OliX’s proprietary RNAi triggering molecule can efficiently internalize and silence a target gene without any delivery vehicles

To develop gene silencing therapy using RNA interference mechanism, it is critical to effectively deliver RNAi triggering molecules (siRNA or asiRNA) inside cells where the target mRNA is located. However, due to the large size and negative charges of RNA molecules, efficient RNA delivery into cells is technically challenging and remains a significant hurdle for the development of RNAi therapeutics.

The most widely used delivery method is to generate a complex between RNA molecule and cationic liposome/polymers. However, cationic delivery vehicles can exert various types of toxicity by interacting with negatively charged cell surface or interacting nonspecifically with proteins in the human body.

cp-asiRNA is a proprietary RNAi triggering structure developed by OliX, which can efficiently internalize and knock down target gene silencing without any transfection reagents. cp-asiRNA shows highly efficient delivery into hard-to-transfect cells including primary cells and suspension cells. It has also been tested for its superb gene silencing activity in animal models.

Because cp-asiRNA can induce gene silencing via simple local administration into organs such as skin and eye without requiring special formulation or instrument, we believe cp-asiRNA is so far the most effective and facile technology for therapeutic development targeting various diseases with unmet needs.

• cp-asiRNA is a novel, chemically modified asymmetric RNAi compound
• No delivery vehicle is required
• It can use both in vitro and in vivo without transfection reagents