Neurogene Reports Positive Interim Phase 1/2 Data for NGN-401 Gene Therapy in Rett Syndrome

Neurogene Inc.

All 8 pediatric participants (ages 4-10) showed functional gains across spectrum of disease severity.
35 developmental milestones/skills acquired across hand function, language/communication, and gross motor domains.
NGN-401 at the 1E15 vg dose was generally well-tolerated with no evidence of HLH in any participant.
Gains were durable with continued skill acquisition observed up to 24 months post treatment.
Company to discuss results at Stifel 2025 Healthcare Conference webcast on November 12, 2025.

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