Novel RNAi Delivery Technology - Isis Project No 4096

Oxford researchers have developed a novel exosome based delivery platform that delivers and targets exogenous genetic cargoes to selected tissues.

Marketing Opportunity

The RNAi therapeutics market is expected to reach sales of >$1B by 2015. However, despite significant investment, current research is concentrated on life threatening diseases or immuno-privileged sites due to the major obstacle in RNAi therapy, RNAi delivery. The use of exosomes, represents a paradigm shift away from the foreign delivery vehicles currently used in RNAi research (such as liposomes and viral vectors, which can be associated with immune recognition and inflammatory toxicity issues), towards natural nanoparticles capable of systemic RNAi delivery. The use of exosomes has the additional benefit of immune evasion allowing for repeat administration. This novel delivery platform has the potential to revolutionise RNAi therapy by opening up the range of diseases treatable using this approach.

The Oxford Invention

Researchers within the laboratory of Dr Wood, located at the Department of Physiology, Anatomy and Genetics, have developed a novel delivery vehicle based on exosomes. Exosomes are membrane-bound vesicles of nanoparticle size (40-100nm) of endocytotic origin. They are released by numerous cell types and are found in abundance in body fluids where they act as natural carriers of mRNA, miRNA and proteins.

Oxford researchers have utilised this natural carrier capability to develop a method to load exosomes isolated from murine immature dendtritic cells with exogenous genetic cargo. Immature dendritic cells do not stimulate a T-cell mediated immune response and are therefore ideal candidates for use for in vivo delivery of genetic material.

The researchers have demonstrated efficient loading and delivery of exogenous plasmid and siRNA, both in vitro and in vivo. Furthermore, researchers have successfully introduced peptide targeting moieties into exosomes to target exosomes to selected tissues.

Patent Status

This project is the subject of two patent applications and Isis would like to talk to companies interested in developing the commercial opportunity.

Request Further Information: Project Number 4096 - Novel RNAi Delivery Technology