Regulators in the U.K. approved the world's first CRISPR-derived gene editing therapy, and U.S. approval is expected soon, according to the journal Nature Biotechnology. Manufacturers Vertex Pharmaceuticals and CRISPR Therapeutics designed the treatment, which is marketed under the trade name Casgevy, to cure the blood disorders sickle cell disease and beta thalassemia.
Invented in 2009, CRISPR technology (short for Clustered Regularly Interspaced Short Palindromic Repeats) can precisely target and edit specific pieces of genetic code in living organisms, according to the Broad Institute.
Sickle cell disease is a result of a faulty gene that alters hemoglobin production and leads to malformed sickle-shaped red blood cells. The irregular shape effectively clogs blood vessels and causes episodes of extreme pain. In beta thalessemia, the body does not produce sufficient hemoglobin, which can cause a number of complications and shorten lifespans.
Casgevy uses CRISPR technology to precisely edit the faulty gene and direct the body to produce functioning hemoglobin, effectively curing the disorders.
Two major obstacles remain for most patients, however — each treatment will cost millions of dollars and require an extended hospital stay, and relatively few physicians have the necessary expertise.
