Taysha Gene Therapies Presents New Supplemental Data Analysis from Part A of the REVEAL Phase 1/2 Trials for TSHA-102 in Rett Syndrome at the 54th CNS Annual Meeting

Core Insights - Taysha Gene Therapies announced a 100% response rate in a pivotal trial for TSHA-102, with all 10 patients gaining or regaining at least one developmental milestone as defined by natural history data [1][3] - A supplemental analysis revealed that 100% of patients demonstrated multiple skill gains beyond the predefined milestones, indicating broad functional improvements [1][3] - The findings highlight significant functional gains across 22 developmental milestones and 165 additional skills, impacting daily living activities [1][3] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on AAV-based gene therapies for severe monogenic diseases of the CNS, with TSHA-102 being developed for Rett syndrome [5][7] - TSHA-102 is designed as a one-time treatment to address the genetic root cause of Rett syndrome by delivering a functional form of MECP2 to CNS cells [5] - The company has received multiple designations from the FDA, including Breakthrough Therapy and Fast Track, indicating its potential significance in treating Rett syndrome [5] Disease Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by loss of communication and motor functions, and is caused by mutations in the MECP2 gene [6] - The disorder affects an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K., with no approved therapies targeting the genetic cause [6]