Where Will CRISPR Therapeutics Be in 10 Years?
CRISPR -- which stands for "clustered regularly interspaced short palindromic repeats" -- has been a hot area of research and investment since scientists discovered that this naturally occurring gene-editing function of bacteria could conceivably be used to treat genetic diseases. Several companies are using gene-editing in their attempts to cure illnesses caused by errors on a single gene such as sickle cell disease, hemophilia, and cystic fibrosis. One of them, CRISPR Therapeutics (NASDAQ: CRSP), has produced results that could not only make it a winner in single-gene disorders, but position it to tackle much more complex -- and profitable -- diseases in the years ahead.
CTX001 is the company's candidate treatment for sickle cell disease and beta-thalassemia -- two disorders that affect the oxygen-carrying cells in our blood. Medical professionals harvest a patient's own cells from bone marrow, use CTX001 to edit the gene affecting red blood cell production, and infuse the cells back into the body.
In 2015, CRISPR Therapeutics formed a partnership with Vertex Pharmaceuticals (NASDAQ: VRTX) to develop a number of treatments using this technology, accepting cash, equity, and future royalties. In exchange, Vertex got the rights to market the treatments developed.
Source Fool.com
