local incentives

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has entered into a lease agreement to occupy and configure an approximately 187,000-square-foot commercial-scale current Good Manufacturing Practices (cGMP) manufacturing facility in Durham, North Carolina for preclinical, clinical and commercial production of its gene therapy pipeline. The Company will invest $75 million and create approximately 200 jobs over a two-and-a-half-year period to build out development, analytical, manufacturing and quality control testing capability for its broad portfolio of gene therapies.

    Multiple production suites, which are expected to be fully commissioned by 2023, will allow production according to the U.S. Food and Drug Administration guidelines. The facility will establish 2,000 liters of capacity and will be designed to support all aspects of scalable manufacturing of gene therapy material for Taysha's pipeline and to meet the foreseeable clinical and commercial demand. This internal capability will bolster the current capacity from Taysha's existing manufacturing collaborations with UT Southwestern's Gene Therapy Program and Catalent. The investment in the facility is part of the Company's comprehensive three-pillar manufacturing strategy to meet the supply demands of multiple concurrent clinical programs emerging from its gene therapy pipeline as the Company anticipates having four open Investigational New Drug applications in 2021.

    "This state-of-the-art facility is an integral part of our manufacturing strategy that will enable us to rapidly and efficiently deliver potentially transformative treatments to patients with monogenic CNS diseases," said RA Session II, President, Founder and CEO of Taysha. "With our outstanding team of experts leading the charge, we expect this facility will serve as a center of excellence for gene therapy development, from preclinical studies through commercialization, and will further our leadership position in gene therapy as well as support our next phase of growth."

    "Given the potential demand of our robust portfolio, establishing internal capacity using our HEK293 suspension process is a key addition to our manufacturing supply chain, allowing us to drive efficiencies and scalability while potentially reducing the time to bring our gene therapy solutions to patients," said Frederick Porter, Ph.D., Chief Technical Officer of Taysha. "We anticipate this facility will complement existing capabilities and secure our long-term supply chain, which aligns well with our strategic goals. We are excited to expand our footprint in North Carolina, home to a thriving gene therapy ecosystem with a talented and seasoned workforce with deep gene therapy manufacturing expertise."

    Taysha's expansion in North Carolina will be facilitated by state and local incentives totaling up to $9.4 million. Specifically, a Job Development Investment Grant (JDIG), approved by the state's Economic Investment Committee earlier today, will provide Taysha up to $4.8 million in funding over 12 years, dependent upon meeting hiring and capital expenditure milestones, as well as a training grant of over $360,000 over a two- to three-year period. The Company will also receive a local incentive investment of up to $4.6 million over four years.

    "The pandemic has highlighted the importance of science and innovation to keep us healthy," said Governor Roy Cooper. "Companies like Taysha Gene Therapies continue to expand in North Carolina because we have the scientists, skilled workers and climate for innovation they need to tackle health care's toughest challenges."

    "Taysha's decision to expand to Durham continues to support our city's reputation as a leading hub for technology, innovation and life sciences," said City of Durham's Mayor Steve Schewel. "Companies globally are seeing Durham as a thriving location to attract talent and grow their business."

    The following North Carolina organizations were instrumental in Taysha's expansion in North Carolina: North Carolina Department of Commerce, the Economic Development Partnership of N.C., the North Carolina General Assembly, the North Carolina Community College System, the North Carolina Biotechnology Center, Durham County, the Greater Durham Chamber of Commerce and Duke Energy.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  45. Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it is set to join the Russell 2000® Index, effective Monday, December 21, 2020, as part of the index's quarterly initial public offering (IPO) additions.

    The Russell 2000 Index measures the performance of the small-cap segment of the U.S. equity market. The index is a subset of the Russell 3000® Index and represents approximately 10 percent of the total market capitalization of that index. Russell indexes are widely used by investment managers and institutional…

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it is set to join the Russell 2000® Index, effective Monday, December 21, 2020, as part of the index's quarterly initial public offering (IPO) additions.

