Approved CRISPR Therapies
Casgevy (exagamglogene autotemcel) was the first approved CRISPR medicine: a patient's own hematopoietic stem cells are edited ex vivo to disrupt the BCL11A erythroid enhancer, de-repressing fetal hemoglobin, and reinfused after conditioning. It treats sickle cell disease and transfusion-dependent beta-thalassemia; in vivo LNP-delivered editors are the following wave.
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Casgevy (exagamglogene autotemcel) was the first approved CRISPR medicine: a patient's own hematopoietic stem cells are edited ex vivo to disrupt the BCL11A erythroid enhancer, de-repressing fetal hemoglobin, and reinfused after conditioning. It treats sickle cell disease and transfusion-dependent beta-thalassemia; in vivo LNP-delivered editors are the following wave.
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