Healthcare
Neurogene Reports Positive Long-term Clinical Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome
NEW YORK, June 29, 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, today announced updated, positive data from its Phase 1/2 clinical trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome.
About this update from Neurogene Inc.
47 total developmental milestones gained across 10 participants, with durable treatment effect and no plateau or milestone loss through 30 months of follow-up 100% of participants improved in CGI-I score and gained ≥1 developmental milestone, with an average of 4.7 milestones per participant, and all gaining ≥1 developmental milestone in the past 12 months Developmental milestones were gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment NGN-401 at the 1E15 vg dose remains generally well-tolerated (N=35) as of data cutoff date of June 16, 2026 Dosing complete in the EmboldenTM registrational trial with no treatment-related SAEs or DLTs as of data cutoff date of June 16, 2026, with topline data anticipated in 2H 2027 Company to host investor/analyst webcast today at 8:00 a.m. ET NEW YORK, June 29, 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, today announced updated, positive data from its Phase 1/2 clinical trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome. Across 10 participants, 47 total developmental milestones were gained (average of 4.7 per participant), with both pediatric and adolescent/adult participants demonstrating clinical response and continued, progressive improvements through 30 months of follow-up, with no plateau or loss of milestones observed. "We are encouraged by the long-term Phase 1/2 data across a broad age range and wide spectrum of disease severity, which we believe demonstrate that NGN-401 is driving gain of durable and clinically meaningful developmental milestones, and continued improvements were observed through 30 months of follow-up," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "Importantly, the gains of developmental milestones are not isolated events – they build over time with participants acquiring multiple milestones across the core Rett syndrome functional domains of hand function, gross motor function and communication. This multidomain progression is not observed in the natural history of Rett syndrome, and we believe supports NGN-401's differentiated profile, driven by its purposeful design and targeted delivery." "In typical development, children gain milestones...