Dr. Nigel Key co-authored a manuscript describing the pivotal multi-center Phase 3 clinical trial of adeno-associated virus 5 (AAV5)- based gene therapy for severe hemophilia A that was published in the New England Journal of Medicine on March 17, 2022. The trial sponsor was Biomarin Pharmaceutical. Of 134 participants with severe hemophilia who were enrolled, 2 were recruited at UNC. The encouraging results observed are expected to be included in the portfolio to be submitted for FDA licensure by Biomarin in the near future.
Click here to read New England Journal of Medicine manuscript.