CRISPR-based sickle cell therapy earns $3M Breakthrough Prize for Thein and Orkin
Two researchers, Swee Lay Thein and Stuart Orkin, won the Breakthrough Prize in Life Sciences worth $3 million for work that led to Casgevy, a CRISPR-based therapy that functionally cures sickle cell disease and beta-thalassemia by disabling the BCL11A repressor to boost fetal hemoglobin. The treatment—developed with Vertex—involves editing a patient’s bone marrow cells and reinserting them, a costly, lengthy process with chemotherapy, and while a major milestone, access remains limited as scientists pursue in vivo approaches to broaden use.