    The Russell 2000 Index measures the performance of the small-cap segment of the U.S. equity market. The index is a subset of the Russell 3000® Index and represents approximately 10 percent of the total market capitalization of that index. Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russell's U.S. indexes. Russell indexes are part of FTSE Russell, a leading global index provider.

    For more information on the Russell 2000 Index and the Russell indexes IPO additions, please visit the "Russell U.S. Index IPO Additions" section on the FTSE Russell website.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    About FTSE Russell

    FTSE Russell is a leading global index provider creating and managing a wide range of indexes, data and analytic solutions to meet client needs across asset classes, style and strategies. Covering 98% of the investable market, FTSE Russell indexes offer a true picture of global markets, combined with the specialist knowledge gained from developing local benchmarks around the world.

    FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. Approximately $16 trillion is currently benchmarked to FTSE Russell indexes. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create investment funds, ETFs, structured products and index-based derivatives. FTSE Russell indexes also provide clients with tools for asset allocation, investment strategy analysis and risk management.

    A core set of universal principles guides FTSE Russell index design and management: a transparent rules-based methodology is informed by independent committees of leading market participants. FTSE Russell is focused on index innovation and customer partnership applying the highest industry standards and embracing the IOSCO Principles. FTSE Russell is wholly owned by London Stock Exchange Group.

    For more information, visit www.ftserussell.com.

    View Full Article Hide Full Article
  46. Designations provide validation of encouraging preclinical data generated to date

    TSHA-103 joins portfolio of rare pediatric disease and orphan drug designated product candidates including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome, TSHA-104 for SURF1-associated Leigh syndrome and TSHA-118 for CLN1

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-103, an AAV-9-based…

    Designations provide validation of encouraging preclinical data generated to date

    TSHA-103 joins portfolio of rare pediatric disease and orphan drug designated product candidates including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome, TSHA-104 for SURF1-associated Leigh syndrome and TSHA-118 for CLN1

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-103, an AAV-9-based gene therapy in development for SLC6A1-related epilepsy.

    "We are pleased by the FDA's acknowledgement of the imperative need to develop therapies for such a severe and life-threatening condition," said RA Session II, President, Founder and CEO of Taysha. "We are encouraged by the early evidence of our gene therapy approach to potentially treat this devastating disease. These designations in now five programs underscore the critical nature of our work and add momentum for these programs. We remain committed to advancing our pipeline of innovative and potentially transformative product candidates as we aim to eradicate monogenic CNS disease."

    SLC6A1 epilepsy is an autosomal dominant genetic disorder characterized by the loss of function of one copy of the SLC6A1 gene, with clinical manifestations of seizures, epilepsy, language impairment and intellectual disability.

    "Haploinsufficiency in the SLC6A1 gene has been identified as a cause of genetic epilepsy, yet there remains a lack of approved disease-modifying therapies," said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. "The designations highlight the innovation of TSHA-103 and the importance of developing a treatment for patients living with this devastating disease."

    "As a mother of a child affected by SLC6A1, Taysha's dedication to developing a treatment for this community is greatly applauded," said Amber Freed, Founder of SLC6A1 Connect. "We are delighted that the FDA recognizes the unmet medical need and the role that TSHA-103 may play."

    The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to December 11, 2022 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

    Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs as well as eligibility for market exclusivity for seven years post approval.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-103, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  47. Appoints former Chief Medical Officer of Spark Therapeutics, Kathy Reape, M.D., and Chief Scientific Officer of Intellia Therapeutics, Laura Sepp-Lorenzino, Ph.D., to board of directors

    Directors bring significant gene therapy translational and development expertise ahead of GM2 gangliosidosis clinical trial initiation in 2020 and submission of four INDs by the end of 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced the appointment of Kathy Reape, M.D., and Laura Sepp-Lorenzino, Ph.D., to the company's board…

    Appoints former Chief Medical Officer of Spark Therapeutics, Kathy Reape, M.D., and Chief Scientific Officer of Intellia Therapeutics, Laura Sepp-Lorenzino, Ph.D., to board of directors

    Directors bring significant gene therapy translational and development expertise ahead of GM2 gangliosidosis clinical trial initiation in 2020 and submission of four INDs by the end of 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced the appointment of Kathy Reape, M.D., and Laura Sepp-Lorenzino, Ph.D., to the company's board of directors.

