What CRISPR gene-editing therapies have been approved for human use?

The answer

In November 2023, the UK's MHRA became the first regulatory body to approve Casgevy (exagamglogene autotemcel), a CRISPR-Cas9 gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics for sickle cell disease and transfusion-dependent beta-thalassemia. The US FDA followed in December 2023. The treatment works by editing patients' own bone marrow stem cells to reactivate fetal hemoglobin production, compensating for defective adult hemoglobin. In clinical trials, 97% of sickle cell patients were free of severe pain crises for at least 12 months. The one-time treatment costs approximately $2.2 million per patient. Beyond blood disorders, CRISPR trials are underway for hereditary blindness (Editas Medicine), high cholesterol (Verve Therapeutics), and certain cancers using edited CAR-T cells.