The UK has authorized a gene-editing medicine for treating sickle cell disease and beta thalassemia, marking the first approval worldwide for a therapy based on CRISPR technology.
Casgevy is made by collecting a patient's stem cells from the bone marrow. In a lab, CRISPR is used to edit a gene in those cells to produce high levels of fetal hemoglobin. Before these modified stem cells are administered, a conditioning regimen is required to kill the remaining cells in a patient that are making faulty hemoglobin. Infused into the patient, the modified stem cells make their way to the bone marrow. Patients may need to remain hospitalized for at least a month while those cells take up residence in the bone marrow and start producing red blood cells that incorporate fetal hemoglobin.
Casgevy offers a potentially curative treatment option for both sickle cell disease and beta thalassemia. But the procedure requires a matched donor and it comes with the risk of rejection.
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