GEM is preclinical cell-based gene therapy program that will focus on developing effective, transiently transfected, off-the-shelf therapeutics for pulmonary diseases or acute inflammatory diseases, such as pulmonary arterial hypertension (PAH) or sepsis.
Mesenchymal stem cells (MSCs) are a type of adult somatic stem cells that can be isolated from various human sources and possess great immunomodulatory potential. MSCs are “immune-privileged”, permitting their use in allo-transplantation (i.e. cells from another individual) without the need for immunosuppressive therapy, making it feasible for MSCs to be delivered as an “off-the-shelf” product for the treatment of acute and chronic inflammatory disorders. Gene-enhanced MSCs are superior to unmodified cells, as the beneficial effects of MSCs can be tailored to specific conditions by selecting from a menu of therapeutic genes, while directly delivered to the site of injury (i.e., the lung).