A husband-and-wife team whose love story began in a Harvard anatomy lab have won one of science’s biggest awards for creating the first approved gene therapy to treat inherited blindness.
Molecular biologist Jean Bennett and ophthalmologist Albert Maguire share the $3 million Breakthrough Prize for life sciences with physician Katherine High. The trio spent 25 years developing Luxturna, a gene therapy that has transformed the lives of people born with Leber congenital amaurosis (LCA) – a genetic disorder that typically causes total blindness by early adulthood.
The therapy was approved in the U.S. in 2017, and during a clinical trial, one patient described seeing their child’s face for the first time, along with the fine grain in wooden furniture and branches waving in the wind. Other patients reported similar life-changing improvements.
“I was overwhelmed,” said Bennett, speaking of those initial tests, and who is now retired from the University of Pennsylvania. “It was one of the most miraculous eureka moments you can imagine.”
Bennett and Maguire first met at Harvard Medical School, where they were paired up to dissect a brain. They later moved to the University of Pennsylvania, where they turned their attention to LCA. Scientists had identified that the disease was linked to faults in a gene called RPE65, but the tools to fix it didn’t yet exist.
Bennett wasn’t discouraged, she said: “The nice thing about being young and naive is I didn’t know what I didn’t know.”
After years of painstaking work, the couple developed a gene therapy that delivered a working copy of the RPE65 gene into retinal cells. Animal testing and human trials – developed alongside Katherine High – confirmed it could restore lost vision. Along the way, Bennett and Maguire treated two blind dogs named Venus and Mercury, who recovered their sight and became the couple’s beloved pets.
The Breakthrough Prizes – often described as the Oscars of science – were handed out Saturday night at a ceremony in Los Angeles. The awards are among the most lucrative in science, funded by Silicon Valley backers.
