UF college of medicine
Barry Byrne, M.D., Ph.D., will serve as ASGCT president in 2027.
By Manny Rea
Continuing UF College of Medicine’s revolutionary work developing the adeno-associated virus (AAV) vector, Dr. Barry Byrne, M.D., Ph.D., Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center, will serve as President of the American Society of Gene and Cell Therapy (ASGCT) during the society’s 30th anniversary year in 2027.
Byrne, a pediatric cardiologist and virologist, has advanced AAV vector development, helping translate gene therapy from concept to clinic — particularly for Pompe disease, Duchenne muscular dystrophy, Friedreich’s ataxia and inherited cardiomyopathies. His research has pioneered DNA delivery to muscle cells using AAVs and led to scalable clinical manufacturing methods for treatments restoring cardiac and skeletal muscle function. In Pompe disease specifically, Byrne’s team developed AAV therapies that deliver the missing gene or protein to correct the underlying muscle and neuronal dysfunction caused by abnormal glycogen accumulation.
Byrne directs the UF Health Center for Advanced Therapeutics and serves as the Chief Medical Advisor to the Muscular Dystrophy Association. Under Byrne’s directorship, UF’s Gene Powell Therapy Center became one of the first institutions in the U.S. to establish its own gene therapy vector manufacturing facilities. He has also published prolifically: of the more than 6,000 publications on AAV, Byrne is one of the three most cited authors. These achievements, among others, underpin his nomination as 2027 ASGCT president.
ASGCT is a non-profit medical organization dedicated to advancing gene, cell, and nucleic acid therapies. A member since its inception in 1996, Byrne has served on its board and chaired both the Cardiovascular and the Regulatory Affairs Committees.
“ASGCT is the premier place for scientists to exchange ideas, to network, and to understand where this complex field is going,” Byrne said. “Unlike small molecule drugs, gene therapies are harder to manufacture and administer. We’re still learning how to manage associated toxicities.”
As president, Byrne will bring a clinical approach to gene therapy research and the commercial use of AAV products.
“I think it helps to balance the goals of these treatments towards human health, as opposed to just disease mechanisms and more basic science questions,” Byrne said.
His leadership comes at a time of explosive growth for gene therapy. Since the FDA approved Luxturna in 2017 for an inherited retinal disease, investment and innovation have surged. Zolgensma, a $2.1 million treatment for spinal muscular atrophy approved in 2019, and other high-cost therapies have sparked research interest in treatments targeting specific organs.
This shift towards specific organs requires physicians trained in the genetics of their specialty, making the clinician-scientist model Byrne will champion in his presidency even more crucial.
“Anything I can do to build on my experience at the interface between academic and clinical practice and to help people navigate that interface in their own careers is a commitment I’m happy to make for the society and the field of gene therapy.”
At UF since 1997, a time when gene therapy’s potential was still emerging, Byrne’s appointment is a testament to the success of his 30-year career in a field now shaping modern medicine. Most recently, UF Health celebrated its 100th gene therapy infusion on May 2nd, enabled in part by his tireless efforts.
“But there’s always more work to do,” he said. “I’m excited to roll up my sleeves and get to it.”