"Breakthrough Gene-Editing Treatment Offers Hope for Sickle Cell Disease Patients"

The U.S. Food and Drug Administration has approved the first gene-editing treatment, Casgevy, for sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy uses CRISPR technology to edit a person's genes and turn on fetal hemoglobin, helping red blood cells maintain their healthy shape. Clinical trials have shown that Casgevy eliminates pain crises in most patients. However, challenges remain in reaching the thousands of people who could benefit from the treatment, including potential cost barriers and the complexity of administering the therapy. The FDA has also approved a separate gene therapy by Bluebird Bio, called Lyfgenia, for the treatment of sickle cell disease.