‘Oscar of science’ awarded to team behind gene therapy that restores lost vision

A team of scientists behind the development of Luxturna, the first approved gene therapy for blindness, has been awarded the $3 million Breakthrough Prize in Life Sciences. The therapy, created by molecular biologist Jean Bennett, ophthalmologist Albert Maguire, and physician Katherine High, has transformed the lives of patients born with Leber congenital amaurosis (LCA), a genetic disorder that typically causes total blindness. Luxturna received US approval in 2017 and has enabled patients to experience sight for the first time, with one clinical trial participant describing the profound moment of seeing their child’s face and everyday details like wood grain and tree branches. Bennett and Maguire, a married couple who met at Harvard Medical School, spent 25 years developing the treatment, overcoming significant scientific challenges linked to the faulty RPE65 gene responsible for LCA. Their work included adopting dogs they treated for blindness, highlighting the therapy’s broader potential. The Breakthrough Prize ceremony in Los Angeles also recognized advances in gene therapies for sickle cell anemia and beta thalassemia, as well as discoveries related to frontotemporal dementia and ALS, the disease that afflicted physicist Stephen Hawking. Bennett, now retired from the University of Pennsylvania, described the moment of success as a “miraculous eureka moment” and emphasized the excitement surrounding scientific progress. However, she expressed concern about the current political climate in the United States, warning that attacks on science and the undermining of research institutions could lead to a damaging brain drain and hamper future innovation. The award highlights the critical role of sustained scientific research and collaboration in developing groundbreaking medical treatments that can dramatically improve patient outcomes.