Casgevy, a new CRISPR-based gene therapy, has received FDA approval for treating sickle cell disease in patients 12 and older experiencing recurrent crises. This groundbreaking treatment uses a patient's own stem cells, edited to produce fetal hemoglobin, thus preventing the sickling of red blood cells and associated pain. While requiring intensive chemotherapy beforehand, Casgevy offers a potential cure and improved quality of life compared to bone marrow transplants, the previous long-term treatment option. Current research is ongoing to assess the long-term safety and efficacy of Casgevy and to broaden access to this life-changing therapy for all those affected by sickle cell disease. The therapy's success represents significant progress in treating this debilitating inherited blood disorder.