Your recent News Feature highlights how people with extremely rare genetic diseases cannot access emerging CRISPR gene-editing technologies, owing to the challenges of developing one-off therapies and the perceived small market (Nature 630, 284–288; 2024). High costs for treatments such as Casgevy, approved in the past year in Europe and the United States to treat the blood conditions sickle-cell disease and β-thalassaemia, also prevent their widespread adoption.
Competing Interests
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The authors declare no competing interests.