Takeaways from AP’s report on access to gene therapies for rare diseases
Associated PressJUN 2024
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.
There is a 50% chance that over 57 gene-edited babies will have been born worldwide by the end of 2029, according to the Metaculus prediction community.