The world's first treatment that uses CRISPR gene-editing technology has been approved.
Exa-cel, also known by its brand name Casgevy, received its first regulatory approval on Nov. 16, 2023 from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to treat two debilitating blood disorders: sickle cell disease and transfusion-dependent beta-thalassemia. Advisors to the U.S. Food and Drug Administration (FDA) determined that the drug was safe for clinical use in late October, and the FDA itself is expected to rule on whether to approve the treatment by December.
