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Taysha Gene Therapies Announces Formation of Independent Scientific Advisory Board

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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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