UK Clears Gene-Editing Therapy For Rare Blood Disorder
UK Clears Gene-Editing Therapy For Rare Blood Disorder
The Facts
The UK has cleared using gene-editing therapy via the National Health Service (NHS) to treat blood disorder beta thalassaemia.The UK's National Health Service (NHS) has been cleared to use gene-editing therapy to treat the 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 therapy, termed Casgevy, may help the estimated 2.3k people in the UK with thalassaemia, a rare group of inherited disorders in which the body produces too little hemoglobin and stunts its ability to carry oxygen through.
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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