A breakthrough in medicine. A major decision made by an American company

Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the treatment, called Casgevy, involves collecting stem cells from a patient’s blood, which are then genetically modified using CRISPR technology and injected back into the patient. In addition to this therapy, the FDA has also approved another gene therapy to treat the same disease.

Sickle cell anemia is a relatively rare but serious genetic disease that disproportionately affects black people. It is associated with pain and the need for regular blood transfusions.

So far, the only treatment is bone marrow transplantation from a donor, which is associated with a risk of transplant rejection. The new treatment β€” used once but requiring a hospital stay of several weeks β€” currently costs $2.2 million. from the patient.

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