Gene Therapy For Sickle Cell Clears Hurdle. (cover story)

The article reports on the announcement by a panel of experts on October 31, 2023 that exa-cel, a groundbreaking treatment for sickle cell disease was safe enough for clinical use setting the stage for likely U.S. Food and Drug Administration (FDA) approval by December 8, 2023. Topics include the cost of lifelong care for patients with the disease, the novel approaches tried by biotechnology companies to treat sickle cell, and the hardships that patients need to endure to get the treatment.