New data showing rapid and durable full-length MECP2 expression in vivo with single-stranded AAV support improvements observed in NGN-401 Phase 1/2 trial

New in vivo data demonstrate that miRNA-based auto-regulatory technology is not responsive to endogenous MeCP2 levels on a cell-by-cell basis

Preclinical data demonstrate broad CNS biodistribution following ICV administration, a routinely performed neurosurgical procedure, reinforcing NGN-401's route of delivery strategy

Neurogene Inc. (NASDAQ:NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, will present data related to its NGN-401 gene therapy program for Rett syndrome at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress, taking place October 27-30, 2026, in Hamburg, Germany. The new preclinical data are expected to further validate NGN-401’s differentiated design, which enables delivery of the fully functional, full-length MECP2 gene and leverages the EXACT™ transgene regulation technology for controlled expression on a cell-by-cell basis.

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