Takeaways from AP’s report on access to gene therapies for rare diseases
Associated PressJUN 21 2024
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 National Institute for Health and Care Excellence said thalassemia mainly affects those of Pakistani, Indian, and Bangladeshi ethnic origin in the UK.
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.