Healthcare Professionals

We are committed to providing healthcare professionals with the information and programs that will support their efforts to treat rare, monogenic diseases of the central nervous system.

Clinical Trials

REVEAL Adolescent and Adult Trial

Taysha is evaluating the safety and preliminary efficacy of TSHA-102 in the REVEAL Adolescent and Adult Trial in Canada and the United States, a Phase 1/2 first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in females aged 12 and older with Rett syndrome due to MECP2 loss-of-function mutation.

For more information on the REVEAL Adolescent and Adult Trial (NCT05606614), visit:

REVEAL Pediatric Trial

Taysha is evaluating the safety and preliminary efficacy of TSHA-102 in the REVEAL Pediatric Trial in the United States and United Kingdom, a Phase 1/2 first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in pediatric females 5-8 years old with Rett syndrome due to an MECP2 loss-of-function mutation.

For more information on the REVEAL Pediatric Trial (NCT06152237), visit:

For questions about programs and clinical trials, please contact .

Scientific Posters & Publications

  • 2024 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting

    TSHA-102 gene therapy for Rett syndrome: First-cohort data from the REVEAL Adolescent/ Adult and Pediatric studies

  • British Paediatric Neurology Association 2024 Conference

    Early safety and efficacy observations following the first use of TSHA-102 gene therapy in patients with Rett Syndrome

  • European Society of Gene & Cell Therapy 30th Annual Congress 2023

    The microRNA-responsive autoregulatory element from TSHA-102 for Rett Syndrome modulates therapeutic transgene expression in response to cellular MeCP2 in mouse and human cell lines

    Early safety and efficacy observations following the first use of TSHA-102 gene therapy in a patient with Rett Syndrome

  • Rett Syndrome Research Trust 2023 Genetic Medicines Summit

    Early safety and efficacy observations following the first use of TSHA-102 gene therapy in a patient with Rett Syndrome

    Design of the REVEAL Pediatric study of TSHA-102 gene therapy for Rett Syndrome

  • American Society of Gene and Cell Therapy 26th Annual Meeting 2023

    A Human-ready Regulated AAV9/miniMECP2- miRARE Gene Therapy (TSHA-102) Improves Survival, Weight, and Behavior After Intracerebroventricular (ICV) Dosing in the Neonatal Knockout Rett (RTT) Mouse Model

  • European Society of Gene & Cell Therapy 29th Annual Congress 2022

    Assessment of Safety and Biodistribution of a miniMECP2 AAV9 Vector for Gene-replacement Therapy of Rett Syndrome in Non-human Primates (NHPs)

    Assessment of Safety of miniMECP2 AAV9 vector (TSHA-102) for Gene-replacement Therapy of Rett Syndrome in Rats

  • American Society of Gene and Cell Therapy 25th Annual Meeting 2022

    Safety and Biodistribution Assessment in Non-Human Primates (NHP) of a miniMECP2 AAV9 Vector for Gene-Replacement Therapy of Rett Syndrome

    Safety Assessment of High-Dose miniMECP2 AAV9 Gene-Replacement Therapy (TSHA-102) for Rett Syndrome in Rats

  • International Rett Syndrome Foundation Meeting 2022

    Safety and Biodistribution Assessment in Non-human Primates (NHPs) of a miniMECP2 AAV9 Vector for Gene-replacement Therapy of Rett Syndrome

  • ASGCT 24rd Annual Meeting 2021

    Keynote Title: Attracting Capital and Building a Company in the Gene Therapy Space
    Presenter: RA Session II, President, CEO and Founder, Taysha Gene Therapies
    Session: Emerging Issues in Market Access Keynote Address

    Panel Title: Working & Thriving in Industry – What You Need to Know to Prepare and Succeed
    Presenter: Steven Gray, Ph.D., Associate Professor, UT Southwestern
    Session: Transitioning from Academics to Industry Session

