Taysha Gene Therapies, Inc. provided an update on the TSHA-120 program in giant axonal neuropathy (GAN). Clinical Program Updates: TSHA-120 in GAN: Receipt of formal written meeting minutes from FDA in January 2023 following completion of Type B end-of-Phase 2 meeting. Overall approach to manufacturing of pivotal/to-be marketed product deemed appropriate pending review of a planned submission of Chemistry, Manufacturing, and Controls (CMC) data package for TSHA-120.

FDA acknowledged MFM32 as an acceptable endpoint with a recommendation to dose additional patients in a double-blind, placebo-controlled design to support BLA submission. Awaiting response from FDA on follow up questions the Company submitted on recommended design and totality of evidence required for BLA submission. TSHA-102 in Rett syndrome: Dosing of the first adult patient with Rett syndrome anticipated in first half of 2023.

Initial available clinical data for TSHA-102 in the adult Rett syndrome study expected in first half of 2023 with planned quarterly updates on available clinical data primarily on safety from the adult study thereafter. The company anticipates submission of a CTA to UK MHRA for TSHA-102 in pediatric patients with Rett syndrome in mid-2023. The company plans to submit an IND application for Rett syndrome to FDA in second half of 2023.