TSHA Taysha Gene

Taysha completes REVEAL pivotal dosing; 6-month topline and FDA feedback expected 1H 2027

Dosing of 17 patients in the REVEAL pivotal trial is now complete, triggering the 6-month interim analysis that will form the basis of the planned BLA submission. Longer-term Part A data show 100% of patients gained or regained ≥1 developmental milestone with durable, deepening functional gains through ≥12 months. Topline results from the pivotal 6-month analysis and FDA feedback on the BLA pathway are expected in 1H 2027.

Taysha Gene Therapies: TSHA-102 BLA-enabling PPQ campaign initiated

Taysha initiated the BLA-enabling Process Performance Qualification (PPQ) campaign for TSHA-102 in April 2026 using the commercial manufacturing process, with completion expected in Q4 2026. The FDA endorsed the PPQ campaign strategy following a Type C meeting in January 2026. This CMC milestone aligns manufacturing activities with the planned BLA submission timeline for TSHA-102 in Rett syndrome.

Taysha: REVEAL pivotal & ASPIRE dosing to finish Q2 2026

The 8-K states that multiple patients have already been dosed in the REVEAL pivotal trial and FDA clearance has been received to start the ASPIRE trial; both trials remain on track to complete dosing in Q2 2026. Written FDA alignment was also reached on the PPQ/comparability CMC strategy that will support the planned BLA package. No new regulatory decision or PDUFA date is announced.

TSHA-102 granted Breakthrough Therapy designation by FDA

FDA granted Breakthrough Therapy designation to TSHA-102 for Rett syndrome based on Part A REVEAL Phase 1/2 data. The designation reflects FDA recognition of the therapeutic potential of TSHA-102 and provides a pathway for expedited development and review.

Taysha receives FDA Breakthrough Therapy Designation for TSHA-102 in Rett syndrome

FDA granted Breakthrough Therapy Designation to TSHA-102 based on Part A REVEAL data showing 100% response rate for developmental milestone gain/regain. Company finalized alignment with FDA on pivotal trial protocol and SAP, including a 6-month interim analysis that may support earlier BLA submission. Enrollment in the single-arm pivotal trial is scheduled to begin in Q4 2025.