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.
Gene-editing therapy faces significant limitations. Its high cost makes it inaccessible to many, particularly in low- and middle-income countries where the diseases are more prevalent. Also, regulatory and intellectual property challenges further complicate accessibility and affordability. Without new funding models and broader access strategies, the transformative benefits of gene-editing therapies may remain out of reach for those who need them most.
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.