    "Drs. Reape and Sepp-Lorenzino bring significant gene therapy translational and development expertise to our board," said RA Session II, President, Founder and CEO of Taysha Gene Therapies. "Their combined gene therapy experience across preclinical and clinical development will be invaluable as we continue to advance our broad portfolio into the clinic. Across all levels of the organization, we are building a team that has the passion, experience and talent to execute on our mission of eradicating monogenic CNS disease."

    Dr. Reape was most recently Chief Medical Officer at Spark Therapeutics where she oversaw clinical development, pharmacovigilance and medical affairs activities and was a key member of the team responsible for the development and commercialization of the first FDA-approved in vivo gene therapy, LUXTURNA®, for an inherited retinal disease caused by mutations in both copies of the RPE65 gene. She also oversaw the development of Spark's pipeline of gene therapies addressing CNS disease, hemophilia, metabolic disorders and inherited retinal dystrophies. She has over 18 years of experience in the pharmaceutical industry in clinical research and development and has been involved with approximately two dozen product approvals including small molecules, biologics, biosimilars and therapeutic devices. She received both her undergraduate and M.D. degrees from the University of Pennsylvania and completed her internship and residency at the University of Florida and University of Medicine and Dentistry of New Jersey.

    "Taysha has built a deep pipeline of potentially transformative gene therapies for patients with life-threatening CNS diseases," said Dr. Reape. "Many of the conditions that Taysha is addressing have no therapeutic alternatives, are associated with a poor quality of life and often result in a shortened life expectancy. It is important that we rapidly advance these gene therapies into the clinic to serve patients so desperately in need."

    Dr. Sepp-Lorenzino is currently the Chief Scientific Officer at Intellia Therapeutics and has held several senior positions over her extensive career. Most recently, she was Vice President and Head of Nucleic Acid Therapies at Vertex Pharmaceuticals. She previously served as Alnylam's Vice President and Entrepreneur-in-Residence, where she led the hepatic infectious disease strategies therapeutic area and was a key figure in partnering and in-licensing activities. She spent 14 years at Merck & Co., including as Executive Director and Department Head of the RNA therapeutics discovery biology unit. Dr. Sepp-Lorenzino received her degree in biochemistry at the University of Buenos Aires, Argentina and her M.S. and Ph.D. in biochemistry from New York University.

    "Joining the Taysha board is a truly exceptional opportunity to contribute to the development of multiple innovative gene therapies," commented Dr. Sepp-Lorenzino. "Taysha is taking a leadership position in the industry by combining decades of gene therapy experience with a portfolio of programs that have the potential to address the underlying biology of various CNS disorders in order create an engine for potential new treatments."

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our clinical trials and our research, development and regulatory plans for our product candidates, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  48. Successfully raised $96 million in Series B financing and completed $181 million IPO

    Established management team with successful track record of developing and commercializing AAV9-based gene therapies for monogenic diseases of the central nervous system

    Company on track to initiate a Phase 1/2 clinical trial of TSHA-101 in GM2 gangliosidosis by the end of 2020 and plans to submit four INDs in 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today reported its financial results for the third quarter of 2020 and provided a…

    Successfully raised $96 million in Series B financing and completed $181 million IPO

    Established management team with successful track record of developing and commercializing AAV9-based gene therapies for monogenic diseases of the central nervous system

    Company on track to initiate a Phase 1/2 clinical trial of TSHA-101 in GM2 gangliosidosis by the end of 2020 and plans to submit four INDs in 2021

    Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today reported its financial results for the third quarter of 2020 and provided a business update.

