Great Britain approves the world’s first Crispr gene therapy

2023-11-16 15:37:28

A gene therapy based on the Crispr genetic scissors has been approved for the first time in the world in Great Britain. The British Medicines Agency (MHRA) gave the green light on Thursday for conditional marketing authorization for the drug Casgevy for patients aged 12 and over to treat two blood disorders – sickle cell anemia and beta thalassemia, a disorder of the production of red blood pigments. Casgevy is the first approved drug to use the CRISPR gene scissors to specifically alter genetic material. This earned its discoverers the Nobel Prize in Chemistry in 2020. The process can be used to specifically cut and modify DNA; Genes can be inserted, removed or turned off.

Sickle cell anemia and beta thalassemia are inherited genetic disorders caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body. “Both sickle cell disease and beta thalassemia are painful, lifelong diseases that, in some cases, can be fatal,” said MHRA Interim Director Julian Beach. In clinical trials, Casgevy was found to restore healthy hemoglobin production and reduce disease symptoms in the majority of participants with sickle cell disease and transfusion-dependent β-thalassemia. According to the MHR, no significant safety concerns were identified during the investigations and the safety of the drug is being closely monitored by the authority.

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The therapy involves taking stem cells from the patient’s bone marrow and modifying the faulty gene in the laboratory so that the body can produce functioning hemoglobin. The modified cells are then reintroduced to the patient via an infusion. The US-based manufacturers of the therapy, Vertex Pharmaceuticals and CRISPR Therapeutics, welcomed the approval. “I hope this is the first of many applications of this Nobel Prize-winning technology to benefit patients with serious illnesses,” said CRISPR Chief Executive Samarth Kulkarni.