United Therapeutics is funding a research program being conducted by Northern Therapeutics, Inc., to which United Therapeutics has exclusive U.S. rights. This program, called GEM, is a cell-based gene therapy program focus on developing effective, transiently transfected, off-the-shelf therapeutics for pulmonary diseases or acute inflammatory diseases. The initial clinical study, called AMETHYST, is being conducted by Northern Therapeutics to study the safety a product candidate called GEM0020 in patients with 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).
The AMETHYST study is being conducted by an affiliate of United Therapeutics in Canada called Northern Therapeutics, Inc. United Therapeutics has a 49.7 percent voting stake and a 71.8 percent financial stake in Northern Therapeutics. United Therapeutics also has the exclusive right to pursue this technology in the United States if the program is successful in Canada.