NGN-401

NGN-401 is an investigational gene therapy developed by Neurogene for the treatment of Rett syndrome. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a full-length human MECP2 gene, which is under the control of Neurogene’s proprietary EXACT transgene regulation technology. This technology enables targeted expression levels of MECP2, aiming to avoid the toxicity associated with overexpression that has limited previous gene therapies for Rett syndrome. NGN-401 is administered as a one-time intracerebroventricular infusion and is currently being evaluated in a Phase 1/2 clinical trial in girls aged 4–10 years with classic Rett syndrome. The therapy has shown promising safety and efficacy results at low doses, including improvements in developmental milestones and core symptoms of Rett syndrome, while high-dose administration led to serious immune-related adverse events[1][3][4][5][6][7][8][9][10].