TSHA-102

May 06, 2026 — Taysha Gene Therapies (Nasdaq: TSHA) reported Q1 2026 financial results and a corporate update focused on TSHA-102, an intrathecal self-complementary AAV9 gene therapy in development for Rett syndrome. After a Breakthrough Therapy Type B FDA meeting, the company reaffirmed alignment on the TSHA-102 BLA pathway, including pivotal trial design/endpoints and a potential approval submission based on the REVEAL pivotal trial 6‑month interim analysis. REVEAL (single-arm, open-label) is dosing 15 females ages 6 to <22 at 1×10^15 vg; primary endpoint is response rate defined by gain/regain of ≥1 of 28 natural history-defined developmental milestones assessed via blinded central video review. ASPIRE is enrolling three females ages 2 to <4 to support safety for broader labeling; at least 3 months of safety data are planned for the BLA. As of a May 2026 cutoff, TSHA-102 showed no treatment-related SAEs or DLTs across REVEAL Phase 1/2 and pivotal trials. Taysha initiated a BLA-enabling PPQ manufacturing campaign in April 2026 (expected completion Q4 2026) and expects to complete dosing in REVEAL and ASPIRE in Q2 2026.

Financials (Q1 2026): R&D $33.8M (vs $15.6M), G&A $9.7M (vs $8.2M), net loss $42.4M ($0.12/share). Cash and equivalents were $276.6M (funding expected into 2028).