    Panel Title: Learning from Experience – Case Studies of Transitions from Academia to Industry
    Presenter: Fred Porter, Ph.D., Chief Technical Officer, Taysha Gene Therapies
    Session: Transitioning from Academics to Industry

    Panel Title: Understanding the Research Process: Being Equipped for Success
    Presenter: Steven Gray, Ph.D., Associate Professor, UT Southwestern
    Session: Patient Advocates’ Role in Advancing Gene Therapy

  • 17th Annual World Symposium 2021
    Virtual Poster Presentation

    Title: Co-creating a gene therapy clinical trial with GM2 gangliosidosis caregivers: A Virtual approach to patient engagement
    Presenter: Kristin LaBounty Phillips, Senior Director, Community Engagement, Taysha Gene Therapies

  • ASGCT 23rd Annual Meeting 2020
    Oral Presentations

    Title: A New Approach for Designing a Feedback-Enabled AAV Genome Improves Therapeutic Outcomes of MiniMeCP2 Gene Transfer in Mice Modeling Rett Syndrome (RTT)
    Presenter: Sarah E. Sinnett, Ph.D., Assistant Professor, Pediatrics, UT Southwestern Medical Center
    Session: AAV Gene Delivery for CNS Disorders

    Title: Intrathecal Delivery of Human Bicistronic Hexosaminidase Vector (TGTX-101) to Correct Sandhoff Disease in a Murine Model: A Dosage Study
    Presenter: Alex E. Ryckman, Centre for Neuroscience Studies, Queen’s University
    Session: Main session, AAV Gene Delivery for CNS Disorders

    Title: Direct Vagus Nerve Injection of AAV9 as a Treatment Approach for Autonomic Dysfunction in Giant Axonal Neuropathy
    Presenter: Rachel M. Bailey, Ph.D., Assistant Professor, Neuroscience, UT Southwestern Medical Center
    Session: New Techniques in Gene Therapy for Neurological Disorders

    Poster Presentations

    Title: SMRT Sequencing Allows High-Throughput Analysis of a Whole Capsid Shuffled AAV Capsid Library Following CNS Selection in Mice and NHPs
    Presenter: Widler Casy, Ph.D., Postdoctoral Researcher, UT Southwestern Medical Center
    Session: AAV Vectors – Virology and Vectorology

    Title: Gene Replacement Therapy for SURF1-Related Leigh Syndrome Using AAV9
    Presenter: Qinglan Ling, Ph.D., Postdoctoral Researcher, UT Southwestern Medical Center
    Session: Neurologic Diseases

    Title: A Dosage Study to Assess the Long-Term Effects of Gene Therapy for AB-Variant GM2 Gangliosidoses in a Mouse Model Using Adeno-Associated Virus Serotype 9
    Presenter: Natalie M. Deschenes, Centre for Neuroscience Studies, Queen’s University
    Session: Main Session: Neurologic Diseases

    Title: Preclinical Safety and Efficacy of AAV9 Gene Replacement Therapy for SLC6A1 Disorder
    Presenter: Frances Shaffo, Ph.D., Postdoctoral Researcher, UT Southwestern Medical Center
    Session: Neurologic Diseases

Expanded Access Policy

At Taysha, we are focused on running well-designed clinical trials to assess the safety and efficacy of investigational therapies and provide the highest probability of bringing an approved therapy to the broader patient community. We are collaborating closely with patients, caregivers, patient organizations and medical experts to conduct clinical trials.

Expanded Access, sometimes called Managed Access, Compassionate Use, Named Patient or Early Access, is a potential pathway for a patient with an immediately life-threatening condition, serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Taysha evaluates physician requests for Expanded Access outside of a clinical study on a case-by-case basis when certain conditions are met. All Expanded Access requests must be submitted by the patient’s treating physician.

Patients or caregivers who are interested in learning more should speak with their physician. Physicians may submit questions about clinical trials and Taysha’s Expanded Access Policy to .