Britain害羞草研究所檚 medicines regulator has authorized the world害羞草研究所檚 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.
In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.
The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics. To date, bone marrow transplants, an extremely arduous procedure that come with very unpleasant side effects, have been the only long-lasting treatment.
害羞草研究所淭he future of life changing cures resides in CRISPR based (gene-editing) technology,害羞草研究所� said Dr. Helen O害羞草研究所橬eill of University College London.
害羞草研究所淭he use of the word 害羞草研究所榗ure害羞草研究所� in relation to sickle cell disease or thalassemia has, up until now, been incompatible,害羞草研究所� she said in a statement, calling the MHRA害羞草研究所檚 approval of gene therapy 害羞草研究所渁 positive moment in history.害羞草研究所�
Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.
In people with sickle cell 害羞草研究所� which is particularly common in people with African or Caribbean backgrounds 害羞草研究所� a genetic mutation causes the cells to become crescent-shaped, which can block blood flow and cause excruciating pain, organ damage, stroke and other problems.
In people with thalassemia, the genetic mutation can cause severe anemia and patients typically require blood transfusions every few weeks, and injections and medicines for their entire life. Thalassemia predominantly affects people of South Asian, Southeast Asian and Middle Eastern heritage.
The new medicine, Casgevy, works by targeting the problematic gene in a patient害羞草研究所檚 bone marrow stem cells so that the body can make properly functioning hemoglobin. Doctors take stem cells from the patient害羞草研究所檚 bone marrow and use genetic editing techniques in a laboratory to fix the gene. The cells are then infused back into the patient for a permanent treatment.
Britain害羞草研究所檚 regulator said its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at least one year after being treated. In the study for thalassemia, 39 out of 42 patients who got the therapy did not need a red blood cell transfusion for at least a year afterwards.
Gene therapy treatments typically cost thousands of dollars and experts have previously raised concerns that they could remain out of reach for the people most in need of treatment. Vertex Pharmaceuticals said they had not yet established a price for the treatment in Britain and were working with health authorities 害羞草研究所渢o secure reimbursement and access for eligible patients as quickly as possible.害羞草研究所�
Medicines and treatments in Britain must be recommended by a government watchdog before they are made freely available to patients in the national health care system.
Casgevy is currently 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. It from places where malaria is or was common, like Africa and India, and is also more common in , such as people of African, Middle Eastern and Indian descent. Scientists believe being a carrier of the sickle cell trait
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