Taysha Gene Therapies Inc (NASDAQ:TSHA) saw its stock leap 39% in premarket trading on Thursday after the company revealed that its gene therapy for Rett syndrome received Breakthrough Therapy designation from the U.S. Food and Drug Administration.
The clinical-stage biotech firm also confirmed that it has reached alignment with the FDA on the pivotal trial protocol and statistical analysis plan for TSHA-102, its intrathecally delivered AAV9 gene therapy. These steps are designed to support the upcoming Biologics License Application (BLA) submission for the therapy.
The FDA granted the Breakthrough Therapy designation, which accelerates development and regulatory review, based on clinical evidence from Part A of the REVEAL Phase 1/2 trials. Results demonstrated a 100% response rate for the primary endpoint of achieving or regaining at least one defined developmental milestone.
“Breakthrough Therapy designation highlights the FDA’s recognition of both the significant unmet medical need across the estimated 10,000 patients suffering from Rett syndrome in the U.S. and the potential of TSHA-102 to redefine the treatment paradigm for this devastating disease,” said Rumana Haque-Ahmed, Chief Regulatory Officer of Taysha.
The REVEAL pivotal trial will assess a single intrathecal dose of high-dose TSHA-102 in 15 female patients aged 6 to under 22 with Rett syndrome. The study includes a six-month interim analysis, which may form the basis for the BLA submission, with patient enrollment scheduled to begin in the fourth quarter of 2025.
Taysha Gene Therapies stock price
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