Neurogene Reports Positive Long-term Clinical Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome

NEW YORK--(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 par