Neurogene to present on ICV delivery in CNS-targeted gene therapy

Neurogene (NGNE) announced an upcoming poster presentation that will demystify the intracerebroventricular route of administration as a well-established, routinely performed neurosurgical procedure and a deliberate delivery strategy designed to maximize efficacy for central nervous system gene therapies at the American Society of Gene and Cell Therapy Annual Meeting, taking place May 11-15, 2026. The presentation will highlight the biological rationale to deliver gene therapy broadly to key regions of the brain, leading to the selection of ICV administration of NGN-401 gene therapy for Rett syndrome. Preclinical data will show that ICV administration resulted in greater biodistribution to brain regions central to Rett syndrome pathophysiology, compared to intrathecal-lumbar administration. The presentation will review that clinical safety in CNS gene therapy is shown to be driven by the product itself, and not the delivery route, with ICV administration showing a generally well-tolerated profile. “Our selection of ICV delivery for NGN-401 was informed by robust preclinical data that showed broader brain and nervous system biodistribution compared to IT-L, critical for a CNS-mediated disease such as Rett syndrome,” stated Rachel McMinn, CEO. “ICV administration is a well-established neurosurgical approach that has been used across multiple therapeutic areas and represents a deliberate choice to enable effective gene delivery. We believe the data we have reported from our ongoing Phase 1/2 trial reinforce our choice of ICV delivery, with participants demonstrating durable, multidomain developmental milestone gains over time and a safety profile at the 1E15 vg dose that supports continued advancement of NGN-401 in our Emboldenregistrational trial.”