first drug using CRISPR technology approved in the world

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorized the first therapy based on CRISPR-Cas9 technology to treat two inherited blood diseases sickle cell anemia and the Transfusion-dependent β-thalassemia in patients aged 12 years or older, as announced by this organization in a statement.

The new drug has been developed by Vertex Pharmaceuticals and CRISPR Therapeutics, and is also being evaluated by the regulatory agencies of the United States (FDA) and Europe (EMA) for both hemoglobinopathies. Exagamglogogen autotemcel (exa-cel) will be marketed under the name casgevy, and has become the first authorized medicine in the world that uses CRISPR/Cas9 gene editing toolwhose creators were awarded the 2020 Nobel Prize in Chemistry.

“I am pleased to announce that we have licensed an innovative, first-of-its-kind gene editing treatment called Casgevy; “In trials it has been found to restore normal hemoglobin production in the majority of participants with sickle cell disease and transfusion-dependent β-thalassemia, alleviating the symptoms of the disease,” Julian Beach explained in the agency’s statement. , director of Healthcare Quality and Access at the MHRA.

How the first approved drug based on gene editing works

Sickle cell disease and β-thalassemia are inherited blood disorders caused by changes in the hemoglobin genes, which red blood cells use to transport oxygen throughout the body. Treatment for beta thalassemia is personalized based on its severity, but many patients need to receive regular transfusions, which interferes with their activities and can lead to iron buildup in the body and reduce their life expectancy.

Sickle cell anemia damages organs and patients can develop painful blockages of blood vessels, which can cause acute chest syndrome, stroke, jaundice and symptoms of heart failure. The treatments available so far are aimed primarily at relieving symptoms. The only cure for this is a stem cell transplant from a compatible donor, but few patients can access this.

“The modification of the patient’s bone marrow stem cells constitutes a true cure for the disease rather than a treatment”

The new medicine has been specifically designed to edit the defective gene in the stem cells of the patient’s bone marrow: once the cells have been extracted from the patient and treated in the laboratory, they are infused; The results obtained are potentially maintained for life. The price of the treatment has not yet been established in the United Kingdom, although taking into account the figures achieved by other gene therapy drugs it will exceed one million dollars per patient.

“This is a big step in advancing medical approaches to address genetic diseases that we never thought would be possible to cure. “The modification of the patient’s bone marrow stem cells avoids the problems associated with immune compatibility, that is, the search for donors compatible with the patient and the subsequent immunosuppression, and constitutes a true cure for the disease rather than a treatment.” Dr. Alena Pance, senior lecturer in genetics at the University of Hertfordshire, told Science Media Center in the United Kingdom.

“This is a historic approval that opens the door to new applications of CRISPR therapies in the future for the possible cure of many genetic diseases,” concludes Kay Davies, a geneticist at the University of Oxford, in statements to the same medium.


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