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2020 Award for Distinguished Research in the Biomedical Sciences

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Jean Bennett, MD, PhD
Jean Bennett, MD, PhD

Jean Bennett, MD, PhD

F.M. Kirby Professor of Ophthalmology
Perelman School of Medicine at the University of Pennsylvania

Albert M. Maguire, MD

Professor of Ophthalmology
Perelman School of Medicine at the University of Pennsylvania

Countless people around the world who were blinded by a once-untreatable disease can now see because of a pioneering gene therapy developed by Jean Bennett, MD, PhD, and Albert M. Maguire, MD.

Albert M. Maguire, MD
Albert M. Maguire, MD

By creating the first gene therapy to treat blindness, Drs. Bennett and Maguire not only reversed the effects of an inherited retinal degenerative disease, Leber congenital amaurosis (LCA), but ignited new research to combat other genetic causes of blindness as well.

“The work has changed medical practice,” says J. Larry Jameson, MD, PhD, dean of the Perelman School of Medicine (PSOM). “Instead of telling patients that there is ‘nothing we can do’ to treat inherited blindness, patients are now genotyped” to see if they can be treated with the therapy Drs. Bennett and Maguire developed or steered toward ongoing research into other blinding diseases caused by genetic mutations.

“Families and individuals born blind due to genetic disease now have hope that there will one day be a treatment for their condition,” Jameson says.

The therapy, approved by the Food and Drug Administration (FDA) in 2017 and named Luxturna, replaces a mutated gene, RPE65, that triggers LCA. The disease typically appears during infancy and gradually destroys photoreceptors, causing various visual impairments and often leading to blindness. The FDA called Luxturna “the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.”

Drs. Bennett and Maguire continue to lead the development of therapies for impaired vision, through their own research and by supporting the work and building the careers of other scientists. At PSOM, they established the Center for Advanced Retinal and Ocular Therapeutics to advance treatments for retinal and ocular diseases through research and training.

Drs. Maguire and Bennett earned their MDs at Harvard Medical School. Bennett earned her PhD in zoology and cell and developmental biology at the University of California at Berkeley.

The duo have won several awards together, including the Charles L. Schepens Award from the American Academy of Ophthalmology, the Retina Research Foundation Pyron Award for Outstanding Achievement in Retina Research, the Antonio Champalimaud Vision Award, the Smithsonian Ingenuity Award, and the Clinical Innovator Award from the National Medical Association.

Learn more about the Award for Distinguished Research in the Biomedical Sciences

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