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The world’s first CRISPR therapy is approved: who will receive it?

The go-ahead for Vertex’s gene editing therapy in sickle cell disease and β-thalassemia is a historic milestone, but this one-time treatment is costly.
  1. Cormac Sheridan

    1. Dublin, Ireland

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Misshapen red blood cells clump and clog blood vessels in sickle cell disease, depriving tissues of oxygen and causing severe pain. Credit: H.S. Photos / Alamy Stock Photo

The UK’s regulator has approved the world’s first CRISPR–Cas9 gene editing therapy, which aims to cure sickle cell disease and transfusion-dependent β-thalassemia. Casgevy (exagamglogene autotemcel) is a first-of-its-kind treatment made by Vertex Pharmaceuticals and CRISPR Therapeutics in Zug, Switzerland. It comes just 11 years after Jennifer Doudna and Emmanuelle Charpentier invented the technology. The green light from the Medicines and Healthcare Products Agency represents a major scientific achievement for Vertex and CRISPR and a landmark for the biotech industry.

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Nature Biotechnology 42, 3-4 (2024)

doi: https://doi.org/10.1038/d41587-023-00016-6

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