Taysha Gene Therapies (NASDAQ:TSHA) has received FDA Breakthrough Therapy designation for TSHA-102, its gene therapy treatment for Rett syndrome. The designation was granted based on clinical evidence from 12 patients in Part A of the REVEAL Phase 1/2 trials, showing a 100% response rate in developmental milestone achievement.
The company has also finalized FDA alignment on the REVEAL pivotal trial protocol and statistical analysis plan. Key elements include a 6-month interim analysis that could expedite BLA submission. The pivotal trial will evaluate 15 female patients aged 6-22 years, with a minimum success threshold of 33% response rate. Patient enrollment is scheduled to begin in Q4 2025.
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