Casgevy was the first CRISPR-based medicine approved by the U.S. Food and Drug Administration.
The FDA approved Casgevy, also called exagamglogene autotemcel, in December 2023 for treating sickle cell disease in patients aged 12 and older with recurrent vaso-occlusive crises. It uses CRISPR-Cas9 gene editing on a patient’s blood stem cells outside the body.
The edited cells are returned after conditioning treatment, helping the body produce more fetal hemoglobin and reducing the tendency of red blood cells to sickle. The therapy is individualized because each dose is made from the patient’s own cells.
Casgevy is not a conventional vaccine and is not the same as CAR-T therapy. Kymriah was the first FDA-approved CAR-T treatment, while Luxturna was the first FDA-approved in vivo gene therapy for an inherited disease.