Neurogene reports rapid, durable gene expression in preclinical study of Rett therapy NGN-401

• Neurogene will present new NGN-401 Rett syndrome data at the ESGCT congress in Hamburg, Germany, October 27-30, 2026.
• Preclinical findings showed rapid, durable expression of the full-length therapeutic gene, supporting early, sustained clinical improvements observed in the Phase 1/2 trial.
• Separate preclinical results showed miRNA-based regulation reduced gene expression regardless of cells' existing MeCP2 levels, challenging assumptions about selective regulation.
• Brain-directed delivery achieved broader central nervous system distribution than lumbar administration in preclinical testing; trial participants experienced no procedure-related adverse events.

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