Barry J. Byrne, M.D., Ph.D.

Professor and Associate Chair, Department of Pediatrics
Director, Powell Gene Therapy Center
University of Florida, College of Medicine

23 U.S. Patents

Barry Byrne, M.D., Ph.D. is a Professor and the Associate Chair of the Department of Pediatrics at the University of Florida College of Medicine. He also serves as the Director for the Powell Gene Therapy Center which merges cutting-edge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer.

A clinician scientist renowned for his pioneering work in rare diseases, Dr. Byrne’s research concentrates on conditions that lead to skeletal muscle weakness and cardiac and respiratory abnormalities, particularly focusing on developing therapies for inherited muscle diseases. As the result of this groundbreaking research, Dr. Byrne is credited with pioneering a new field of gene therapy. Dr. Byrne’s innovations have identified novel methods to deliver DNA to muscle cells using recombinant adeno-associated virus (AAV) vectors, including utilizing AAV gene therapies to cure rare genetic diseases with a focus on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function.

Under his leadership, Dr. Byrne’s research group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing, which has significantly advanced genetic therapies aimed at restoring cardiac and skeletal muscle function in diseases such as Duchenne muscular dystrophy, Friedreich’s ataxia, Pompe disease, and other inherited neuromuscular disorders.

In addition to his academic roles, Dr. Byrne founded four startup companies: AGTC, AavantiBio, Lacerta Therapeutics, and Ventura Life Sciences. Dr. Byrne holds a number of positions within organizations dedicated to combating muscular dystrophy, including Chief Medical Advisor of the Muscular Dystrophy Association; and he contributes his expertise as a member of the Therapeutics for Rare and Neglected Diseases (TRND) Advisory Committee (TACT) of the TREAT-NMD Alliance.

Dr. Byrne holds 23 U.S. Patents. He received the See the Light Award from the Mathew Forbes Romer Foundation. On two separate occasions, he received the Research Professor Award from the University of Florida. He also received the Faculty Research Prize in Clinical Research from the University of Florida. Dr. Byrne is a Fellow of the American Academy of Pediatrics and the American College of Cardiology and is a board-certified pediatrician and pediatric cardiologist. He also serves as an advisor for the FDA Advisory Committee on Cellular, Tissue, and Gene Therapy. Dr. Byrne received a B.S. from Denison University. He went on to complete his M.D. and Ph.D. in Microbiology and Immunology at the University of Illinois.