The UK's medicines regulator has granted approval for the world's first gene therapy treatment for sickle cell disease. The treatment, called Casgevy, is the first medicine to be licensed using the gene editing tool CRISPR. It works by targeting the problematic gene in a patient's bone marrow stem cells, allowing the body to produce properly functioning hemoglobin. Casgevy is currently being reviewed by the US Food and Drug Administration for approval. The makers of the treatment, Vertex Pharmaceuticals and CRISPR Therapeutics, are working with health authorities to establish a price and secure reimbursement for eligible patients.
The UK's medicines regulator has granted approval for the world's first gene therapy treatment for sickle cell disease. The treatment, called Casgevy, is the first medicine to be licensed using the gene editing tool CRISPR. It works by targeting the problematic gene in a patient's bone marrow stem cells, allowing the body to produce properly functioning hemoglobin. Casgevy is currently being reviewed by the US Food and Drug Administration for approval. The makers of the treatment, Vertex Pharmaceuticals and CRISPR Therapeutics, are working with health authorities to establish a price and secure reimbursement for eligible patients.
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