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 provided an update on recent achievements for NGN-401 gene therapy for the treatment of Rett syndrome and plans for multiple value-creating catalysts anticipated in 2026.
“We are pleased to share that multiple participants have been dosed in our Embolden registrational trial of NGN-401 gene therapy for Rett syndrome, an important step toward completing dosing in the second quarter,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “We continue to track the progress of the participants in our Phase 1/2 trial, with plans to report updated interim safety and efficacy data by mid-year, including at least 12 months of data for all participants. As we rapidly advance NGN-401 towards commercialization, we are actively engaging with payors and other key stakeholders while leveraging our internal CMC capabilities for commercial-grade production to ensure future launch readiness. With cash runway expected through the first quarter of 2028, we believe we are well-positioned to advance NGN-401 through key upcoming milestones, including a BLA submission, to achieve its potential as a best-in-class gene therapy for Rett syndrome.”
NGN-401 Program Updates and Recent Accomplishments
- Dosed multiple participants in the Embolden™ registrational trial in the fourth quarter of 2025
- Initiated 13 U.S. clinical trial sites conducting Embolden
- Reported positive interim data from the Phase 1/2 trial evaluating NGN-401 in females with Rett syndrome; data showed durable multidomain improvements across a full spectrum of disease severity
- Completed rigorous market payor research confirming strong reimbursement potential of NGN-401
Key Anticipated Milestones in 2026
- Complete dosing participants in the Embolden registrational trial in the second quarter of 2026
- Present interim safety and efficacy data on the pediatric cohort (ages 4-10; n=8) and the adolescent/adult cohort (ages ≥ 11; n=2) from the Phase 1/2 trial in mid-2026, including at least 12 months of follow-up for all participants
- Initiate additional early commercial-readiness activities
Company management will participate in the 44th Annual J.P. Morgan Healthcare Conference with a presentation on Wednesday, January 14 at 7:30 a.m. PT. A webcast of the presentation will be accessible from the Investor Relations section of Neurogene’s website under events, where a replay of the event will also be available for a limited time.
About Neurogene
The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com.
About NGN-401
NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT™ transgene regulation technology. EXACT technology is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy.
NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA).
Company Contact
Mike Devine
Executive Director, Corporate Communications
[email protected]
Investor Contact
Melissa Forst
Argot Partners
[email protected]