First Pediatric Dosing in Rett Syndrome Trial with TSHA-102

Taysha Gene Therapies, a company at the forefront of gene therapy, has recently dosed its first pediatric patient with TSHA-102 in a Phase 1/2 clinical trial in the United States. This marks a significant stride in the company's quest to assess the safety and efficacy of TSHA-102 in treating Rett syndrome, a severe neurodevelopmental disorder predominantly affecting young girls.

The trial, known as REVEAL, is designed to evaluate the impact of TSHA-102 on stage three female patients aged between 5 and 8 years. The therapy in question is an innovative gene transfer treatment delivered via an AAV9 vector, utilizing a miRNA-Responsive Auto-Regulatory Element (miRARE) technology. This technology is intended to regulate MECP2 levels in the central nervous system, mitigating the risk of overexpression.

The initial dosing took place at RUSH University Medical Center in Chicago under the supervision of Principal Investigator Elizabeth Berry-Kravis, a renowned figure in pediatrics, neurology, and anatomy. Berry-Kravis expressed optimism about the potential of TSHA-102 to alter the course of Rett syndrome by intervening at an early stage of the disease.

Sukumar Nagendran, President and Head of R&D at Taysha, highlighted the importance of this trial in extending the evaluation of TSHA-102 to a wider demographic, including younger patients with earlier stages of Rett syndrome. The study builds upon the ongoing adolescent and adult trial, which has shown promising improvements in multiple clinical areas for patients with advanced stages of the disease.

TSHA-102 is also under evaluation in Canada for adolescent and adult females with Rett syndrome. The company anticipates completing dosing and reporting initial safety and efficacy data for the pediatric trial's first cohort in mid-2024.

Rett syndrome, caused by mutations in the MECP2 gene, leads to profound intellectual disabilities and a range of other debilitating symptoms. Currently, there are no approved treatments that address the genetic root of the disorder. Taysha Gene Therapies is dedicated to changing this by developing transformative therapies for monogenic CNS diseases.

The company's mission is underpinned by a robust platform that integrates gene therapy drug development and commercialization expertise with the UT Southwestern Gene Therapy Program. With a focus on delivering curative treatments, Taysha aims to make a significant impact on the lives of patients and their families.

TSHA-102 has received several designations from regulatory bodies, including Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, and Orphan Drug designation from the European Commission, underscoring its potential as a groundbreaking treatment for Rett syndrome.