U.S. patients with sickle cell disease now have a new treatment option: the first-ever CRISPR-based therapy.
Another gene therapy for sickle cell disease, called Lyfgenia and developed by biotech company bluebird bio, based in Somerville, Mass., was also approved on December 8.
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., finds it exciting to get approval for the first CRISPR-based medicine. However, he personally finds it more compelling that the drug addresses the unmet needs of underserved patients, rather than the fact that it utilizes CRISPR technology.
There are approximately 100,000 people in the United States, mostly Black or Latino, who have sickle cell disease. This disease is caused by a genetic defect in hemoglobin, the protein in red blood cells that carries oxygen. Unlike normal blood cells that can easily pass through blood vessels, sickled blood cells are rigid and can get stuck, leading to restricted blood flow and severe pain (SN: 2/1/22). Individuals with severe forms of the disease often require multiple hospitalizations throughout the year.
2023-12-08 12:46:08
Article from www.sciencenews.org
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