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Virus-Like Particles Mediated Protein and RNA Delivery

Posted Jan 10 2013 7:00pm

Description of Invention:
The invention is directed to novel virus-like particles (VLPs) that are capable of binding to and replicating within a target mammalian cell, including human cells. The claimed VLPs are safer than viral delivery because they are incapable of re-infecting target cells. The present VLPs can optionally comprise inhibitory recombinant polynucleotides, such as microRNA, antisense RNA or small hairpin RNA, to down regulate or turn off expression of a particular gene within the target cell. Alternatively, recombinant polynucleotides packaged within VLPs can comprise a gene encoding a therapeutic protein so as to enable expression of that protein within the target cell. Specifically, VLPs of the invention are composed of an alphavirus replicon that contains a recombinant polynucleotide, a retroviral gag protein, and a fusogenic envelope glycoprotein.

While the claimed VLPs have a variety of applications, therapeutic uses of the VLPs include directing antibody synthesis and converting cancer cells into antigen presenting cells. Additional applications include using VLPs to induce fast (approx. 3-4 hrs) and high levels of protein production in mammalian cells.

Applications:
  • Delivery of microRNA and small hairpin RNA to reduce express of targeted genes in a human cell
  • Delivery of coding RNA for robust expression in mammalian systems
  • Direct antibody production by in vivo injection of replicons (no antigen purification)
  • Therapeutic applications


Advantages:
  • High level (~million copies per cell) of RNA production/synthesis within target cell
  • Fast expression (approx. 3-4 hrs compared to 1-2 days) following VLP introduction into target cells
  • Obviates need to use expensive antigen purification for proteins or antigens produced inside target cells


Development Status:
  • Pilot
  • Pre-clinical
  • In vitro data available
  • In vivo data available (animal)


Inventors:
Deb K Chatterjee (NCI)
Stanislaw J Kaczmarczyk (NCI)


Patent Status:
HHS, Reference No. E-264-2011/0
US, Application No. 61/615,687 filed 26 Mar 2012


Collaborative Research Opportunity:
The National Cancer Institute is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize Novel Delivery of Packaged RNA to Mammalian Cells. For collaboration opportunities, please contact Kevin Brand at brandk@mail.nih.gov .


For Licensing Information Please Contact:
Lauren Nguyen-Antczak Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Email: nguyenantczakla@mail.nih.gov
Phone: 301-496-7057
Fax: 301-402-0220


Ref No: 2513

Updated: 01/2013

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