US approves drug based on CRISPR gene editing technology

In the USA, the drug Casgevy, which works on the CRISPR gene editing system, has been approved. This is stated in the report of the American Food and Drug Administration (FDA).

The drug will be used to treat sickle cell anemia in patients over 12 years old. In the United States, about 100 thousand people suffer from this hereditary pathology.

The main problem in sickle cell anemia is a mutation of hemoglobin, a protein found in red blood cells that delivers oxygen to the body's tissues. This mutation causes the red blood cells to take on a crescent or sickle shape. They restrict blood flow in blood vessels and oxygen delivery to body tissues, resulting in severe pain and organ damage. This can result in disability or early death of the patient.

With this CRISPR technology, which has been dubbed "genetic scissors," it becomes possible to precisely edit (remove, add or replace) DNA. The modified blood stem cells are transplanted back into the patient, where they attach and multiply in the bone marrow, increasing the production of fetal hemoglobin, which facilitates oxygen delivery.

The FDA publication says the gene therapy promises to provide more targeted and effective treatments, especially for people with rare diseases for which current treatment options are limited.

The U.S. became the second country in the world to approve this treatment option. The drug was previously approved in the UK.