Crispr: UK launches world’s first Crispr gene therapy

Great Britain was the first country in the world to approve a gene therapy based on the Crispr genetic scissors. The UK Medicines Agency MHRA has granted the drug Casgevy a conditional marketing authorization for patients aged twelve years and over for the treatment of two blood disorders. This affects sickle cell anemia and beta thalassemia, a disorder in the formation of red blood pigments.

Casgevy is the first approved drug to use Crispr genetic scissors for targeted genetic modification. Emmanuelle Charpentier and Jennifer A. Doudna received the 2020 Nobel Prize in Chemistry for the discovery of Crispr. The process allows DNA to be specifically cut and modified. Individual genes can then be inserted, removed or deactivated.

According to the MHR, no significant concerns emerged during the investigation and the safety of the drug is being closely monitored by authorities.

“I hope this is the first of many applications of this Nobel Prize-winning technology to benefit patients with serious diseases,” said Samarth Kulkarni, CEO of Crispr Therapeutics. The company, along with Vertex Pharmaceuticals, is one of the manufacturers of the therapy. Both are based in the United States.

During Casgevy therapy, stem cells are taken from the patient’s bone marrow and the faulty gene is modified in the laboratory so that the body can produce functioning hemoglobin. The modified cells are then reintroduced to the patient via an infusion.

Both diseases are hereditary

Both sickle cell anemia and beta thalassemia are inherited genetic disorders. They are caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body. Both are painful and lifelong illnesses which in some cases can be fatal, explained MHRA acting director Julian Beach.

Clinical studies have shown that Casgevy restores healthy hemoglobin production and relieves disease symptoms in the majority of participants with sickle cell disease and transfusion-dependent beta-thalassemia.

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