Both the U.K. MHRA and the U.S. FDA approved their first CRISPR-based gene therapy in 2023. Crispr Therapeutics AG and partner Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) was approved by the MHRA in November and the FDA on Dec. 8. The U.K. approval is for both severe sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT). In the U.S., the approval is for severe SCD, with a PDUFA date for TDT coming up in spring 2024.