Completion of Part A Dosing
TSHA-102 has completed dosing of ten patients in Part A, including six in the high dose cohort and four in the low dose cohort, demonstrating an encouraging safety profile with no treatment-related serious adverse events or dose-limiting toxicities.
Positive Safety Profile
TSHA-102 was generally well tolerated across all dosed patients, with no treatment-related SAEs or DLTs observed, which is encouraging for advancing to the pivotal phase.
Encouraging Functional Gains
Patients treated with TSHA-102 demonstrated functional gains such as improvements in motor function, socialization, communication, and seizure events, which are meaningful improvements for caregivers.
Strong Financial Position
Taysha Gene Therapies ended 2024 with $139 million in cash and cash equivalents, expected to support planned operations into the fourth quarter of 2026.
Regulatory Progress with FDA
Ongoing productive discussions with the FDA through the RMAT mechanism to solidify the regulatory pathway for TSHA-102, with an update on pivotal Part B trial design expected in the first half of 2025.