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.
Arbutus has a worldwide, non-exclusive license to a novel RNA trigger technology called Unlocked Nucleobase Analog (UNA) from Marina Biotech, Inc.
UNA technology can be used in the development of RNAi therapeutics, which treat diseases by silencing specific disease-causing genes. UNAs can be incorporated into RNAi drugs and have the potential to improve them by increasing their stability and reducing off-target effects.
Our license to Marina's UNA technology enables us to diversify RNAi therapeutics. By leveraging our expertise in LNP delivery in combination with our knowledge of therapeutic RNA trigger design, we can optimize UNAs in our development pipeline. We also can provide our pharmaceutical partners the opportunity to license UNAs combined with our LNP delivery technology to develop RNAi therapeutics.