Our Proprietary Lipid Nano-Particles Platform

Lipid nanoparticles are spherical vesicles made of ionizable lipids, which are positively charged at low pH (enabling RNA complexation) and neutral at physiological pH (reducing potential toxic effects, as compared with positively charged lipids, such as liposomes). Due to their size and properties, lipid nanoparticles are taken up by cells via endocytosis, and the ionizability of the lipids at low pH (likely) enables endosomal escape, which allows release of the cargo into the cytoplasm. 

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In addition, lipid nanoparticles usually contain a helper lipid to promote cell binding, cholesterol to fill the gaps between the lipids, and a polyethylene glycol (PEG) to reduce opsonization by serum proteins and reticuloendothelial clearance. The relative amounts of ionizable lipid, helper lipid, cholesterol and PEG substantially affect the efficacy of lipid nanoparticles, and need to be optimized for a given application and administration route. Moreover, lipid type, size and surface charge impact the behavior of lipid nanoparticles in vivo.

Our NLP Platform:

Our initial work started with lipids licensed from Prof. Dan Peer's Laboratory. Through our work we have optimized these for effective muscle delivery. This is based on a unique and proprietary LNP-based formulation. Our delivery system enables efficient delivery of mRNA to the muscles via systemic administration. This reflects years of work scanning numerous lipids and optimizing the formulations for delivery.