NEW YORK, NY — Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that it will present data from its NGN-401 gene therapy program for Rett syndrome at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress, taking place October 27-30, 2026, in Hamburg, Germany.
New preclinical data to be featured are aimed at addressing two important questions in AAV gene therapy for Rett syndrome: how rapidly single-stranded AAV delivery translates into meaningful transgene expression and how different miRNA-based approaches regulate MECP2 transgene expression on a cell-by-cell basis. The presentations will include preclinical data characterizing expression kinetics following single-stranded AAV delivery alongside observations of early time-to-response in the NGN-401 Phase 1/2 trial. In addition, results from an in vivo evaluation of miRNA-based transgene regulation approaches, including the Company’s EXACT™ transgene regulation technology, in a preclinical model of Rett syndrome will be shared. Additional presentations will focus on intracerebroventricular (ICV) administration as a well-established, routinely performed neurosurgical procedure and a deliberate delivery strategy designed to maximize efficacy for central nervous system gene therapies, as well as an overview of the EXACT platform.
The details of Neurogene’s presentations are as follows:
Wednesday, October 28
Invited Talk during CNS & Sensory I Session: 11:00 – 11:30 a.m. CET
- Self-regulating gene therapy for Rett syndrome
Poster Presentations: 1:30 – 3:30 p.m. CET
- Myth versus measurement: direct CNS comparison of two miRNA based regulated gene therapies in a mosaic model of Rett syndrome
- Gene Therapy Targeting CNS Diseases: ICV Administration as a Growing Standard for Delivery
Thursday, October 29
Poster Presentation: 2:00 – 4:00 p.m. CET
- Expression Kinetics and Clinical Onset of Benefit Following Single-Stranded AAV Delivery of NGN-401 in Rett Syndrome
About NGN-401
NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Data from the Phase 1/2 trial (as of June 16, 2026) have shown that participants experienced multidomain, durable gains with continued developmental milestone acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency.
About Neurogene
Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on X.
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