Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA) and continuing with our robust AAV-based pipeline, which includes treatments for Rett syndrome and Friedreich’s ataxia. We are powered by the world’s largest gene therapy manufacturing footprint, enabling us to bring gene therapy to patients around the world at quality and scale.