FDA grants Breakthrough Therapy designation for TSHA-102 in Rett syndrome; pivotal trial protocol finalized

Taysha Gene Therapies, Inc.

FDA Breakthrough Therapy designation for TSHA-102 based on Part A data from 12 patients showing 100% response rate on milestone gain.
FDA alignment finalized on REVEAL pivotal trial protocol and SAP; 6-month interim analysis could expedite BLA by at least two quarters.
Pivotal trial to enroll 15 females aged 6-22 with primary endpoint of milestone gain; enrollment on track for Q4 2025.
Clinical data also showed dose-dependent improvements on R-MBA and CGI-I; safety generally well-tolerated.

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