Neurogene reports rapid, durable gene expression in preclinical study of Rett therapy NGN-401
NGNE•Neurogene reported preclinical findings showing rapid, durable expression of NGN-401, its Rett syndrome therapy, and broader central nervous system distribution with brain-directed delivery than with lumbar administration.
1. Preclinical findings
Preclinical findings showed rapid, durable expression of the full-length therapeutic gene, which Neurogene said supported early, sustained clinical improvements observed in its Phase 1/2 trial. Separate results showed miRNA-based regulation reduced gene expression regardless of cells’ existing MeCP2 levels. Brain-directed delivery achieved broader central nervous system distribution than lumbar administration, and trial participants experienced no procedure-related adverse events.
2. Congress presentation
Neurogene will present new NGN-401 data at the ESGCT congress in Hamburg, Germany, from October 27 to 30, 2026.
