Neurogene presents preclinical data on Rett syndrome therapy
NEW YORK - Neurogene Inc. presented preclinical data on its NGN-401 gene therapy for Rett syndrome at the European Society of Gene and Cell Therapy 33rd Annual Congress in Hamburg, Germany, according to a press release statement.
The company reported that its single-stranded AAV9 vector achieved expression of full-length MeCP2 protein in brain and nervous system tissues by Day 7 in preclinical studies, with expression remaining durable through three months. NGN-401 uses single-stranded AAV9 to accommodate the full-length MECP2 gene and regulatory elements.
The company stated that participants in its Phase 1/2 trial demonstrated clinical improvements at a median of two months post-treatment, with data through June 16, 2026 showing continued improvement through 30 months of follow-up.
A separate preclinical study evaluated miRNA-based transgene regulation technology in a mosaic model of Rett syndrome. The study found that both miRNA-based auto-regulatory technology and another technology produced comparable transgene expression in both MeCP2-null and MeCP2-expressing neurons, attenuating transgene output consistent with global transgene dampening.
Neurogene also presented data on intracerebroventricular administration, which the company said demonstrated broader central nervous system biodistribution compared with intrathecal lumbar administration in preclinical studies. In the Phase 1/2 trial, ICV administration showed no procedure-related adverse events among 10 participants who received the 1E15 vg dose of NGN-401.
NGN-401 is being evaluated in the Embolden registrational clinical trial. The therapy has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.
