NEW YORK – Taysha Gene Therapies on Monday said it has dosed the first patient in a Phase I/II trial for an investigational Rett syndrome treatment.
Rett syndrome is a rare neurodevelopmental disorder in which patients can experience intellectual disabilities, loss of communication, and seizures, among other symptoms. The disorder is caused by mutations in the MECP2 gene and is most commonly seen in women.
Taysha Gene Therapies' first-in-human trial, dubbed REVEAL, is a randomized, dose-escalation study to evaluate TSHA-102, an intrathecally delivered adeno-associated virus 9 vector-based gene therapy designed to regulate cellular MECP2 protein expression. Investigators are testing TSHA-102 specifically in women with Rett syndrome caused by a loss-of-function mutation in the MECP2 gene. The study is being conducted at CHU Sainte-Justine in Montreal.
Dallas-based Taysha Gene Therapies said it plans to share early clinical safety data from the Phase I/II trial at the company's R&D Day on June 28.
The company is planning to submit a clinical trial application to test the gene therapy in pediatric patients to the UK Medicines and Healthcare products Regulatory Agency in mid-2023 and an investigational new drug application to the US Food and Drug Administration in the second half of the year.