Clinical-stage biotechnology company developing genetic medicines for rare neurological diseases. Its EXACT platform uses microRNA-based circuits to regulate transgene expression and address overexpression toxicity. Its lead investigational AAV gene therapy, NGN-401 for Rett syndrome, is being evaluated in the Embolden registrational trial. It operates an in-house gene therapy manufacturing facility in Houston and became publicly traded on Nasdaq as NGNE following its December 2023 reverse merger with Neoleukin Therapeutics.
Founded 2018
Team
Andrew MulbergSenior Vice President, Regulatory Affairs, Quality Assurance, and Quality Control
Christine MikailPresident and Chief Financial Officer · Board Member