The FDA is considering the approval of exa-cel, a new drug developed by Vertex Pharmaceuticals that could be the first cure available for many patients with sickle cell disease. The illness affects an estimated 100,000 people in the U.S., mostly Black, and causes anemia, pain, and organ damage. If approved, exa-cel would be the first drug to use the groundbreaking gene-editing tool CRISPR and provide a universally available, potentially curative option for individuals with sickle cell. The FDA is expected to make a decision on the drug in the coming months.
