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Snapshot 1:Thu, Aug 8, 2024 8:23:10 AM GMT last edited by Harish Chander

UK Clears Gene-Editing Therapy For Rare Blood Disorder

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The Facts

  • The UK has cleared using gene-editing therapy via the National Health Service (NHS) to treat blood disorder beta thalassaemia.

  • The therapy, termed Casgevy, is "life-changing" for people with thalassemia, a rare group of inherited disorders in which the body produces too little hemoglobin.


The Spin

Gene-editing therapy will revolutionize thalassemia treatment with the UK’s approval of the groundbreaking Casgevy therapy. It corrects the faulty gene responsible for thalassemia, offering a potential lifelong cure. Previously, the only permanent solution was a bone marrow transplant, requiring a closely matched donor. Casgevy, instead, uses the patient’s own stem cells to produce healthy hemoglobin, drastically reducing the need for regular blood transfusions.


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