Disconnect exists between the media hype around gene-editing treatment and the reality for patients
In November 2023, the UK became the first country in the world to authorise a Crispr gene-editing therapy .
Casgevy (exagamglogene autotemcel) is for patients with debilitating forms of sickle cell disease and transfusion-dependent β-thalassaemia – both of which are caused by errors in genes that code for haemoglobin – a protein found in red blood cells which carries oxygen around the body.
But how is the world’s first Crispr-based treatment performing in UK patients so far?