TSHA Taysha Gene Therapies Inc.

25.72
-1.15  -4%
Previous Close 26.87
Open 27.24
52 Week Low 18.16
52 Week High 33.35
Market Cap $971,224,108
Shares 37,761,435
Float 18,190,922
Enterprise Value $728,085,857
Volume 23,077
Av. Daily Volume 124,238
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Upcoming Catalysts

Drug Stage Catalyst Date
TSHA-101
GM2 gangliosidosis
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
TSHA-104
SURF1-associated Leigh syndrome
Phase 1/2
Phase 1/2
Phase 1/2 trial to be initiated by end of 2021.
TSHA-102
Rett syndrome
Phase 1/2
Phase 1/2
Phase 1/2 trial to be initiated by end of 2021.
TSHA-118
CLN1 Batten disease
Phase 1/2
Phase 1/2
Phase 1/2 trial to be initiated in 2021.

Latest News

  1. 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.

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  2. 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.

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  3. 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.

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  4. 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.

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  5. 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.

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