Neurogene Inc. announced that the first participant was dosed in the Embolden?? registrational clinical trial of NGN-401, a gene therapy in development for the treatment of Rett syndrome. NGN-401 has the potential to be a best-in-class and first-in-class gene therapy for Rett syndrome, a neurodevelopmental disorder with significant unmet medical need.
Embolden is currently enrolling in the U.S., and the Company expects to complete enrollment in three to six months. EXACT technology is an important advancement in gene therapy for Rett syndrome., specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 gene expression without causing overexpression-related toxic effects associated with conventional gene therapy. NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA.
Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan drug designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA).
