I AM OPTIMISTIC. SEVERAL GENE AND CELL THERAPIES ARE APPROVED or in late-stage development. Physician-researchers are now studying the ability of platforms such as Sangamo’s zinc finger proteins (ZFPs) and CRISPR Cas9 to address mutations that lead to some genetic diseases. As a physician, I can’t overstate the impact this will have on people’s lives.
It is also exciting to look to the next wave of treatments. The science is advancing toward allogeneic cell therapies (modified healthy donor cells introduced to patients) and in vivo genome engineering therapies where the entire DNA modification occurs inside the body. With every advance, we come closer to the day when there will be three primary modes of medicine: prevent with vaccines, treat with small molecules, and cure with genomic medicine.
SANDY MACRAE, M.B., CH.B., PH.D., is CEO of Sangamo Therapeutics.