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UF Health gene therapy scientist named to Florida Inventors Hall of Fame

UF Health geneticist and pediatric cardiologist Barry Byrne, M.D., Ph.D., sits in the laboratory next to purification equipment used in research involving adeno-associated viruses. (Photo courtesy of Barry Byrne)

UF Health geneticist and pediatric cardiologist Barry Byrne, M.D., Ph.D., sits in the laboratory next to purification equipment used with research involving adeno-associated viruses. (Photo courtesy of Barry Byrne)

GAINESVILLE, Fla. — The kid was curious about how things worked. One day in his grandparents’ New Jersey home, the 8-year-old spied an old mantel clock that hadn’t worked in years, its hands frozen in time. The boy, unable to resist, looked at it and wondered if he could fix it.

He began taking it apart.

In a sense, University of Florida Health pediatric cardiologist and geneticist Barry Byrne, M.D., Ph.D., never stopped trying to repair that errant timepiece.

Byrne, director of the UF Powell Center for Rare Disease Research and Therapy and a gene therapy pioneer with about 23 patents in the field, will be inducted into the 2024 class of the Florida Inventors Hall of Fame in the fall, the organization announced May 30.

The group will recognize Byrne and eight other inductees at an Oct. 25 ceremony in Tampa.

Byrne rose from that curious childhood tinkerer to become one of the world’s leading gene therapy scientists. His research offers hope to patients and families who are all too used to hearing that a child’s genetic illness is incurable.

He’s contributed to numerous gene therapies focusing on inherited muscle diseases.

The field of muscle gene therapy didn’t exist before Byrne and his team discovered 27 years ago that a harmless adeno-associated virus, or AAV, could be used to introduce a functional copy of a defective gene to muscle cells to permanently produce therapeutic proteins. The discovery

reversed years of conventional thinking that had blocked the development of muscle gene therapy.

AAVs, which can be used with a variety of cell types, were even then an unusually fertile area of investigation at the university. UF researchers Nicholas Muzyczka, Ph.D., and Kenneth Berns, M.D., Ph.D., conducted groundbreaking research on AAVs in the 1980s.

Byrne, a professor and associate chair for research in the UF College of Medicine’s department of pediatrics and the Earl and Christy Powell University Chair in Gene Therapy and Genetics Research, thanked the Florida Inventors Hall of Fame for selecting him.

He said UF is a vibrant hub of gene therapy knowledge and the entrepreneurship so essential today in bringing drugs to market. That, he said, profoundly influenced his 30-plus-year career.

“We often have our heads down in the research, toiling in the details, so it’s nice to be recognized,” Byrne said. “At the same time, we never forget the urgency of patients and their families. It’s why we work tirelessly. We all share the same spirit of discovery at the Powell Center and the hope that our work can meaningfully change lives.”

Byrne made significant contributions to the understanding and treatment of Pompe disease, a neuromuscular disorder caused by a nonfunctioning gene that leads to a buildup of complex blood sugars. The disease can rapidly lead to the death of newborns. A long list of other muscle diseases is on the list of clinical programs being pursued at UF.

One ranking shows that Byrne was the world’s third most-cited AAV author and first among clinicians from 1991 to 2022. During that time frame, he published 62 studies and his research was cited 6,510 times.

The scientist has founded four companies to bring potentially lifesaving therapies to market faster. He also helped develop new methods for large-scale AAV clinical manufacturing.

“I have known Dr. Byrne for over 25 years and can attest to his extraordinary talent as a physician-scientist,” wrote Philip R. Johnson, M.D., CEO of Interius Biotherapeutics, in a nomination letter to the Florida Inventors Hall of Fame. “The singular driving force behind his work has always been and remains to this day: ‘How can I improve the lives of patients and families stricken with otherwise incurable diseases?’”

One great scientific discovery is invariably preceded by failed experiments that nonetheless provide a scientist with important insight. And so it went for the broken clock young Byrne, driven by a spirit of discovery, disassembled all those years ago.

“At that age, I didn’t appreciate how complex a clock was,” Byrne recalled, smiling at the audacity of the scientist-in-waiting. “It remained in a lot of pieces and never worked again. But I was willing to try to understand how something worked.”

About the author

Bill Levesque
Science Writer

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