Breakthrough prizes in gene therapy

Gene therapy works by changing a patient’s own cells or adding a working gene, and the 2026 Breakthrough Prize put two of those approaches in the spotlight on April 18. (breakthroughprize.org) The Breakthrough Prize Foundation gave one $3 million Life Sciences award to Jean Bennett, Katherine High, and Albert Maguire for work that led to Luxturna, the first United States gene therapy approved for an inherited disease. (breakthroughprize.org) (fda.gov) It gave a second $3 million Life Sciences award to Stuart Orkin and Swee Lay Thein for research on fetal hemoglobin control that led to Casgevy for sickle cell disease and beta thalassemia. (breakthroughprize.org) (hhmi.org) In inherited blindness, the basic idea is replacement: deliver a healthy copy of the RPE65 gene into retinal cells so the eye can process light signals again. Luxturna won Food and Drug Administration approval on December 19, 2017 for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy who still have viable retinal cells. (fda.gov) (aao.org) In sickle cell disease, the strategy is switching genes back on rather than replacing one. Orkin’s lab identified BCL11A as a brake on fetal hemoglobin in 2008, and turning that brake down lets patients make the fetal form of hemoglobin that does not sickle. (dana-farber.org) (childrenshospital.org) Casgevy became the first Food and Drug Administration-approved CRISPR treatment on December 8, 2023 for sickle cell disease in patients 12 and older with recurrent vaso-occlusive crises, and the agency expanded approval to transfusion-dependent beta thalassemia on January 16, 2024. (fda.gov) (drugs.com) The blood-disease prize tracks a shift in gene medicine from theory to approved care. The European Commission cleared Casgevy in February 2024, and Vertex and CRISPR Therapeutics said in February 2026 that they expected Casgevy revenue to nearly triple this year as treatment access expands. (news.vrtx.com) (biospace.com) The vision prize reflects an older arc in the field. Penn and Children’s Hospital of Philadelphia said this week that the Luxturna program helped establish a regulatory and clinical template for more than 140 retinal gene therapy trials, with about 80 more underway. (biospace.com) The prizes arrived as genetic medicines show both progress and strain. Alliance for Regenerative Medicine figures cited by Inside Precision Medicine put cell and gene therapy investment at about $15.2 billion in 2024, while 2025 funding through the first three quarters was about $7.9 billion. (insideprecisionmedicine.com) The through line is that both winning teams found a way to make genes behave differently in the right cells. One therapy adds a working instruction to the retina, and the other lifts BCL11A’s block so blood cells can make fetal hemoglobin again. (fda.gov) (hhmi.org)