FDA Approves First Gene-Editing Treatment

On December 8, 2023, the US Food and Drug Administration approved Casgevy (exagamglogene autotemcel, exa-cel) for sickle cell disease, the first CRISPR-based gene-editing therapy approved for clinical use anywhere in the world. The therapy edits a patient's own bone marrow stem cells using CRISPR-Cas9 to reactivate fetal hemoglobin production, suppressing the sickle-shaped red blood cells that cause the disease. Casgevy was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The same day, the FDA approved a second sickle cell gene therapy, Lyfgenia (lovotibeglogene autotemcel), which uses lentiviral gene addition rather than gene editing.