Taysha Gene Therapies, Inc.

We are a clinical-stage biotechnology company focused on developing AAV-based gene therapies for severe monogenic diseases of the central nervous system (CNS). Our primary clinical program, TSHA-102, is being developed to treat Rett syndrome, a rare neurodevelopmental disorder for which there are no approved disease-modifying therapies that target the genetic cause. We aim to address significant unmet medical needs and improve patient and caregiver lives. Our management team has experience in gene therapy development and commercialization. We utilize this experience, our manufacturing process, and a clinically and commercially proven AAV9 capsid to advance treatments. We are currently evaluating TSHA-102 in two clinical trials for females with Rett syndrome: REVEAL and ASPIRE. The REVEAL Phase 1/2 Adolescent and Adult Trial (Part A) is an open-label, randomized, dose-escalation and dose-expansion study assessing the safety and preliminary efficacy of a single lumbar intrathecal administration of TSHA-102 in adolescent and adult females aged 12 years and older with Rett syndrome caused by MECP2 loss-of-function mutation. This trial is conducted in Canada and the United States. Six participants aged 15 to 21 years were treated, and enrollment is complete. Part A also includes the REVEAL Phase 1/2 Pediatric Trial, an open-label, randomized, dose-escalation and dose-expansion study evaluating TSHA-102’s safety and preliminary efficacy via a single lumbar intrathecal administration in pediatric females aged 5 to 8 years with Rett syndrome due to MECP2 loss-of-function mutation. This trial is conducted in the United States and Canada. Six participants aged 6 to 8 years were treated, and enrollment for this trial is also complete.