Taysha to Present TSHA-102 Data at 9th World Rett Syndrome Congress

DALLAS, Sept. 26, 2024 — Taysha Gene Therapies, Inc., a biotechnology firm specializing in adeno-associated virus (AAV)-based gene therapies for severe monogenic central nervous system (CNS) disorders, announced today that it will present clinical data on its TSHA-102 program for Rett syndrome. This presentation will be made during the 9th World Rett Syndrome Congress in Queensland, Australia, from October 2-5, 2024.

The presentation will highlight previously disclosed positive clinical data from the REVEAL Phase 1/2 trials. These include data up to 52 weeks from adolescent and adult trials, as well as initial data up to 22 weeks from pediatric trials evaluating TSHA-102 in Rett syndrome. Taysha plans to provide further updates on clinical data for both high-dose and low-dose cohorts from these trials in the first half of 2025.

Dr. Sukumar Nagendran, President, and Head of Research and Development at Taysha Gene Therapies, will present the data. The session, titled "First cohort data from the REVEAL adolescent/adult and pediatric studies of TSHA-102 gene therapy for Rett syndrome," is scheduled for Friday, October 4, 2024, from 12:40-1:00 PM AEST.

TSHA-102 is an investigational gene therapy designed to treat Rett syndrome, a severe neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. The therapy involves intrathecal delivery of AAV9 to administer a functional form of MECP2 to CNS cells. The therapy utilizes miRNA-Responsive Auto-Regulatory Element (miRARE) technology to mediate MECP2 levels on a cell-by-cell basis, reducing the risk of overexpression. TSHA-102 has received several designations from regulatory bodies including Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease from the FDA, Orphan Drug from the European Commission, and Innovative Licensing and Access Pathway from the UK Medicines and Healthcare products Regulatory Agency.

Rett syndrome is characterized by mutations in the MECP2 gene, which is crucial for neuronal and synaptic function. Symptoms include loss of communication and hand function, developmental regression, motor and respiratory issues, seizures, intellectual disabilities, and reduced life expectancy. The disorder progresses through four stages: early onset stagnation, rapid regression, plateau, and late motor deterioration. Predominantly affecting females, Rett syndrome is a leading genetic cause of severe intellectual disability, with an estimated 15,000 to 20,000 affected individuals in the U.S., EU, and UK. There are currently no approved therapies addressing the genetic root cause of Rett syndrome.

Taysha Gene Therapies is committed to developing transformative medicines for severe monogenic CNS diseases. Its lead program, TSHA-102, represents a significant step forward for Rett syndrome, a disorder for which no disease-modifying therapies currently exist. Taysha's management team has extensive experience in gene therapy development and commercialization, which they leverage to expedite the transition of treatments from research to clinical application.