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

Key Points:

• Molecular biologist Jean Bennett and ophthalmologist Albert Maguire, along with physician Katherine High, received the $3 million Breakthrough Prize for life sciences for developing Luxturna, the first approved gene therapy for blindness caused by Leber congenital amaurosis (LCA).
• Luxturna, approved in 2017, has restored vision for patients with LCA by delivering a functional RPE65 gene to retinal cells, with clinical trials showing profound improvements, including patients seeing faces and fine details for the first time.
• Bennett and Maguire's 25-year project began at Harvard and continued at the University of Pennsylvania, where they also adopted two dogs they treated for blindness; their work was foundational despite early scientific challenges.
• Another life sciences Breakthrough Prize honored Swee Lay Thein and Stuart Orkin for developing a gene therapy targeting BCL11A to treat sickle cell disease and beta thalassaemia by reactivating fetal hemoglobin production, leading to the Casgevy therapy.
• Both Bennett and Orkin expressed concern over recent US governmental attacks on science funding and infrastructure, warning that politicization and reduced support could undermine biomedical research progress and innovation.