LONDON (AP) — Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K.
The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over.
Casgevy is currently being reviewed by the U.S. Food and Drug Administration; the agency is expected to make a decision early next month, before considering another sickle cell gene therapy.
Millions of people around the world, including about 100,000 in the U.S., have sickle cell disease.
Scientists believe being a carrier of the sickle cell trait helps protect against severe malaria.
Persons:
”, Helen O'Neill
Organizations:
Medicines and Healthcare Regulatory Agency, Vertex Pharmaceuticals, CRISPR Therapeutics, Helen O'Neill of University College London, Medicines, U.S . Food, Drug Administration, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP
Locations:
Europe, CRISPR, South Asian, Britain, U.S, Africa, India, Eastern