UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia

By MARIA CHENG
AP Medical Writer

LONDON (AP) — Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K. In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020. The medicine was approved for patients with sickle cell and thalassemia. Both diseases are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.