Landmark Breakthrough: UK Approves Gene Therapy for Sickle Cell Disease and Thalassaemia
The UK drug regulator recently approved a groundbreaking gene therapy for the cure of sickle cell disease and thalassaemia, marking a significant milestone in medical advancement. This therapy, named Casgevy, is the world's first licensed treatment based on the revolutionary gene editing technology Crispr-Cas9, which earned its innovators a Nobel Prize in 2020. The Crispr-Cas9 Innovation The Crispr-Cas9 technology, often referred to as "genetic scissors," became available in 2012 and has since revolutionised the field of biotechnology. Casgevy utilises this technology to edit the faulty gene responsible for blood disorders, offering a potential lifelong cure. Understanding the Therapy Casgevy targets the gene BCL11A, crucial for the transition from foetal to adult haemoglobin. By editing the patient's own blood stem cells, the therapy stimulates the production of foetal haemoglobin, mitigating the symptoms of sickle cell disease and thalassaemia. This ap