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U.K. Is First to Approve a CRISPR-Based Therapy, Covering Two Blood Disorders - MedCity News

November 17, 2023
MedCity News
Summary
UK first to approve CRISPR-based therapy, Casgevy, for treating blood disorders sickle cell disease and beta thalassemia. Vertex Pharmaceuticals and CRISPR Therapeutics commercialize gene-edited, one-time treatment. MHRA approval given after trial results indicate reduced pain, transfusion needs; side effects comparable to similar procedures. Post-approval research required; authorization potentially renewed annually. Upcoming FDA decisions await for US approval.
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