Intellia is developing treatments with CRISPR/Cas9 that will focus on genome editing in somatic cells in the liver. In preclinical (non-human) studies, we observed that the target gene in the liver was edited, resulting in reduced production of the target protein. We also observed that circulating levels of protein produced by the target gene were permanently reduced throughout the body, except for in the eye and brain, which are closed systems.
Furthermore, the genes in somatic cells can only affect the cells of an individual’s own body. Our investigational treatments are focused on improving the health of the individual and not on future generations.