FDA Approves First Gene Therapies for Sickle Cell Anemia

Despite the breakthrough, concerns about high costs and medical mistrust persist, particularly within the Black community.

The FDA has approved the first gene therapies for sickle cell anemia, Casgevy and Lyfgenia, costing $2.2 million and $3.1 million respectively.
Casgevy, developed by Vertex Pharmaceuticals, is the first therapy to use the CRISPR gene-editing tool.
Lyfgenia, developed by Bluebird Bio, modifies a patient's blood stem cells and transplants them, adding normal hemoglobin to the cells to lower the risk of sickling.
Despite the potential of these therapies, there is concern about accessibility due to the high costs and the historic legacy of medical mistrust within the Black community, which is primarily affected by sickle cell disease in the U.S.
Gene therapy treatments also require long hospital stays, constant support from staff and doctors, and carry associated medical risks, including infertility, increased cancer risk, and treatment-related mortality.