    "Just over a year ago, we founded Taysha in partnership with UT Southwestern with a mission to develop gene therapies to address monogenic diseases of the central nervous system, and to that end we have made significant progress in building a talented team that can advance our broad portfolio," said RA Session, II, President, Founder and CEO of Taysha. "During the third quarter, we raised over $275 million between our Series B financing and our IPO, providing the capital resources to support our industry-leading pipeline of 18 gene therapy programs. We have also bolstered our GMP manufacturing capabilities to ensure we have the capacity to support our development plans for multiple programs by utilizing the UTSW vector core, our internal Taysha manufacturing facility that is currently in development and our newly announced partnership with Catalent."

    Program Highlights

    TSHA-101 for GM2 gangliosidosis – Taysha secured rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-101, its bicistronic vector designed to treat GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff disease. Taysha anticipates initiating a Phase 1/2 clinical trial of TSHA-101 in Canada by end of 2020.

    TSHA-102 for Rett syndrome –Taysha announced that FDA has granted rare pediatric disease and orphan drug designation for TSHA-102, which is under development for the treatment of Rett syndrome, one of the most common genetic causes of severe intellectual disability. TSHA-102 utilizes the miRARE platform, which is designed to regulate the expression of therapeutic transgenes on a cellular basis. Taysha remains on track to file an Investigational New Drug (IND) application for the program in 2021.

    TSHA-118 for CLN1 – Taysha announced the in-licensing of an AAV9-based gene therapy program for the treatment of CLN1. TSHA-118 was originally developed in the academic lab of Steven Gray, Ph.D., Taysha's Chief Scientific Advisor. CLN1 Batten disease is a rapidly progressive rare lysosomal storage disease with no approved treatments. The company intends to initiate a Phase 1/2 clinical trial of TSHA-118 in 2021 under a currently open IND.

    Recent Business Highlights

    In October, Taysha announced a partnership with Invitae to support two different screening programs to enable rapid identification of patients with genetic disorders. DETECT Lysosomal Storage Diseases provides genetic testing for lysosomal storage disorders, including GM2 gangliosidosis and CLN1. The Behind the Seizures® program supports the diagnosis of patients with genetic epilepsies.

    In November, Taysha announced a development and manufacturing partnership with Catalent to support future preclinical and clinical supply for several of Taysha's gene therapy programs, including CLN1 and Rett syndrome.

    Taysha has also invested in building its internal management team to advance the development of its broad gene therapy pipeline. The company anticipates more than tripling its headcount from its IPO by the end of 2020 across all areas of the organization.

    Financial Results

    As of September 30, 2020, cash and cash equivalents totaled $279 million, which includes gross proceeds of $181 million from Taysha's IPO and $96 million from its Series B financing. Taysha was incorporated in September of 2019 and thus has no previous nine-month results for comparison.

    Taysha reported R&D expenses for the nine months ended September 30, 2020 of $19.6 million. The $19.6 million was primarily attributable to $10.0 million of expenses recognized pursuant to the Queen's University Agreement and the Abeona CLN1 Agreement, $4.1 million related to the manufacture of clinical trial material and $3.9 million in other sponsored research agreements for the Company's various product candidates. Additionally, Taysha incurred regulatory and clinical consulting expenses of $0.8 million and employee compensation and benefits expenses of $0.8 million, which included $0.4 million of non-cash stock-based compensation.

