MHRA authorises Casgevy, world-first CRISPR gene therapy, for sickle-cell disease and beta-thalassemia
The Medicines and Healthcare products Regulatory Agency has authorised Casgevy (exagamglogene autotemcel) for patients aged 12 and over with sickle-cell disease or transfusion-dependent beta-thalassemia. It is the first medicine to be licensed that uses CRISPR, the gene-editing tool whose inventors won the Nobel Prize in 2020. The MHRA reached its decision after assessing the therapy's safety, quality and effectiveness. Both conditions stem from errors in the genes for haemoglobin, which red blood cells use to carry oxygen around the body. Sickle-cell disease is particularly common in people with an African or Caribbean family background and can cause attacks of severe pain, serious infections and anaemia. Beta-thalassemia mainly affects people of Mediterranean, south Asian, southeast Asian and Middle Eastern origin.
Casgevy is the first CRISPR-based medicine to receive a licence, giving patients with sickle-cell disease and beta-thalassemia a treatment authorised after a full MHRA safety and efficacy review.
Source: MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia (gov.uk).
Written by the Genomes desk from the primary source linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].
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