REVEAL Part A data show multi-domain functional gains sustained through 12 months with TSHA-102 across age cohorts. Preclinical studies demonstrate superior MeCP2 expression with self-complementary AAV9 and natural history data confirm a plateau after age six, supporting the developmental milestone assessment as the primary endpoint.
The Phase 1/2 REVEAL Part A trial evaluated TSHA-102 in pediatric and adolescent/adult cohorts, revealing broad multi-domain functional improvements that deepened over time and remained durable through at least 12 months post-dose, regardless of initial age or disease severity.
Head-to-head preclinical comparisons showed self-complementary AAV9 achieved higher levels of MeCP2 expression than single-stranded AAV9 in central nervous system models, reinforcing intrathecal delivery as an effective method to ensure robust gene transfer.
Analysis of natural history data identified a clear developmental plateau after age six, confirming a stable, well-defined patient population and validating the developmental milestone assessment as an FDA-supported primary endpoint for the upcoming pivotal REVEAL trial.
