Neurogene’s NGN-401 gene therapy for Rett syndrome has exhibited promising safety results in initial clinical trials. After several months of observation, the therapy has been well-tolerated by all three patients, with no significant adverse events or signs of excessive MeCP2 protein production.
NGN-401 utilizes Neurogene’s EXACT transgene regulation technology to deliver a full-length MECP2 gene to specific brain regions affected by Rett syndrome. Dr. Rachel McMinn, Neurogene’s CEO, emphasizes the importance of this early safety data, as conventional gene therapy approaches have faced challenges in achieving therapeutic protein levels without causing harmful overexpression.
Neurogene plans to present detailed safety data at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting. The expected safety and efficacy update in the fourth quarter of 2024 will provide further insights into the potential of NGN-401 as a treatment for Rett syndrome. The therapy aims to address the narrow therapeutic window associated with this complex neurological disorder and offers hope for patients and families affected by it.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
