Dr. Kathryn Wagner serves as the Clinical Lead for Cure Rare Disease, where she oversees clinical strategy and the translation of next-generation genetic therapies for ultra-rare neuromuscular diseases.
Dr. Wagner previously served as Global Head of Neuroscience, Translational Medicine at Novartis Institutes for Biomedical Research in Basel, Switzerland, and as Global Head of Neuromuscular Disorders at Roche Pharma, where she played a key role in advancing the approvals of risdiplam, the first oral therapy for spinal muscular atrophy, and delandistrogene moxeparvovec, the first gene therapy for Duchenne muscular dystrophy.
Before entering industry, she founded and directed the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and was Professor of Neurology and Neuroscience at the Johns Hopkins School of Medicine. Her pioneering research has advanced the multidisciplinary care of muscular dystrophy patients, established novel preclinical models including xenografts, and deepened understanding of myostatin’s role in muscle disease and repair.
