The study showed that a specific Locked Nucleic Acid (LNA) antimiR that binds to the liver-specific microRNA-122 can readily enter the liver and antagonize the miRNA target molecule. Remarkably, administration of just three doses over the course of one week markedly altered serum lipid profiles for seven weeks, resulting in a significant decrease in total cholesterol, with no evidence of adverse toxicity.
Matthew Lawrence, RxGen’s president and chief scientific officer, said: “LNA-antimiR compounds are an effective approach to silence microRNAs in primates. We are all very excited to see how experimentally straightforward and safe it was to achieve a sustained and pronounced physiologic change with silencing of the liver-specific microRNA-122 using Santaris Pharma’s LNA technology.”