Taysha Gene Therapies Releases TSHA-102 Clinical Program Updates

Details of  Taysha Gene Therapies, Inc. (NASDAQ:TSHA)’ Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting, which will review recent TSHA-102 Clinical Program updates, have been announced.

Taysha Gene Therapies releases TSHA-102 clinical program updates

A laboratory technician preparing a gene therapy sample in a sterile environment.

Safety and early efficacy have been shown by phase 1/2 REVEAL study data from low (5.7×10¹⁴ vg) and high (1×10¹⁵ vg) dosages in pediatric and adult patients.

FDA alignment on outcomes for the key Part B trial was supported by caregiver-reported milestone gains. Taysha Gene Therapies, Inc. (NASDAQ:TSHA)’s preclinical biodistribution data, caregiver insights, and TSHA-102 trial outcomes are among the presentations.

TSHA-102 is an AAV9-based gene therapy that is administered intrathecally to treat Rett syndrome by providing functional MECP2. It uses miRARE technology to control MECP2 expression while minimizing toxicity. In the US, the treatment is designated as an RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease; in the EU and UK, it is designated as an Orphan Drug/ILAP.

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