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Genome editing technologies such as CRISPR–Cas9 are being developed as therapeutic platforms, especially for monogenic diseases that could be treated by targeting the associated mutations. The first CRISPR–Cas9-based medicinal product, Casgevy (exagamglogene autotemcel), has recently been granted marketing authorization in the European Union for sickle cell disease and β-thalassemia, and a substantial number of other genome-editing medicinal products (GEMPs) are in development.