The US approves treatment of a deadly disease using CRISPR gene editing technology

The U.S. Food and Drug Administration (FDA) has approved two drugs for the treatment of sickle cell disease. This is stated in the message on the FDA website.

It is a painful and fatal disease that has not yet had a universal successful treatment. It affects about 100 thousand people in the United States alone. But now the FDA has taken an important step to fight the disease.

The FDA approved two gene therapies – Casgevy and Lyfgenia. Vertex Pharmaceuticals and Bluebird Bio Inc. received marketing authorizations for their use, respectively. Both drugs are made from the patient’s own modified blood stem cells and are approved for the treatment of people aged 12 years and older.

However, Casgevy’s peculiarity is that it is the first drug based on CRISPR technology for gene editing. The FDA notes that this indicates innovative progress in the field of gene therapy.

“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health,” the Department said in a statement.

Recently, it was reported that the US is considering the possibility of approving a therapy based on CRISPR technology. Thanks to it, genetic changes are made in stem cells and then “returned” to the patient.