A drug based on CRISPR/Cas gene scissor technology has been approved in Great Britain for the first time in the world. The Medicines and Healthcare products Regulatory Agency (MHRA) announced this on Thursday.
The drug, called Casgevy, is approved for the treatment of sickle cell disease and beta-thalassemia, two genetic blood diseases, in patients aged 12 and older. Both diseases are caused by errors in the gene for hemoglobin. This is an iron-containing protein complex found in red blood cells and used to transport oxygen.
Beta thalassemia and sickle cell anemia are two of the most common hereditary diseases worldwide. They are caused by a mutation of a gene called HBB. HBB is responsible for the production of beta-globin, an important component of the red blood pigment hemoglobin.
Anemia, insufficient supply of oxygen to the body
The mutation causes beta globins to be produced incorrectly. This leads to a lack of functioning hemoglobin. This can usually lead to, among other things, red blood cells dying prematurely. Anemia occurs. The organs and the entire body are then chronically insufficiently supplied with oxygen.
The drug should change defective genes
The so-called gene scissors can specifically target individual genes. Casgevy is designed to change the faulty genes in patients’ bone marrow stem cells so that they can produce functioning hemoglobin, the MHRA said in a statement.
For this purpose, stem cells are extracted from the bone marrow, processed in the laboratory and then reintroduced into the patient. The result has the potential to have a lifelong effect, the report said.
Source: Krone
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