MDRNA focuses on discovering and developing new siRNA therapeutics to treat liver diseases, especially hepatocellular carcinoma (HCC), a type of liver cancer with significant unmet medical need. The company’s scientists study multiple gene targets related to oncology and liver cancer and use the proprietary UsiRNA Platform to help inhibit these targets efficiently.

To deliver these therapeutics effectively, MDRNA applies its DiLA2 Platform, which supports targeting of liver tissue. Because delivery to the liver is accessible and integrated with these technologies, the company considers this program a strong opportunity for advancing preclinical research and moving toward future clinical evaluation.

Preclinical studies are ongoing, including in vitro testing in liver cancer cells and in vivo evaluation in relevant models. Current work focuses on selecting appropriate molecular targets, determining dose levels, optimizing dose schedules, and assessing the durability of RNAi effects. MDRNA also collaborates with clinical experts to ensure studies focus on liver cancer phenotypes most likely to respond to siRNA-based therapies.