Pipeline Focus

MDRNA is focusing its pipeline efforts on the discovery and development of novel siRNA therapeutics to treat diseases of the liver, in particular hepatocellular carcinoma (liver cancer), a critical unmet medical need. MDRNA’s scientists have been working extensively on multiple gene targets for oncology indications and those most relevant to liver cancer. To inhibit these targets most efficiently, the Company is using its proprietary UsiRNA Platform, which provides an immediate inroad into this indication. In addition, since delivery to the liver is readily accessible with MDRNA’s DiLA² Platform, the Company believes that this program represents an outstanding opportunity for near term success in pre-clinical studies and rapid progress toward human clinical trials.

Various pre-clinical studies are in progress and the Company expects to have in vitro data from liver cancer cells and in vivo data from models of liver cancer in the third quarter of 2009. Current efforts are focused on selection of a molecular target or targets, dose levels, dose schedules and durability of RNAi in appropriate studies. Along with the pre-clinical data, MDRNA will consult with clinical experts in order to allow its scientists to maximize and focus the pre-clinical studies on liver cancer phenotypes most likely to respond to siRNA-based therapy.