In 2023, the UK was the first country to approve a Crispr therapy. How’s it doing?

Sickle cell and thalassaemia centre

Source: © David Gee/Alamy Stock Photo

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?