Crispr: UK launches world’s first Crispr gene therapy

Britain was the first country in the world to approve a gene therapy based on the Crispr gene scissors. The British Medicines Agency (MHRA) granted the medicine 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 that uses the Crispr genetic scissors for targeted genetic modification. Emmanuelle Charpentier and Jennifer A. Doudna received the Nobel Prize in Chemistry in 2020 for the discovery of Crispr. The process allows DNA to be specifically cut and modified. Individual genes can therefore be inserted, deleted or inactivated.

According to the MHR, no major concerns were identified during investigations and the authority is closely monitoring the safety of the drug.

“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 US.

During Casgevy therapy, stem cells are removed 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 inherited.

Both sickle cell anemia and beta thalassemia are inherited genetic diseases. They are caused by errors in the genes for hemoglobin, which red blood cells use to transport oxygen around the body. Both are painful, life-long diseases that can be fatal in some cases, explained MHRA acting director Julian Beach.

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

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