Crispr: UK launches world’s first Crispr gene therapy

Britain was the first country in the world to approve a gene therapy based on Crispr genetic scissors. The British Medicines Agency (MHRA) has granted conditional marketing authorization to the drug Casgevy for patients aged 12 and over to treat 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 were awarded the 2020 Nobel Prize in Chemistry for their discovery of Crispr. The process allows you to specifically cut and modify DNA. Individual genes can therefore be inserted, removed or turned off.

According to the MHR, no significant concerns were identified during investigations and the safety of the medicine is being closely monitored by the authority.

“I hope this is the first of many applications of this Nobel Prize-winning technology to benefit patients with serious illnesses,” 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 USA.

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

Both diseases are hereditary

Both sickle cell anemia and beta thalassemia are inherited genetic diseases. 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, lifelong illnesses and can be fatal in some cases, explained MHRA acting director Julian Beach.

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

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