Clinical TrialJun 22, 2026, 08:17 AM
Taysha Completes REVEAL Pivotal Dosing; TSHA-102 Shows Durable Clinical Gains
AI Summary
Taysha Gene Therapies announced the completion of dosing in its REVEAL pivotal trial for TSHA-102, an investigational gene therapy for Rett syndrome. The company also reported positive longer-term clinical data from Part A of the REVEAL Phase 1/2 trials, showing that 100% of patients achieved at least one developmental milestone by 12 months post-treatment, with an average of 26 functional gains per patient. TSHA-102 demonstrated a durable and deepening treatment effect and was generally well-tolerated. These robust results support a potential Biologics License Application (BLA) submission, with topline data and FDA feedback anticipated in the first half of 2027.
Key Highlights
- Completed dosing of 17 patients in the REVEAL pivotal trial for TSHA-102 in Rett syndrome.
- 100% of REVEAL Part A patients (N=12, 6-21 years) gained/regained at least one developmental milestone by 12 months.
- Observed 310 total functional gains (~26 per patient) at 12 months post-TSHA-102 treatment.
- TSHA-102 was generally well-tolerated with no treatment-related serious adverse events or dose-limiting toxicities (N=29).
- Topline data from 6-month interim analysis and FDA feedback on BLA submission pathway expected 1H 2027.
- ASPIRE trial ongoing, dosing 4 patients (aged 2 to <4 years) to support a broad label for TSHA-102.
- BLA-enabling Process Performance Qualification (PPQ) campaign for TSHA-102 expected in Q4 2026.
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