Taysha Secures FDA BLA Alignment for TSHA-102 and Targets Q2 Dosing Completion
Taysha Gene Therapies secured FDA alignment on its TSHA-102 BLA pathway, agreeing on pivotal trial design and a potential six-month interim analysis filing from the REVEAL study. Dosing in REVEAL and ASPIRE trials is set to complete in Q2 2026 with no treatment-related serious adverse events or dose-limiting toxicities reported.
1. FDA Alignment on BLA Pathway
Taysha Gene Therapies reaffirmed alignment with FDA on TSHA-102’s BLA submission pathway following a breakthrough therapy Type B multidisciplinary meeting, agreeing on pivotal trial design, endpoints and the option to submit based on a six-month interim analysis from the REVEAL pivotal trial.
2. Trial Enrollment and Dosing Status
The REVEAL pivotal trial has advanced dosing of high-dose TSHA-102 across multiple sites in female patients aged 6 to <22 years with Rett syndrome, while enrollment continues in the ASPIRE safety trial for patients aged 2 to <4 years with scaled dosing; both trials are on track to complete dosing in the second quarter of 2026.
3. Safety and Efficacy Data Timeline
TSHA-102 maintains a favorable tolerability profile, with no treatment-related serious adverse events or dose-limiting toxicities reported in either Phase 1/2 or pivotal studies as of May 2026, and the company plans to present longer-term safety and efficacy data, including 12-month follow-up, from Part A of the Phase 1/2 trial in Q2 2026.
4. BLA-Enabling Manufacturing Progress
Manufacturing and regulatory preparations are underway, with a BLA-enabling Process Performance Qualification campaign launched in April 2026 using the commercial manufacturing process and scheduled for completion in Q4 2026 to support the planned BLA submission.