Synthetic RNAi trigger molecules are designed to suppress the production of proteins through the RNAi mechanism. Sequencing of the human genome has provided the information needed to design RNAi trigger molecules to target a wide range of disease-causing proteins. In fact, RNAi trigger molecules can be designed in a shorter time frame than synthesizing and screening conventional small molecule drugs. Further, RNAi trigger molecules can bind to a target protein mRNA with great specificity. When RNAi trigger molecules are introduced into the cell cytoplasm, they are rapidly incorporated into an RNA-induced silencing complex (RISC) and guided to the target mRNA. The RISC cuts and destroys the target mRNA, preventing the subsequent production of this specific protein. The RISC can remain stable inside the cell for weeks, destroying many more copies of the target mRNA and suppressing this specific protein for long periods of time.
Arbutus has a worldwide license for the discovery, development and commercialization of products using RNAi trigger molecules directed to 13 gene targets.