Taysha Gene Therapies says IDMC recommends continuation of REVEAL trial

Taysha Gene Therapies announced that the Independent Data Monitoring Committee recommended the continuation of the REVEAL Phase 1/2 trial and that dosing of the second patient in the first cohort can proceed. The decision follows a pre-specified IDMC review of initial clinical data from the first patient dosed with TSHA-102 following the 42-day evaluation period. The REVEAL Phase 1/2 trial is a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome due to MECP2 loss-of-function mutation. TSHA-102 is administered as a single lumbar intrathecal injection. Dose escalation will evaluate two dose levels of TSHA-102 sequentially. The maximum tolerated dose or maximum administered dose established will then be administered during dose expansion.