UK first to OK gene therapy for sickle cell & thalassemia

Gene-Editing, Sickle Cell Treatment , Gets UK Approval . Regulators in the United Kingdom have authorized the world's first gene therapy treatment for people with sickle cell disease. Regulators in the United Kingdom have authorized the world's first gene therapy treatment for people with sickle cell disease. 'Time' reports that the decision could offer relief for thousands of people in the U.K. who suffer from the crippling disease. On November 16, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, which uses the gene-editing tool CRISPR. The U.K. regulator approved Casgevy for the treatment of patients over the age of 12 with sickle cell disease and thalassemia. . The future of life-changing cures resides in CRISPR-based (gene-editing) technology, Dr. Helen O'Neill, University College London, via 'Time'. The use of the word ‘cure’ in relation to sickle cell disease or thalassemia has, up until now, been incompatible. , Dr. Helen O'Neill, University College London, via 'Time'. Dr. Helen O'Neill, of University College London, called the MHRA's approval of Casgevy , “a positive moment in history.”. 'Time' reports that sickle cell disease can block blood flow and result in excruciating pain, organ damage, strokes and other serious health problems. The new treatment is currently under review by the United States Food and Drug Administration, which is expected to deliver a decision next month. High costs have caused experts to express concerns that gene therapy treatments could be financially out of reach for most people.