Doing business as Gencia Corporation, Gencia LLC, and now Gencia Biotech, the firm is privately held focused on the discovery and development of first-in-class therapeutics for important medical needs, based on proprietary mitochondrial targeting technology and novel insights into the role of mitochondria in human health, aging, and disease. Working on treatment for mitochondrial diseases, Gencia Corporation developed a method for manipulating mitochondrial genomes, protofection. The firm can place mitochondrial genomes in living cells, in vitro and in vivo, including reporter genes and other genetic cargo, express proteins in the mitochondria, and even export them to other subcellularlocations. Able to make a huge amount of protein without ever entering the nucleus, or using a viral and immunogenic vector, or a synthetic and cytotoxic cationic lipid. Marking its first partnership, in 2015 Gencia LLC penned a deal with Takeda Pharmaceutical Co. Ltd. for the development of the smaller firms mitochondrial agonists of the glucocorticoid receptor (MAGR). The agreement called for the partners jointly to select two final candidates, with Takeda holding the exclusive right to advance each into clinical trials and through the development process. Gencia was paid an undisclosed upfront sum plus milestone payments along the way referneced in various sources as upwards of $500 million.
