U.S. patients with sickle cell disease now have a novel treatment option: the first-ever CRISPR-based therapy.for use in patients age 12 years and older. In addition to offering hope of relief for people with severe forms of the painful blood disorder, the treatment, called Casgevy, is the world’s first to genetically tweak cells using theAnother gene therapy for sickle cell disease, called Lyfgenia and developed by biotech company bluebird bio, based in Somerville, Mass.
, was also approved December 8. Getting a green light for the first CRISPR-based medicine is exciting, says David Altshuler, chief scientific officer at Boston-based Vertex Pharmaceuticals, which developed the drug in a joint venture with CRISPR Therapeutics, a company in Cambridge, Mass. But the fact that the drug fills an unmet need for underserved patients is “more compelling to me, personally, than the fact that it’s CRISPR.” , most of them Black or Latino, have sickle cell disease. It is caused by a genetic defect in hemoglobin, the oxygen-carrying protein in red blood cell