Neurogene Inc. reported positive Interim Data in Pediatric Cohort from NGN-401 Gene Therapy Trial for Rett Syndrome Multidomain, durable gains with continued skill acquisition over time. All 8 participants showed functional gains across spectrum of disease severity. 35 total developmental milestones/skills acquired across 8 participants.
NGN-401 at the registrational dose continues to be generally well-tolerated, with no evidence of HLH. Neurogene management to discuss results during Stifel 2025 Healthcare Conference webcast at 4:40 p.m. ET. Moreover, company is the only company evaluating a gene therapy in children with Rett syndrome as young as three years old in a single trial, which provides an efficient path to market and first-in-class potential.
In gene therapy for Rett syndrome, caregivers value improvements across multiple domains - hand function, gross motor skills and communication - especially when new abilities are gained and sustained over time. The interim NGN-401 results are particularly encouraging, showing continued skill acquisition across these areas with durable gains observed up to 24 months post treatment," said Bernhard Suter, M.D., Medical Director of the Blue Bird Circle Rett Center at Texas Children's Hospital, Associate Professor of Pediatrics and Neurology at Baylor College of Medicine, and principal investigator in the NGN-401 Phase 1/2 clinical trial. These gains also translate into meaningful improvements in daily function, reduced caregiver burden and greater.
ability to express needs and wants, while supporting more complex tasks that foster greater independence. Safety Data (N=10; as of data cutoff date of October 30, 2025); NGN-401 at the 1E15 vg dose has been generally well-tolerated with a favorable safety profile across the pediatric cohort and the adolescent/adult cohort (ages <11 cohort); All treatment-related adverse events have been mild (Grade 1) or moderate (Grade 2) in severity and the majority are known potential risks of AAV and have resolved or are resolving. 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).
