Can Neurogene’s NGN-401 restart developmental progress in patients with Rett syndrome?
Long-term Phase 1/2 data suggest that Neurogene’s NGN-401 gene therapy may produce continuing developmental gains across several functional domains in Rett syndrome. The small, uncontrolled dataset remains preliminary, however, leaving the larger Embolden registrational trial to determine whether those improvements are reproducible, clinically persuasive and sufficient for regulatory approval.