    Taysha reported G&A expenses of $5.0 million for the nine months ended September 30, 2020. The $5.0 million was primarily attributable to $2.7 million of compensation and benefits related to new hires, which included $0.9 million of non-cash stock-based compensation, $1.7 million in consulting fees, $0.5 million of legal expenses related to general corporate matters and $0.1 million related to insurance and other administrative expenses.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our clinical trials and our research, development and regulatory plans for our product candidates, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed and the success of our partnerships with Invitae and Catalent. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which we intend to file shortly hereafter and will be available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    Taysha Gene Therapies

    Selected Condensed Consolidated Financial Information

    (In thousands, except share and per share data)

    (Unaudited)

     

    Statements of Operations

     
    For the

    Three Months Ended

    September 30,

    2020
    For the

    Nine Months Ended

    September 30,

    2020
    For the

    Period from

    September 20, 2019

    (date of inception)

    to

    September 30,

    2019
     
    Operating expenses:
    Research and development

    $

    11,057

     

    $

    19,633

     

    $

    -

     

    General and administrative

     

    3,984

     

     

    5,002

     

     

    31

     

    Total operating expenses

     

    15,041

     

     

    24,635

     

     

    31

     

    Loss from operations

     

    (15,041

    )

     

    (24,635

    )

     

    (31

    )

    Other expense:
    Change in fair value of preferred stock tranche liability

     

    -

     

     

    (17,030

    )

     

    -

     

    Interest expense

     

    (1

    )

     

    (28

    )

     

    -

     

    Total other expense

     

    (1

    )

     

    (17,058

    )

     

    -

     

    Net loss

    $

    (15,042

    )

    $

    (41,693

    )

    $

    (31

    )

     
    Net loss per common share, basic and diluted

    $

    (1.28

    )

    $

    (3.73

    )

    $

    (0.00

    )

    Weighted average common shares outstanding, basic and diluted

     

    11,733,170

     

     

    11,176,429

     

     

    8,715,999

     

    Balance Sheet

     
    September 30,

    2020
    December 31,

    2019
     
    ASSETS
    Current assets:
    Cash and cash equivalents

    $ 278,634

    $ -

    Prepaid expenses

    604

    -

    Deferred offering costs

    -

    15

    Total current assets

    279,238

    15

    Property and equipment, net

    28

    -

    Total assets

    $ 279,266

    $ 15

     
    LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT)
    Current liabilities
    Accounts payable

    $ 8,837

    $ -

    Accrued expenses

    2,727

    150

    Due to related party

    60

    -

    Total current liabilities

    11,624

    150

    Total liabilities

    11,624

    150

     
     
    Stockholders' equity (deficit)
    Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of September 30, 2020; no shares authorized, issued and outstanding as of December 31, 2019

    -

    -

    Common stock, $0.00001 par value per share; 200,000,000 shares authorized and 37,761,435 issued and outstanding as of September 30, 2020; 10,895,000 shares authorized, 10,894,999 issued and outstanding as of December 31, 2019

    -

    -

    Additional paid-in capital

    310,450

    980

    Accumulated deficit

    (42,808)

    (1,115)

    Total stockholders'equity (deficit)

    267,642

    (135)

    Total liabilities and stockholders' equity (deficit)

    $ 279,266

    $ 15

     

    View Full Article Hide Full Article
  49. Partnership to support Taysha's broad gene therapy pipeline

    Development and manufacturing partnership will support future preclinical and clinical supply for several gene therapy programs, including CLN1 and Rett syndrome

    Taysha Gene Therapies Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing adeno-associated virus (AAV)-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, and Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced a partnership to support the…

    Partnership to support Taysha's broad gene therapy pipeline

    Development and manufacturing partnership will support future preclinical and clinical supply for several gene therapy programs, including CLN1 and Rett syndrome

    Taysha Gene Therapies Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing adeno-associated virus (AAV)-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, and Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced a partnership to support the development and manufacturing of Taysha's gene therapies at Catalent's Maryland-based gene therapy facilities.

    "Through this partnership, we will be able to enhance our existing manufacturing capabilities to support Taysha's broad gene therapy pipeline," said RA Session, II, President, Founder and CEO of Taysha. "We are focused on ensuring that we can provide access to potentially curative gene therapies for thousands of patients by establishing this robust infrastructure early."

