Taysha Gene Therapies announced on October 2, 2025, that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to TSHA-102 for the treatment of Rett syndrome. This designation is intended to expedite the development and regulatory review of investigational therapies that demonstrate preliminary clinical evidence of substantial improvement over available treatments for serious conditions.
The Breakthrough Therapy designation was based on the FDA’s review of positive clinical evidence of safety and efficacy from all 12 patients treated in Part A of the REVEAL Phase 1/2 trials. This data showed a generally well-tolerated safety profile and a 100% response rate for the pivotal trial primary endpoint of gaining or regaining at least one defined developmental milestone, with natural history data indicating a less than 6.7% likelihood of such gains without treatment.
Additionally, Taysha finalized alignment with the FDA on the REVEAL pivotal trial protocol and statistical analysis plan (SAP), resolving remaining clinical and statistical queries. Key design elements, including a 6-month interim analysis, remain unchanged and have the potential to expedite the Biologics License Application (BLA) submission by at least two full quarters. The company remains on track to initiate patient enrollment for the REVEAL pivotal trial in the fourth quarter of 2025.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.