TSHA-102

May 06, 2026 — Taysha Gene Therapies, Inc. (Nasdaq: TSHA) reported Q1 2026 financial results and provided a corporate update focused on TSHA-102, its intrathecal self-complementary AAV9 gene therapy for Rett syndrome. Following a Breakthrough Therapy Type B meeting, the company reaffirmed FDA alignment on the TSHA-102 Biologics License Application (BLA) pathway, including pivotal trial design/endpoints and the potential to submit for approval based on the REVEAL pivotal trial 6‑month interim analysis.

Operationally, dosing has progressed in the single-arm, open-label REVEAL pivotal trial (n=15 females age 6 to <22; high dose 1×10^15 vg; primary endpoint response rate defined as gain/regain of ≥1 of 28 natural-history milestones). Enrollment continues in the ASPIRE safety trial (n=3 females age 2 to <4; scaled high dose), with both trials on track to complete dosing in Q2 2026. As of a May 2026 cutoff, TSHA-102 showed no treatment-related serious adverse events or dose-limiting toxicities across REVEAL Phase 1/2 and pivotal patients. A BLA-enabling PPQ manufacturing campaign began April 2026 (expected completion Q4 2026).

Financially, Q1 2026 R&D was $33.8M (vs $15.6M Q1 2025), net loss $42.4M ($0.12/share), and cash/cash equivalents were $276.6M (expected runway into 2028).