Timeline milestone
Casgevy's approval expands to a second blood disorder
From From Bacterial Defence to Medicine: The CRISPR Gene-Editing Revolution
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Approval
Casgevy's approval expands to a second blood disorder
The FDA approved Casgevy for transfusion-dependent beta thalassemia, weeks after its sickle-cell approval, followed by UK and EU authorizations, extending the CRISPR therapy to tens of thousands more eligible patients.
National Institutes of Health Weeks after its sickle-cell approval, the FDA cleared Casgevy for transfusion-dependent beta thalassaemia, a disease that shares the same underlying gene target. The European Commission followed in February 2024, and UK and other regulators granted authorisations across 2023 and 2024, together making more than 8,000 additional patients eligible. The rapid, multi-geography rollout showed that a single approved CRISPR therapy could scale into a genuine clinical product line rather than a one-off treatment.
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