Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) is now the first licensed treatment that uses the gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.
SCD and beta-thalassaemia are both genetic conditions caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.
For those with SCD, this genetic error can lead to attacks of very severe pain known as vaso-occlusive crises (VOCs), serious and life-threatening infections and anaemia, while in beta-thalassaemia, it can lead to severe anaemia, and patients often require regular blood transfusions throughout their lives…
