Neurogene Inc.’s NGN-401 gene therapy for Rett syndrome has been selected for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. This program provides enhanced communication between sponsors and the FDA to expedite the development of therapies for rare diseases.
NGN-401 was chosen for its potential clinical benefits and the maturity of its development program. The START Program will offer Neurogene frequent guidance and ad-hoc discussions to address development issues, including clinical study design, patient population selection, and control group choice.
NGN-401 is being evaluated in a Phase 1/2 clinical trial, assessing its safety, tolerability, and preliminary efficacy in pediatric female patients with Rett syndrome. Neurogene has reported positive safety data from the first three patients treated; interim efficacy data is expected in the fourth quarter of 2024.
The FDA’s selection of NGN-401 highlights the potential of this therapy to address the unmet medical needs of patients with Rett syndrome. The START Program will accelerate NGN-401’s development and bring it closer to a potential registrational study for this devastating condition.
About NGN-401:
NGN-401 is an investigational AAV9 gene therapy designed as a single-dose treatment for Rett syndrome. It delivers the full-length human MECP2 gene, which is deficient or mutated in Rett syndrome patients. NGN-401 aims to restore the normal function of MECP2 and improve the symptoms of Rett syndrome.
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.
