Taysha Advances Rett Gene Therapy Program With Pivotal Dosing, FDA Alignment and Q2 Data Timeline

Overview

• The FDA granted Breakthrough Therapy designation for TSHA-102 and provided written alignment on the REVEAL pivotal and ASPIRE study designs, including support for pooling data and a PPQ plan.
• Taysha initiated the REVEAL pivotal trial in Q4 2025, has now dosed multiple patients across sites, and remains on track to complete dosing in the second quarter of 2026.
• Early Part A results showed a 100% response across 10 treated patients and an 83% six‑month response in the high‑dose cohort, with no treatment‑related serious adverse events or dose‑limiting toxicities reported to the March 2026 cutoff.
• Twelve‑month follow‑up data from REVEAL Part A are expected in the second quarter of 2026.
• The company reported a 2025 net loss of $109.0 million and ended the year with $319.8 million in cash plus $50 million raised in Q4, which management says funds operations into 2028 as commercialization planning progresses.