Posted by Michael Wonder on 25 May 2019
RegenxBio announces first FDA approval of a gene therapy based on its proprietary NAV technology platform
24 May 2019 - FDA approval of Novartis' Zolgensma for the treatment of paediatric patients with SMA marks the first regulatory approval based on RegenxBio's NAV Technology.
RegenxBio today announced the first U.S. FDA approval of a gene therapy that leverages RegenxBio's proprietary NAV Technology Platform, Novartis AG's Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101). Zolgensma was approved by the FDA as a one-time infusion for paediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.
In 2009, RegenxBio secured exclusive rights to key intellectual property covering novel recombinant adeno-associated viral vectors discovered at the University of Pennsylvania in the lab of James Wilson, M.D., Ph.D. AveXis, a Novartis company, licensed RegenxBio's NAV adeno-associated virus 9 vector in 2014 and entered into an expanded license for rights to the entire NAV Technology Platform for the treatment of SMA in 2018.
