Taysha Gene Therapies Announces Details for Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting Reviewing Recent Updates from the TSHA-102 Clinical Program

Core Insights - Taysha Gene Therapies is advancing its TSHA-102 program for Rett syndrome, focusing on the potential to improve developmental milestones and quality of life for patients [2][3] - The company will present recent updates on TSHA-102 at the 2025 International Rett Syndrome Foundation Scientific Meeting in Boston [2][4] - TSHA-102 is a gene therapy designed to address the genetic root cause of Rett syndrome by delivering a functional form of the MECP2 gene [5] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company specializing in AAV-based gene therapies for severe monogenic diseases of the central nervous system [7] - The company has received multiple designations from the FDA for TSHA-102, including Regenerative Medicine Advanced Therapy and Fast Track designations [5] - Taysha aims to address significant unmet medical needs in the field of gene therapy, leveraging its experienced management team and proven AAV9 capsid technology [7] Disease Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by loss of communication, motor function, and intellectual disabilities [6] - The disorder is caused by mutations in the MECP2 gene, with an estimated 15,000 to 20,000 patients affected in the U.S., EU, and U.K. [6] - Currently, there are no approved therapies that modify the disease's genetic root cause, highlighting the importance of TSHA-102 [6]