Neurogene to Present ICV NGN-401 Data Showing Superior Brain Biodistribution
Neurogene will present its ICV NGN-401 delivery strategy at the ASGCT meeting May 12, highlighting preclinical data showing broader brain biodistribution versus intrathecal-lumbar administration in Rett syndrome models. Interim Phase 1/2 results at a 1E15 vg dose show durable multidomain gains and a well-tolerated safety profile, supporting Embolden trial advancement.
1. Poster Presentation Overview
Neurogene will host a poster presentation at the American Society of Gene and Cell Therapy Annual Meeting on May 12 from 5:00 to 6:30 p.m. ET in the MCEC Exhibit and Poster Hall. The session, led by Dr. Daniel J. Curry, will outline the rationale for using intracerebroventricular administration to optimize CNS gene therapy delivery.
2. Preclinical Biodistribution Findings
Preclinical studies comparing ICV to intrathecal-lumbar administration of NGN-401 in Rett syndrome models demonstrated significantly broader distribution to key brain regions with the ICV route. These data underpin the selection of ICV delivery to maximize therapeutic reach and efficacy for NGN-401.
3. Phase 1/2 Interim Data
Interim results from the ongoing Phase 1/2 trial at a 1E15 vg dose reported durable, multidomain developmental milestone gains among participants. The ICV administration was generally well tolerated, with safety observations attributed primarily to the gene therapy product rather than the delivery method.
4. Next Steps and Registrational Trial
Based on preclinical and interim clinical findings, Neurogene plans to advance NGN-401 into its Embolden registrational trial for Rett syndrome. The company views ICV delivery as a strategic enabler for achieving robust brain targeting in its pivotal development program.