LONDON (AP) — The Medicines and Healthcare Regulatory Agency in Britain has given the green light to the world’s first gene therapy treatment for sickle cell disease. Casgevy, the first drug to be authorized using the gene editing technology CRISPR, has been approved for patients in the U.K. who suffer from sickle cell disease and thalassemia and are 12 years or older. This news brings hope to thousands of people afflicted with these debilitating diseases. Casgevy is produced by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics, marking a significant development in the treatment of these conditions that have long lacked a lasting cure.
Dr. Helen O’Neill of University College London commented on the approval, stating, “The future of life changing cures resides in CRISPR based (gene-editing) technology.” She added that the use of the word “cure” in relation to sickle cell disease or thalassemia has up to this point been incompatible, making this a historic turning point in the treatment of these conditions.
The revolutionary drug, Casgevy, targets the problematic gene in a patient’s bone marrow stem cells to enable the body to produce properly functioning hemoglobin. The treatment involves extracting stem cells from the patient’s bone marrow and using genetic editing techniques in a laboratory to fix the gene. The edited cells are then reintroduced into the patient’s body, providing a permanent cure.
According to Britain’s regulator, a study on 29 patients revealed that 28 reported no severe pain problems for at least a year after treatment. Additionally, in a study for thalassemia, 39 out of 42 patients who received the therapy did not require a red blood cell transfusion for at least a year after treatment.
While gene therapy treatments typically come with a hefty price tag, Vertex Pharmaceuticals has not yet determined the cost of the treatment in Britain. They are actively working with health authorities to ensure reimbursement and access for eligible patients in need.
Casgevy is currently being reviewed by the U.S. Food and Drug Administration and may soon be available to patients in the U.S. as well. This advancement in gene therapy offers hope to millions of people worldwide who are affected by sickle cell disease, particularly those from regions where malaria is common.
Learn more about sickle cell disease here
And for information on anemia and sickle cell disease, visit the American Society of Hematology
Knowing about the prevalence of the sickle cell trait can help protect against severe malaria. Find out more .
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