UK Approves World's First CRISPR-Based Medicine

Britain's drugs regulator has approved a groundbreaking treatment for two painful and debilitating lifelong blood disorders, which works by "editing" the gene that causes them. From a report: The Medicines and Healthcare products Regulatory Agency (MHRA) has given the green light for Casgevy to be used to treat sickle cell disease and beta thalassemia. It is the first medicine licensed anywhere that works by deploying gene editing that uses the "genetic scissors," known as CRISPR, for which its inventors won the Nobel prize for chemistry. Casgevy's developers hope the pioneering treatment could banish the pain, infections and anaemia sickle cell disease brings and the severe anaemia experienced by those with beta thalassemia. About 15,000 people in the UK, almost all of African or Caribbean heritage, have sickle cell disease. About 1,000 -- mainly of Mediterranean, south Asian, south-east Asian and Middle Eastern background -- have beta thalassemia and need regular blood transfusions to treat their anaemia. Experts in the illnesses hope Casgevy may be a cure, making it no longer necessary for people with the conditions to have a bone marrow transplant. Until now this has been the only treatment available, even though the body can reject the donor marrow. The Sickle Cell Society welcomed the MHRA's decision as a "historic moment for the sickle cell community" which "offers [them] newfound hope and optimism."

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