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# Taysha Discontinues Gene Therapy Program
- URL: https://www.fdaweb.com/taysha-discontinues-gene-therapy-program/
- Published: 2023-09-20T12:00:00.000Z
- Updated: 2026-09-14T18:57:21.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5155377

Taysha Gene Therapies says it is discontinuing the development of its gene therapy TSHA-120 program as a treatment for giant axonal neuropathy (GAN) after receiving FDA feedback in a recent Type C meeting. The company says its decision is “due to challenges related to the feasibility of the study designs to support a potential BLA submission in this ultra-rare neurodegenerative disease.”

Stopping the development program will reserve cash to “support the continued clinical development of TSHA-102 in Rett syndrome, a rare neurodevelopmental disorder with no approved treatments that target the genetic root cause of the disease,” Taysha says. “We remain focused on continuing to evaluate the therapeutic potential of TSHA-102 in our ongoing REVEAL Phase 1/2 trial in adults and our planned pediatric trial.”

The company says that in 2022 it received FDA feedback that it needed to “address the heterogeneity of disease progression in GAN and the effort-dependent nature of MFM32 \[32-item Motor Function Measure is a clinician-reported outcome measure\] as a primary endpoint in an unblinded study.” At the recent meeting, Taysha submitted a new comprehensive analysis of the totality of data from a natural history and interventional trial comparing functional and biological measurements against a disease progression model.

Taysha says FDA’s new feedback indicated that the agency continues to recommend a randomized, double-blind, placebo-controlled trial as the optimal path to demonstrate efficacy in TSHA-120\. “Among other areas of feedback, the FDA also provided a potential path for a single-arm trial with an external control group matched with to-be-treated patients by multiple prognostic factors and recommended longer-term follow up to account for potential bias,” it says.