FDA approves two genetic treatments for sickle cell disease

The Food and Drug Administration on Friday approved the first medicine that uses the gene-editing tool CRISPR. In November, regulators in the United Kingdom approved the treatment called Casgevy, which edits a patient’s own stem cells to correct genetic errors that cause sickle cell disease before re-infusing them into the patient. Sickle cell disease is caused by a mutation in the gene responsible for producing hemoglobin, an oxygen-carrying protein in red blood cells. The condition affects more than 100,000 people in the United States, most of whom are black.

What is the other treatment? The FDA on Friday also approved the treatment called Lyfgenia, which employs an older gene therapy and also uses the patient’s cells. The treatment uses a virus to deliver a healthy hemoglobin-producing gene. The FDA approved both therapies for people age 12 and older. Both treatments are marketed as one-time treatments and are expected to cost several million dollars.

Dig deeper: From the WORLD archives, read Sophia Lee’s report on the ethics of gene editing.