Penn Medicine, CHOP team awarded Breakthrough Prize for developing gene therapy for inherited blindness
Jean Bennett, Albert Maguire, and Katherine High have been honored for their trailblazing work on the first FDA-approved gene therapy for an inherited condition.
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Physician-scientists Jean Bennett and Al Maguire (right and left, respectively, pictured in their home), and Katherine High received the 2026 Breakthrough Prize in Life Sciences for their trailblazing work on 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.
(Image: Peggy Peterson)
Their discovery started with a group of blind dogs living at a vet school. Now, the work has been awarded the prestigious Breakthrough Prize at the “Oscars of Science.”
Katherine High is a recipient of the 2026 Breakthrough Prize in Life Sciences.
(Image: Courtesy of CHOP)
Their work blazed a trail for the 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. Eighty more trials are currently underway.
“Even 20 years ago, treating people with gene therapy was seen by some as an impossibility,” says Jonathan Epstein, dean of the Perelman School of Medicine and executive vice president of the University of Pennsylvania for the Health System. “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. Their belief in the power of life-changing science has led to breathtaking results and richly deserved global recognition.”
The Breakthrough Prizes are called the “Oscars of Science” for their high-profile celebration of research and support from celebrities spanning numerous areas of pop culture. Created in 2012 by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Yuri and Julia Milner, and Anne Wojcicki, the prizes are given out in five categories including Life Sciences, Fundamental Physics, and Math, each with an accompanying $3 million award.
Mathew Madhavacheril, an assistant professor of physics and astronomy in SAS, also received recognition at this year’s Breakthrough Prize ceremony when he was honored with the New Horizons in Physics award, given to researchers early in their careers.
“Science is rarely a straight path, and those who make the most profound discoveries are resilient and persistent, overcoming obstacles along the way,” says Penn President J. Larry Jameson. “That is exactly what I see in this year’s awardees, and it has been true of all our remarkable faculty who have been recognized for scientific breakthroughs. Whether they are discovering what lies beneath Alzheimer’s Disease, curing cancer by engineering a patients’ own immune cells, or reversing blindness—they have persisted with imagination and rigor. Their steadfastness has pushed the boundaries of what medicine can achieve.”
“Developing cell and gene therapies has long been a top priority for our organization,” says Madeline Bell, CHOP’s CEO. “This breakthrough is the result of decades of investment and collaboration, and reflects our commitment to translating scientific discoveries into therapies that will transform patients’ lives. It has paved the way for many more cell and gene therapy innovations and has given hope to families around the world.”
Bennett and Maguire met and married during medical school in the 1980s. It was then that they both became intrigued by the concept of genetic therapy, the practice of replacing a mutated or faulty gene with a functional copy, and started dreaming of treating inherited forms of blindness with the technique, which at that time remained the stuff of science fiction.
It was “like thinking you wanted to go to the moon in 1950,” Maguire said many years later.
Together, Bennett and Maguire joined Penn’s Scheie Eye Institute in the 1990s and began working on their ideas with lab mice. They learned that Gustavo Aguirre, at the University of Pennsylvania School of Veterinary Medicine had identified a spontaneous canine model of blindness analogous to LCA that would prove to be invaluable to their research.
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