Nucleic Acid Delivery

Nucleic acids are relatively large molecules that are unstable in the bloodstream and unable to readily diffuse across membranes into target cells. To realize the tremendous therapeutic potential of RNAi-based drugs therefore, effective delivery is critical.

LNP

Tekmira’s siRNA delivery technology platform is termed LNP for "lipid nanoparticle". LNPs fully encapsulate and systemically deliver a variety of nucleic acid molecules such as siRNA. Pre-clinical studies have shown them to be effective in delivering the drug to target organs and into cells where the nucleic acid-based drug can carry out its desired effect while minimizing systemic toxicity.

LNP technology relies on something called the "enhanced permeability and retention effect", which occurs because these nucleic acid-containing particles have a long circulation time in the blood, resulting in increased accumulation at sites of vascular leak such as those found at sites of tumor cell growth, infection or inflammation. Once at the target site, cells take up the LNP through endocytosis and the nucleic acid payload is delivered inside the cell resulting in unparalleled potency.