Jean Bennett and Albert Maguire, both emeritus professors of ophthalmology in the Perelman School of Medicine, and Katherine High, an emeritus professor of pediatrics, have received this year’s Breakthrough Prize in Life Sciences for their work in developing the first FDA-approved gene therapy for an inherited condition, which dramatically improves sight in people with a form of blindness called Leber Congenital Amaurosis (LCA).
Their work has led to more than 140 gene therapy trials for retinal conditions, including macular degeneration and diabetic retinopathy, diseases that collectively impact about 30 million people in the US.
Bennett and Maguire, who met and married during medical school in the 1980s, joined Penn’s Scheie Eye Institute in the 1990s and began working on treating blindness with genetic therapy, first on mice and then on blind dogs living in Penn’s School of Veterinary Medicine. High founded the Raymond G. Perelman Center for Molecular Therapeutics at the Children’s Hospital of Philadelphia in 2004 and the next year began successfully collaborating with Bennett and Maguire on clinical human trials [“Gazetteer,” Mar|Apr 2010]. By 2017 the therapy received FDA approval, and today hundreds of people around the world have successfully received the treatment.
“Even 20 years ago, treating people with gene therapy was seen by some as an impossibility,” Jonathan Epstein, dean of the Perelman School of Medicine and executive vice president of the University of Pennsylvania for the Health System, said in a statement. “But this group of incredible physician-scientists persisted and created something that is providing sight to people who would have been completely blind as early as kindergarten.”
Dubbed the “Oscars of Science,” the Breakthrough Prizes come with a $3 million award and have now been won by nine Penn-affiliated researchers (tied for the most with Harvard University), including most recently Carl June for the development of CAR T-cell immunotherapy [“Gazetteer,” Nov|Dec 2023].
