FDA Approves Groundbreaking CRISPR Treatment for Sickle Cell Disease

The FDA has approved Casgevy, a groundbreaking treatment for sickle cell disease that utilizes the gene-editing tool CRISPR. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is the first medicine in the US to use CRISPR and offers a potential cure for the devastating blood disorder. The therapy genetically modifies a patient's own stem cells to remove the gene responsible for sickle cell disease, eliminating the need for a donor. However, the treatment is extremely expensive, priced at $2.2 million per patient, which may limit accessibility for many families. Another gene therapy for sickle cell disease, called Lyfgenia, was also approved by the FDA.