Taysha Gene Therapies
Taysha Gene Therapies works on 3 rare diseases tracked on Trial Friend, including Leigh Syndrome, Rett Syndrome, Tay-Sachs Disease, with 4 recruiting clinical trials.
Taysha Gene Therapies is a clinical-stage biotechnology company developing AAV-based gene therapies for rare monogenic diseases of the central nervous system. The company is focused on TSHA-102 for Rett syndrome, its lead program now in a pivotal trial. Taysha previously had programs for giant axonal neuropathy and CLN1 Batten disease but has deprioritized these to concentrate resources on Rett.
Focus areas at Taysha Gene Therapies
As a rare disease specialist, Taysha Gene Therapies has active clinical trial programs and drug development efforts across 3 rare diseases, including Leigh Syndrome, Rett Syndrome, Tay-Sachs Disease. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Taysha Gene Therapies, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Taysha Gene Therapies is headquartered in Dallas, United States, founded in 2019, publicly traded under the ticker symbol TSHA. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Taysha Gene Therapies Drug Pipeline
Taysha Gene Therapies has 4 active clinical trials across 2 development stages, with 4 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Taysha Gene Therapies's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Taysha Gene Therapies Clinical Trials (4)
Active and recruiting clinical trials sponsored by Taysha Gene Therapies, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
Taysha Gene Therapies Trial Locations
Taysha Gene Therapies clinical trials are running at 18 sites in 3 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (3)
Diseases targeted by Taysha Gene Therapies's clinical trial and drug development programs
Leigh syndrome is a rare inherited disorder in which the mitochondria, the parts of cells that make energy, cannot supply enough energy to the brain. Damage builds up in deep-brain regions that contro...
Rett syndrome is a rare genetic neurological disorder affecting brain development, almost exclusively in girls. After normal development for the first 6-18 months, girls experience developmental regre...
Tay-Sachs disease is a rare autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme hexosaminidase A, leading to accumulation of GM2 gangliosides in nerve cells. This progres...
Patient Resources
Organizations and resources related to Taysha Gene Therapies's rare disease focus areas
Frequently Asked Questions About Taysha Gene Therapies
Common questions about Taysha Gene Therapies's rare disease programs, clinical trials, and treatments.