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FDA Grants Breakthrough Therapy for Taysha's Rett Syndrome Treatment

FDA recognizes Taysha's Rett syndrome treatment as a breakthrough. Early trial results show potential for significant developmental gains.

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In the image we can see there is a bottle on which it's written "Share a coke with TRAVIS".
In the image we can see there is a bottle on which it's written "Share a coke with TRAVIS".

FDA Grants Breakthrough Therapy for Taysha's Rett Syndrome Treatment

Taysha Gene Therapies has received a significant boost for its Rett syndrome treatment, TSHA-102. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation, and the company has finalized alignment with the FDA on the pivotal trial protocol and statistical analysis plan (SAP).

TSHA-102, an intrathecally delivered AAV9 gene therapy, has shown promising results in early trials. In Part A of the REVEAL study, all patients gained or regained one or more developmental milestones, resulting in a 100% responder rate. Notably, no treatment-related serious adverse events or dose-limiting toxicities were reported.

The REVEAL pivotal trial protocol includes a 6-month interim analysis, which could serve as the basis for a Biologics License Application (BLA) submission. However, the path to approval is not without risks. The small study population and the slightly subjective nature of the developmental milestones should be considered.

Taysha Gene Therapies has a strong cash position of $312.8 million, expected to last into 2028. Sukumar Nagendran, President and Head of R&D, expressed satisfaction with the FDA alignment on the pivotal trial protocol and positive regulatory updates on TSHA-102 for Rett syndrome. The Breakthrough Therapy designation and finalized FDA alignment were based on positive clinical evidence from Part A of the REVEAL Phase 1/2 trials. A response rate of 33% (5 out of 15 patients) is the minimum threshold for success sufficient to reject the null hypothesis in the REVEAL pivotal trial.

TSHA-102's progress towards FDA approval is encouraging, with positive trial results and regulatory support. While challenges remain, Taysha Gene Therapies' robust cash position and commitment to the REVEAL trial indicate a strong commitment to advancing this potential treatment for Rett syndrome.

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