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.

Acknowledgements

We thank EMA colleagues S. Thirstrup, P. Celis and J. Jansen for their valuable comments on the manuscript.

The authors declare no competing interests.