The UK has approved a groundbreaking gene therapy, Casgevy, for two inherited blood disorders, sickle-cell disease, and transfusion-dependent beta thalassaemia. Using CRISPR gene editing, the treatment edits the faulty gene in a patient’s bone marrow stem cells, enabling the production of healthy haemoglobin. Casgevy has shown promising results in clinical trials, with the majority of participants experiencing relief from symptoms. The approval marks a world-first for CRISPR-based therapy, offering a new option beyond the risk-prone bone marrow transplant.
How do you see this development impacting the future of gene therapies and personalised medicine?