Weill Cornell Department of Genetic Medicine initiates NIH-funded gene therapy clinical trial aimed at preserving heart health in individuals with Friedreich’s ataxia

February 2022 - The investigational gene therapy involves a single, intravenous administration of a modified adeno-associated virus that delivers the normal frataxin (FXN) gene to the heart (and other organs). This is the first clinical gene therapy to treat Friedreich’s ataxia, and the goal of this initial clinical study is to assess safety and initial estimates of efficacy in treating heart disease associated with Friedreich’s ataxia. This clinical trial is funded by a grant with Co-PIs from the Department of Genetic Medicine (Dr. Ronald G. Crystal and Dr. Stephen Kaminsky) and the Department of Cardiology (Dr. Johnathan W. Weinsaft).