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Pharmaceuticals

Casgevy becomes first CRISPR drug to get approval

by Laura Howes
November 23, 2023 | A version of this story appeared in Volume 101, Issue 39

Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics’ drug to treat sickle cell disease and β-thalassemia, has become the first CRISPR-based medicine in the world to be approved. The UK Medicines and Healthcare Products Regulatory Agency approved Casgevy for patients on Nov. 16; US and European regulators are still considering the drug for approval. Casgevy works by editing blood stem cells to restart making fetal hemoglobin. Clinical trials have shown this dramatically improves the lives of people with either condition.

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