Rare disease drugmaker · based in Dallas, United States · founded 2019 · ticker TSHA · tayshagtx.com
Taysha Gene Therapies works on 3 rare diseases we track, including Leigh Syndrome, Rett Syndrome and 1 more. It has 4 active clinical trials, though none are looking for new volunteers right now.
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 axonalneuropathy and CLN1Batten disease but has deprioritized these to concentrate resources on Rett.
Taysha Gene Therapies pipeline
Each row lists the diseases Taysha Gene Therapies is studying at that stage, from the ones closest to approval down to the earliest work.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
These are Taysha Gene Therapies trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
4 trials underway, not taking new volunteers
Underway, not taking new volunteersPhase 3Recently updatedNCT07480564
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.
The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigationalgene therapy, in pediatric females with Rett Syndrome.
The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.
GM2 gangliosidoses are a group of autosomal recessiveneurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of 2 subunits, α- and β-, coded by the HEXA and HEXB genes, respectively. The primary purpose of the current study is to assess the safety and tolerability of TSHA101 administered via IT injection.
Follow a condition Taysha Gene Therapies is studying
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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 active and recruiting rare disease 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.
Where Taysha Gene Therapies trials take place
The trials above run at 18 study sites in 3 countries. Tap a country to see its states, cities and hospitals.
United States
14▼
Canada
3▼
United Kingdom
1▼
Rare diseases Taysha Gene Therapies works on
Each disease page lists every open trial, not only Taysha Gene Therapies's, plus approved treatments.
Tay-Sachs disease is a rare autosomal recessivelysosomal storage disorder caused by deficiency of the enzyme hexosaminidase A, leading to accumulation of GM2 gangliosides in nerve cells.
How common: 1 in 30,000 births; 1 in 3,500 in Ashkenazi Jewish population
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Taysha Gene Therapies's own site is tayshagtx.com, and our help paying for treatment page lists drugmaker programs and charity funds.
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 axonalneuropathy and CLN1Batten disease but has deprioritized these to concentrate resources on Rett.
Taysha Gene Therapies has active programs across 3 rare diseases: Leigh Syndrome, Rett Syndrome, Tay-Sachs Disease. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Taysha Gene Therapies is a rare disease specialist headquartered in Dallas, United States, founded in 2019. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Leigh Syndrome, Rett Syndrome, Tay-Sachs Disease.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Taysha Gene Therapies, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
None that we track as of November 2026. No Taysha Gene Therapies label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions.
In development: the trials list on this page shows what Taysha Gene Therapies is recruiting for now, and the FDA calendar lists any application once a decision date is public.
Taysha Gene Therapies was founded in 2019 and is headquartered in Dallas, United States. The company has been active for 7 years. It trades publicly under the ticker symbol TSHA. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.