Taysha Gene Therapies Announces FDA Alignment on Pivotal Part B Trial Design for TSHA-102 in Rett Syndrome

Taysha Gene Therapies, Inc.

100% of 10 patients in Part A gained/regained ≥1 developmental milestone post-TSHA-102, with ~0% likelihood from natural history data.
FDA supports single-arm open-label pivotal trial (N=15) with primary endpoint of developmental milestone gain/regain in patients ≥6 years.
High dose outperformed low dose, with dose-dependent effects deepening over time; no treatment-related SAEs or DLTs.
Analysis of IRSF natural history study (N~1100) showed patients ≥6 years in developmental plateau, <6.7% likelihood of milestone gain.
Company expects to submit protocol this quarter and initiate pivotal trial in Q3 2025.

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