    Taysha has an established partnership with UT Southwestern Medical Center that allows it to access the institution's CGMP-compliant manufacturing suite, which has a capacity of over 500 liters as well as additional 100-liter toxicology material capacity. In addition, Taysha intends to establish its own commercial-scale, CGMP manufacturing facility to meet future demand for its gene therapy product candidates. This new partnership with Catalent intends to rapidly expand Taysha's manufacturing capacity and will support future manufacturing needs for several of Taysha's gene therapy programs, including treatments for CLN1 and Rett syndrome.

    "Given Taysha's large and growing pipeline of gene therapies, we wanted to plan for potential increased manufacturing needs above the GMP facility at UT Southwestern and our own planned manufacturing facility," said Fred Porter, Ph.D., Chief Technical Officer for Taysha. "We believe that this partnership is critical to our strategy for future clinical and commercial supply of our gene therapy product candidates."

    "Catalent is committed to gene therapy partnerships at all stages of development and manufacturing," commented Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy. "With our experience in process and analytical development and deep expertise in adeno-associated viral vectors, combined with our growing footprint, we are able to help companies manufacture patient material and reach the clinic faster."

    Catalent has five gene therapy facilities in Maryland that provide clinical- through commercial-scale services, and house multiple CGMP manufacturing suites, including fill/finish, central services and testing laboratories, warehousing, and supply chain capabilities.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, Taysha aims to rapidly translate its treatments from bench to bedside. It has combined its team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, Taysha leverages its fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    About Catalent Cell & Gene Therapy

    With deep experience in viral vector scale-up and production, Catalent Cell & Gene Therapy is a full-service partner for adeno-associated virus (AAV) and lentiviral vectors and CAR-T immunotherapies. When it acquired MaSTherCell, Catalent added expertise in autologous and allogeneic cell therapy development and manufacturing to position it as a premier technology, development and manufacturing partner for innovators across the entire field of advanced biotherapeutics. Catalent has a global cell and gene therapy network of dedicated, large-scale clinical and commercial manufacturing facilities, and fill-finish and packaging capabilities located in both the U.S. and Europe. An experienced partner, Catalent Cell & Gene Therapy has worked with industry leaders across 70+ clinical and commercial programs.

    Taysha Gene Therapies Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates and our plans to establish a commercial-scale manufacturing facility. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission ("SEC") on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article
  50. Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021

    Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1

    Taysha Gene Therapies Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development…

    Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021

    Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1

    Taysha Gene Therapies Inc. (NASDAQ:TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Taysha anticipates it will submit an Investigational New Drug (IND) application to the FDA for TSHA-104 in 2021.

    "We have now obtained rare pediatric disease and orphan drug designations in multiple gene therapy programs, which we believe will allow us to work more effectively with the FDA as we advance our broad portfolio," said RA Session II, President, CEO and Founder of Taysha. "The receipt of these designations highlights the dedication that our team has to advancing our gene therapy pipeline as efficiently and rapidly as possible."

    Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation.

    "Being diagnosed with a mutation in the SURF1 gene is a truly devastating event for families," said Kasey Woleben, Founder of Cure SURF1 Foundation. "Taysha's commitment to developing a gene therapy for SURF1 deficiency is greatly welcomed by the patient community and has the potential to save the lives of children afflicted with this progressive disorder."

    Taysha has secured rare pediatric disease designation and orphan drug designation for multiple of its programs, including GM2 gangliosidosis, CLN1, Rett syndrome and now SURF1. In addition to these designations, the company also has fast track status for the CLN1 program.

    "SURF1 deficiency is a monogenic mitochondrial disorder and is the most common cause of cytochrome c oxidase deficient Leigh syndrome," said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. "Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency."

    The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as "rare" affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to December 11, 2022 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

    Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs as well as eligibility for market exclusivity for seven years post approval.

    About Taysha Gene Therapies

    Taysha Gene Therapies (NASDAQ:TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team's proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients' lives. More information is available at www.tayshagtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-104, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission ("SEC") on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SEC's website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

    View Full Article Hide Full Article