A Gene Therapy That Can Reverse Blindness Just Won a $3 Million Prize

The breakthrough treatment, developed over 25 years, has already helped restore vision for some patients.

BY LEILA SHERIDAN, NEWS WRITER

Dr. Albert Maguire and Dr. Jean Bennett. Photo: Getty Images

A gene therapy that can restore sight to people born with degenerative blindness has earned its creators one of science’s most prestigious prizes, often described by its Silicon Valley founders as the “Oscars of science.”

Molecular biologist Jean Bennett and ophthalmologist Albert Maguire were awarded the $3 million Breakthrough Prize in Life Sciences alongside physician Katherine High for developing a treatment that can reverse a rare form of inherited blindness. The award recognizes 25 years of work that ultimately led to a therapy now restoring vision in patients who once had none, The Guardian reported. 

The couple’s work focused on Leber congenital amaurosis (LCA), a condition caused in some cases by mutations in the RPE65 gene that typically leads to total blindness by early adulthood. When Bennett began studying the disease, the idea of fixing a faulty gene inside the human eye was still largely theoretical.

“The nice thing about being young and naive is I didn’t know what I didn’t know,” Bennett said, according to The Guardian.

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Working at the University of Pennsylvania, Bennett and Maguire spent years developing a way to deliver a healthy copy of the gene directly into retinal cells. Along the way, they tested the therapy in animals, including two dogs, Venus and Mercury, whose sight was restored and who later became the couple’s pets.

The breakthrough became most apparent in human trials, when patients confirmed what earlier experiments had suggested: the therapy could restore vision.

Patients described seeing things they had never experienced before, details like the grain in wood, branches moving in the wind, even their child’s face for the first time. A pivotal study published in The Lancet found that those treated showed significant improvement in functional vision compared to those who did not receive the therapy.

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