Taysha Gene Therapies to Present Biodistribution Data from an Analysis Evaluating AAV9 Gene Therapy Delivery at the Upcoming 31st Annual ESGCT Congress

Core Insights - Taysha Gene Therapies, Inc. is advancing AAV-based gene therapies for severe monogenic diseases of the central nervous system (CNS) and has presented biodistribution data from studies on AAV9 gene therapy vectors in non-human primates (NHPs) [1][2][5] - The analysis indicates that intrathecal (IT) administration is an effective, safe, and minimally invasive method for delivering gene therapies targeting CNS diseases, including Rett syndrome [1][2][3] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on developing AAV-based gene therapies for severe CNS diseases, with its lead program TSHA-102 targeting Rett syndrome [5] - TSHA-102 is designed as a one-time treatment to deliver a functional form of the MECP2 gene to address the genetic root cause of Rett syndrome [3][4] - The company has received multiple designations from regulatory agencies, including Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA [3] Research Findings - The analysis of five NHP studies showed that both IT and intra-cisterna magna (ICM) administration resulted in comparable biodistribution of AAV9 vectors across brain and spinal cord regions [1][2] - The findings support the clinical potential of IT administration for outpatient use in both children and adults, reinforcing Taysha's clinical development strategy [2] Rett Syndrome Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by loss of communication and motor functions, with no approved disease-modifying therapies currently available [4] - It is estimated that Rett syndrome affects between 15,000 and 20,000 patients in the U.S., EU, and U.K. due to mutations in the MECP2 gene [4]