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FDA Approves First CRISPR Gene Editing Treatment for Sickle Cell Disease | Scientific American

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Most people with sickle cell disease who received a new gene editing treatment saw their pain resolve for at least one year, but longer follow up is needed BY SARA REARDON Sickle cell disease causes red blood cells to take on a sickle-like shape, making them fragile and less able to transport oxygen. BSIP SA/Alamy Stock Photo CRISPR, the gene-editing technology that has revolutionized biological research, is finally available as a medical treatment with regulatory approval. On December 8 the U.S. Food and Drug Administration approved the first CRISPR treatment for sickle cell disease. The treatment, called exa-cel and made by the companies Vertex and CRISPR Therapeutics, edits a gene involved in red blood cell shape and function. It appears to functionally cure the disease for at least one year. The FDA’s decision makes the U.S. the second country to approve a CRISPR therapy, following exa-cel’s approval for sickle cell disease in the U.K. in November. Scientifically speaking, exa-cel

December 8, 2023 5 min read Add Us On Google Add SciAm FDA Approves First CRISPR Gene Editing Treatment for Sickle Cell Disease Most people with sickle cell disease who received a new gene editing treatment saw their pain resolve for at least one year, but longer follow up is needed By Sara Reardon edited by Tanya Lewis Sickle cell disease causes red blood cells to take on a sickle-like shape, making them fragile and less able to transport oxygen. BSIP SA/Alamy Stock Photo CRISPR, the gene-editing technology that has revolutionized biological research, is finally available as a medical treatme

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