About Us | ScientistTSHA Taysha Gene Therapies Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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TSHA-101
GM2 gangliosidosis
|
Phase 1/2
Phase 1/2
|
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|
|
TSHA-104
SURF1-associated Leigh syndrome
|
Phase 1/2
Phase 1/2
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TSHA-118
CLN1 Batten disease
|
Phase 1/2
Phase 1/2
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|
TSHA-102
Rett syndrome
|
Phase 1/2
Phase 1/2
|
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
|
Latest News
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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 source version on businesswire.com: https://www.businesswire.com/news/home/20210802005344/en/
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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:
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Frederick Porter, Ph.D.
Chief Technical Officer, Taysha Gene Therapies
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Greg Gara
Senior Vice President, Manufacturing, Taysha Gene Therapies
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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 source version on businesswire.com: https://www.businesswire.com/news/home/20210720005181/en/
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Frederick Porter, Ph.D.
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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 source version on businesswire.com: https://www.businesswire.com/news/home/20210708005154/en/
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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 source version on businesswire.com: https://www.businesswire.com/news/home/20210621005244/en/
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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:
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Steven Gray, Ph.D.
Chief Scientific Advisor, Taysha Gene Therapies
Associate Professor, Department of Pediatrics at UT Southwestern
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Berge Minassian, M.D.
Chief Medical Advisor, Taysha Gene Therapies
Division Chief, Pediatric Neurology at UT Southwestern
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Rachel Bailey, Ph.D.
ssistant Professor, Department of Pediatrics at UT Southwestern
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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 source version on businesswire.com: https://www.businesswire.com/news/home/20210617005380/en/