Clinical TrialJun 25, 2026, 04:02 PM
Taysha Gene Therapies Presents Positive TSHA-102 Rett Syndrome Data
AI Summary
Taysha Gene Therapies announced multiple presentations highlighting its TSHA-102 program for Rett syndrome at the 2026 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting. The presentations include longer-term REVEAL Part A data demonstrating broad, multi-domain functional impact that deepened over time. New data also supports the developmental milestone assessment (DMA) as a valid, FDA-supported primary endpoint for interventional studies, and preclinical data showed superior MeCP2 expression.
Key Highlights
- Longer-term REVEAL Part A data showed broad, multi-domain functional impact of TSHA-102 that deepened over time.
- Rett syndrome natural history data supports a stable, well-defined population for the REVEAL pivotal trial.
- New data supports the developmental milestone assessment (DMA) as a psychometrically valid, FDA-supported primary endpoint.
- Preclinical data demonstrated superior MeCP2 expression of self-complementary AAV9 for TSHA-102.
- Multiple presentations on TSHA-102 program for Rett syndrome at the 2026 IRSF Rett Syndrome Scientific Meeting.
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