Taysha Gets Breakthrough-Therapy Designation for Investigational Treatment
By Robb M. Stewart
Taysha Gene Therapies' investigational treatment for Rett syndrome has received breakthrough-therapy designation from the Food and Drug Administration.
The clinical-stage biotechnology company on Thursday said it also finalized alignment with the FDA on trial protocol and a statistical-analysis plan intended to support its planned biologics-license application submission for TSHA-102, an intrathecally delivered AAV9 gene therapy with disease-modifying potential. This follows the resolution of remaining clinical and statistical queries, said the Dallas-based company.
Taysha's shares jumped on the back of the news, rising 41% in premarket trading. The shares last closed at $3.18, up 84% in 2025.
Breakthrough-therapy designation is aimed at expediting the development and regulatory review of an investigational therapy intended to treat a serious condition. A drug is eligible for the designation if it demonstrates preliminary clinical evidence of substantial improvement over available treatments in one or more clinically significant endpoints.
The designation and finalized FDA alignment on the pivotal-trial protocol and analysis plan were based on the FDA's review of positive clinical evidence from part A of Phase 1/2 trials.
Write to Robb M. Stewart at robb.stewart@wsj.com
(END) Dow Jones Newswires
October 02, 2025 09:01 ET (13:01 GMT)
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