Gene-editing drugs are opening a new frontier in medicine
From our UK edition
Older readers with bulging medicine cabinets, or parents eyeing their children’s distant futures, are not encouraged to read the New England Journal of Medicine – despite its prestige. A paper just published there, however, matters to them and to me. ‘In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia’ is not a catchy title, but what it describes is going to catch on. VERVE-102 is a combination drug designed to edit the bases of a gene – PCSK9 – using a development of CRISPR technology, all packaged so the edit reaches the cells that matter in the liver. Some of those born with superior versions of the PCSK9 gene have their lifetime risk of coronary heart disease – the world’s biggest killer – halved. For others it is all but abolished.