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Neurogene Highlights Data at ASGCT Meeting Supporting Therapeutic Rationale for ICV Delivery Method in CNS-targeted Gene Therapy
NEW YORK, May 12, 2026--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, highlighted data at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting supporting the intracerebroventricular (ICV) route of administration as a routine approach for delivering medicines, including gene therapies, to treat central nervous system (CNS) disorders. The presentation further
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[{"type":"text","content":"ICV is a commonly used neurosurgical procedure performed across multiple therapeutic settings, including gene therapy","length":117,"tagName":"p","attribs":{}},{"type":"text","content":"Presentation adds to body of evidence that ICV administration results in broad biodistribution of gene therapy to the brain and nervous system","length":142,"tagName":"p","attribs":{}},{"type":"text","content":"Use of ICV administration in NGN-401 gene therapy program for Rett syndrome provides potential rationale for the multidomain and durable improvements observed in Phase 1/2 trial","length":177,"tagName":"p","attribs":{}},{"type":"text","content":"NEW YORK, May 12, 2026--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, highlighted data at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting supporting the intracerebroventricular (ICV) route of administration as a routine approach for delivering medicines, including gene therapies, to treat central nervous system (CNS) disorders. The presentation further highlights that the ICV procedure is routine, and administration takes approximately 10 minutes; therefore, the clinical safety and monitoring in gene therapy programs is driven primarily by factors related to transgene expression, dose and total viral load rather than the delivery procedure itself.","length":817,"tagName":"p"},{"type":"text","content":""AAV-based gene therapies can only be administered once, making delivery to the affected cells one of the most critical components in designing a potential treatment," said Daniel J. Curry, M.D., Director of Functional Neurosurgery and Epilepsy Surgery at Texas Children's Hospital, Professor of Neurological Surgery at Baylor College of Medicine, and neurosurgeon in the NGN-401 clinical program. "With broader brain biodistribution compared to other delivery routes and a well‑established, low‑complexity neurosurgical profile, ICV administration is the most compelling delivery route to date for CNS gene therapies such as those for Rett syndrome. The clinical safety and efficacy data observed in the ICV-delivered NGN-401 gene therapy program for Rett syndrome reaffirm the potential for this route of administration to target the...