Neurogene Reports Positive Interim Results for NGN-401 Gene Therapy in Pediatric Rett Syndrome Trial

Reuters11-13

Neurogene Reports Positive Interim Results for NGN-401 Gene Therapy in Pediatric Rett Syndrome Trial

Neurogene Inc. has announced updated interim clinical data from its ongoing Phase 1/2 trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome. The data, drawn from the pediatric cohort (ages 4-10), indicate that all eight participants experienced functional gains and acquired a total of 35 developmental milestones or skills across varying levels of disease severity. The reported improvements have shown durability, with continued skill acquisition observed over time and sustained gains up to 24 months post-treatment. NGN-401 at the registrational dose has been generally well-tolerated, with no evidence of HLH. Neurogene management will discuss these results and the Embolden™ registrational trial at the Stifel 2025 Healthcare Conference. The webcast presentation is scheduled for today at 4:40 p.m. ET, with a replay to be available on the company's website. Additional clinical data are expected to be reported in 2026.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Neurogene Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251112150450) on November 12, 2025, and is solely responsible for the information contained therein.

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The reported improvements have shown durability, with continued skill acquisition observed over time and sustained gains up to 24 months post-treatment. NGN-401 at the registrational dose has been generally well-tolerated, with no evidence of HLH. Neurogene management will discuss these results and the Embolden™ registrational trial at the Stifel 2025 Healthcare Conference. The webcast presentation is scheduled for today at 4:40 p.m. ET, with a replay to be available on the company's website. Additional clinical data are expected to be reported in 2026.\n      \n\nDisclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Neurogene Inc. published the original content used to generate this news brief via Business Wire (Ref. 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