FDA approves first gene-editing therapy

The Food and Drug Administration (FDA) recently approved Casgevy (exagamglogene autotemcel), the first cell-based gene-editing therapy to utilise the CRISPR/Cas9 system (CRISPR) in history, to treat transfusion-dependent beta thalassemia. In December last year, the FDA also approved Casgevy for the treatment of life-threatening forms of sickle cell disease. This means that adult patients in the US with this red-blood-cell disorder – or ‘hemoglobinopathy’ – will be able to access this as a one-time treatment.