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Breakthrough Prize 2026

A gene therapy that helps children see

Jean Bennett, Katherine A. High and Albert Maguire share a 2026 Breakthrough Prize in Life Sciences for the first FDA-approved gene replacement therapy. It has helped children who were going blind to gain independence, go to regular schools and even play outside at night.

· USA

Research areas: Gene Therapy Ophthalmology Genetics

Layers of nerve cells in the retina in bright green, red, blue and purple, seen under a microscope
Layers of nerve cells in the retina, the part of the eye that responds to light. Not from the prize-winning work. Photo: National Institutes of Health, public domain, via Wikimedia Commons

On 18 April 2026, the Breakthrough Prize Foundation announced that Jean Bennett, Katherine A. High and Albert Maguire share one of the three 2026 Breakthrough Prizes in Life Sciences. Each prize is worth US$3 million.

Their therapy treats Leber congenital amaurosis, a rare inherited eye disease that usually leads to total blindness in early adulthood. It replaces a faulty gene called RPE65, which makes a protein the retina needs to respond to light.

Husband-and-wife team Jean Bennett, a molecular biologist, and Albert Maguire, an eye surgeon, invented the therapy and first made it work in animals, including restoring sight to a number of Swedish Briard dogs, which they went on to adopt! In 2005, Katherine A. High, a physician-scientist at Children’s Hospital of Philadelphia, invited them to work with her on a trial in people.

Why it matters

Patients treated more than ten years ago have kept their improved vision.

It also showed that gene therapy can work safely and effectively, which opened the door to gene therapies for a range of genetic diseases. Since then, hundreds of trials have followed, including more than 100 for gene therapies in the retina.

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