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Commercial development of short interfering RNA-lipid nanoparticles (siRNA-LNP) for in vivo functional genomics

  • Project Leaders: Pieter Cullis, Marco Ciufolini
  • Institutions: University of British Columbia (UBC)
  • Budget: $747083
  • Program/Competition: Proof of Concept
  • Genome Centre(s): Genome British Columbia
  • Status: Closed

This project was a collaboration between UBC and Precision NanoSystems Inc. (PNI), a UBC spin-out company founded in 2010 based around microfluidic technology (the NanoAssemblr) that enables easy manufacture of sophisticated nanoparticles.

The project provided proof-of-concept for short interfering RNA-lipid nanoparticles (siRNA-LNP) as a research tool for in vivo functional genomics. The commercial product from this work (Reagent Kits) enable a rapid, simple and cost-effective alternative to knockout animals for the study of gene function and interactions in normal and diseased tissues. The Reagent Kit is designed to be used with the NanoAssemblr, enabling scientists to prepare siRNA-LNP that can be used for functional genomics studies in vivo, specifically in the liver.

PNI has placed 15 NanoAssemblr instruments in industry and academic research labs worldwide. Moreover, the company has attracted significant traction with the opportunity and business model around the Reagent Kits. This traction has enabled PNI to close an early seed-financing round that raised more than $400,000, and attracted key investors who bring additional business and commercialization expertise to the PNI team. PNI has targeted revenues of $175,000 from Reagent Kits arising from this research.

The activities and outcomes of this program are critical to the generation of new intellectual property, creation of new products and the generation of revenue in 2013.