Taysha Gene Therapies(US:TSHA) Benzingaยท2025-05-29 17:06Core Insights - Taysha Gene Therapies Inc. announced details of its pivotal Part B trial design for TSHA-102 after receiving alignment from the FDA [1] - The company revealed clinical data from Part A of the REVEAL Phase 1/2 trials for TSHA-102 in treating Rett syndrome, a rare genetic disorder [2] Trial Design and Data - The pivotal Part B trial will include a single-arm, open-label design with an intended enrollment of 15 female patients aged 6 years and older [6] - The primary endpoint will assess the gain or regain of developmental milestones, while safety will be evaluated in a younger cohort aged 2-6 years [6] - The FDA advised the company to submit the trial protocol as an amendment to its IND application, streamlining the process [6] Clinical Efficacy - Efficacy data showed that 100% of patients gained at least one developmental milestone across key functional domains post-treatment with TSHA-102 [7] - A total of 22 developmental milestones were achieved among 10 patients, with significant early gains observed [7] - The high-dose cohort demonstrated a 100% responder rate 25% faster than the low-dose cohort, indicating accelerated functional benefits [7] Safety Profile - Both high and low doses of TSHA-102 were generally well tolerated, with no serious adverse events or dose-limiting toxicities reported [7] - All treatment-emergent adverse events related to TSHA-102 were mild to moderate in severity [7] Financial Activity - Taysha Gene Therapies priced an underwritten public offering of 46.9 million shares at $2.75 per share, raising $200 million in gross proceeds [4] - The company's stock saw a 9% increase, reaching $2.66 [5]