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Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy and a 2023 Corporate Outlook
Type B end-of-Phase 2 meeting with U.S. Food and Drug Administration (FDA) provided additional clarity for TSHA-120 for the treatment of giant axonal
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"Type B end-of-Phase 2 meeting with U.S. Food and Drug Administration (FDA) provided additional clarity for TSHA-120 for the treatment of giant axonal neuropathy (GAN) ultra-rare disease program - FDA acknowledged MFM32 as an acceptable endpoint with a recommendation to dose additional patients in a double-blind, placebo-controlled design to support Biologics License Application (BLA) submission Organizational and business review by new management with operational, structural and personnel changes implemented to enhance execution Dosing of first adult patient with Rett syndrome from ongoing trial in Canada expected in H1 2023; update of initial available clinical data anticipated in H1 2023 with quarterly updates primarily on safety thereafter Submission of Clinical Trial Application (CTA) to United Kingdom (UK) MHRA for TSHA-102 in pediatric patients with Rett syndrome expected in mid-2023 Submission of an Investigational New Drug (IND) application for TSHA-102 for Rett syndrome to FDA planned in H2 2023 Conference call and live webcast today at 4:30 PM Eastern Time DALLAS, Jan. 31, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical -stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic rare diseases of the central nervous system (CNS), today provided an update on the TSHA-120 program in giant axonal neuropathy (GAN) and a corporate outlook for 2023. “We expect to deliver on several key milestones in 2023, including the generation of first-in-human adult clinical data in Rett syndrome, CTA submission to MHRA to enable initiation of our pediatric Rett syndrome program and submission of an IND for Rett syndrome in the U.S. to further expand our clinical study footprint. For our GAN program, we received the formal FDA meeting minutes and recently submitted follow up questions to clarify some of their recommendations including the feasibility of a proposed study design and the totality of evidence required for BLA submission. Their feedback will help inform next steps for the program in this ultra-rare indication with no approved treatments,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “I believe that the operational, structural and personnel actions recently implemented position us well to exe...