The US is considering treatment of a deadly disease using CRISPR gene editing technology
The United States is considering a new therapy that could potentially cure people of sickle cell disease. This is a painful and fatal disease that has no universal successful treatment, reports Slashdot.
We are talking about the exa-cel method, developed by the Boston-based Vertex Pharmaceuticals and the Swiss CRISPR Therapeutics. It is based on a gene editing technology called CRISPR.
Exa-cel is currently being studied by the US Food and Drug Administration (FDA). If approved, this therapy could become the first such treatment to receive the green light from the FDA.
The principle of exa-cel treatment is to use a person with anemia’s own stem cells. Thanks to CRISPR technology, genetic changes are made in these cells and then “returned” to the patient.
Vertex Pharmaceuticals says that this treatment was found to be safe in studies. It also had a “very high benefit-risk ratio for patients with severe sickle cell disease.”
The FDA is expected to make a decision on exa-cel by December 8.