FDA Approves New CRISPR Gene-Editing Treatment

ToplineThe Food and Drug Administration approved the use of Casgevy, a therapy from Vertex Pharmaceuticals and CRISPR Therapeutics that uses CRISPR gene-editing to treat the serious blood disorder transfusion-dependent beta thalassemia—marking the second major U.S. regulatory approval for the emerging gene-editing technology.FILE - This microscope photo provided on Oct. 25, 2023, by the Centers for Disease Control and ... [+] Prevention shows crescent-shaped red blood cells from a sickle cell disease patien ...