Taysha's Rett Syndrome Gene Therapy Gets FDA Support: Key Trial Design OKd Without Phase Meeting

Taysha Gene Therapies (NASDAQ: TSHA) reported Q1 2025 financial results and provided updates on its TSHA-102 program for Rett syndrome. The company received FDA alignment on pivotal Part B trial design, allowing direct submission of the trial protocol as an IND amendment in Q2 2025, potentially expediting study initiation. Safety data shows both high dose (1x1015 vg) and low dose (5.7x1014 vg) of TSHA-102 are well-tolerated with no treatment-related SAEs or DLTs in 10 patients across REVEAL trials. Financially, Q1 2025 showed R&D expenses of $15.6M (down from $20.7M in Q1 2024), net loss of $21.5M ($0.08 per share), and cash position of $116.6M, expected to fund operations into Q4 2026. The company will present three oral presentations at the upcoming IRSF Rett Syndrome Scientific Meeting in June 2025.

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