Taysha Gene Therapies Announces Progress Across TSHA-102 Pivotal Gene Therapy Program in Rett Syndrome

Core Insights - Taysha Gene Therapies has initiated dosing in the REVEAL pivotal trial for TSHA-102, a gene therapy for Rett syndrome, with the first patient dosed in Q4 2025 and enrollment progressing across multiple sites [1][2] - The company has reached written alignment with the FDA regarding the inclusion of at least three months of safety data from the ASPIRE trial in the planned Biologics License Application (BLA) submission, aiming for a broad label for patients aged two years and older with Rett syndrome [2][5] - Completion of dosing in both the REVEAL and ASPIRE trials is anticipated by Q2 2026, with longer-term safety and efficacy data updates expected in H1 2026 [1][6] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases affecting the central nervous system [9] - The lead program, TSHA-102, is designed as a one-time treatment targeting the genetic root cause of Rett syndrome by delivering a functional form of the MECP2 gene [7] - The company has received multiple designations from the FDA, including Breakthrough Therapy and Fast Track, indicating its potential significance in addressing unmet medical needs [7] Disease Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by severe developmental regression and loss of motor and communication skills [8] - It is estimated that between 15,000 and 20,000 patients in the U.S., EU, and U.K. are affected by Rett syndrome, highlighting a significant market opportunity for effective treatments [2][8]