Taysha Gene Therapies Provides Update on Expansion of TSHA-102 Study

Taysha Gene Therapies Provides Update on Expansion of TSHA-102 Study

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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 execute across our near-term milestones and deliver on our commitments to key stakeholders, especially patients.”

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 H1 2023
  • Initial available clinical data for TSHA-102 in the adult Rett syndrome study expected in H1 2023 with planned quarterly updates on available clinical data primarily on safety from the adult study thereafter
  • Company anticipates submission of a CTA to UK MHRA for TSHA-102 in pediatric patients with Rett syndrome in mid-2023
  • Company plans to submit an IND application for Rett syndrome to FDA in H2 2023

Read the Full Press Release

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