Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
November 11, 2024 PDF Version All participants experienced a 2-point improvement in the clinician-rated Clinical Global Impression-Improvement (CGI-I) scale from baseline
All participants improved in the caregiver-completed Rett Syndrome Behavior Questionnaire (RSBQ), ranging from 28 to 52 percent improvement from baseline
All participants with disruptions in sleep, constipation, and dysphagia at baseline demonstrated objective improvements
Gains in skill and developmental milestones were consistent, durable, deepened over time and demonstrated improvements not expected based on natural history data
Low-dose NGN-401 well-tolerated with favorable safety profile
Company plans to provide an update of registrational trial design in the first half of 2025
Company to host investor/analyst webcast today, November 11, 2024, at 4:30 p.m. ET
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