Letter to the Rett Syndrome Community: Neurogene’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology
Dear Rett Syndrome Community, Earlier this year Neurogene shared that the FDA cleared our Investigational New Drug (IND) application for NGN-401 gene therapy for the treatment of female children with Rett syndrome. NGN-401 is an investigational adenoassociated virus (AAV) gene therapy, using Neurogene’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology. NGN-401 contains a full-length humanized MECP2 gene which is designed to express therapeutic levels of the MECP2 protein while avoiding overexpression. Since that announcement, we have received requests from the community, seeking additional information about the clinical trial. The purpose of this letter is to provide additional details. Following, are frequently asked questions we’ve received:
