Penn professors awarded $3 million prize for gene replacement research

Three Penn researchers were awarded the 2026 Breakthrough Prize in Life Sciences last week for their role in developing a novel gene replacement therapy. 

The prize — dubbed the “Oscars of Science” — recognizes advances in scientific research with a $3 million award. Penn professors Jean Bennett, Albert Maguire, and Katherine High were honored for their contributions to the development of Luxturna, the first-ever gene therapy treatment for an inherited condition approved by the Food and Drug Administration. 

Bennett, F.M. Kirby professor of ophthalmology at the Perelman School of Medicine, told The Daily Pennsylvanian that “it was a total surprise to hear about it.”

Bennett and Maguire, who are married, first worked together as neuroanatomy lab partners during their time studying medicine at Harvard University. Their early discussions about treating genetic blindness through gene replacement went on to shape decades of research.

“At the time I realized, these are single-gene-based diseases, and now people are cloning genes, so you can actually get the physical piece of DNA,” Maguire explained. “I thought, ‘Why can’t you just put that back into the disease cell and fix the disease that way?’”

After arriving at Penn, the two focused on developing methods to deliver functional genes to retinal cells. Their work led to the identification of a delivery system capable of efficiently introducing genetic material into non-dividing cells, such as photoreceptors.

Experiments in the development process involved dogs with inherited blindness. In 2000, the team treated several puppies by injecting the therapy into one eye. Within weeks, the animals began responding to visual stimuli.

“Their whole behavior had changed,” Bennett said. “They were clearly visual.”

Human clinical trials began in 2005, led in part by High. Researchers observed significant vision improvements in the first patients treated in 2007. 

“It was clear from each one of those that they had improved,” Bennett explained.

Subsequent trials in children also produced notable results, with some patients regaining the ability to navigate independently and read classroom materials.

“The classic was, ‘What are those dots up in the sky?’” Maguire said, referring to children seeing stars for the first time. “They had never seen stars before, so they had never known those were stars.” 

Luxturna was approved by the FDA in 2017 and is now used to treat certain forms of inherited retinal disease. The therapy costs approximately $800,000, or $425,000 per eye.

“My hope is that as gene therapy products become more common and they’re more of them, there will be ways of streamlining the production so that the cost will come down,” Bennett said.

She emphasized that the award reflects the work of a broader research community.

“We are delighted by the Breakthrough award, but we accept it representing this brilliant team of people at Penn,” Bennett said. “Yeah, it’s really a Penn achievement.”

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