Targeted Delivery of siRNA

The biological phenomenon of RNA interference (RNAi) has much promise for developing therapeutics to a variety of diseases. However, development of RNAi therapies remains mainly in preclinical stages largely because of difficulties in delivering small inhibitory RNAs (siRNA) and short hairpin RNAs (shRNA) into target cells. Although viral vector-based siRNA delivery systems have been widely used, their specificity and safety remain significant issues. Without a solution to these delivery problems, RNAi cannot fulfill its therapeutic promise.

Scientists at the National Institutes of Health, National Institute on Aging have developed new compositions and methods for delivering inhibitory oligonucleotides to cells in a targeted and efficient manner. The compositions and methods are based on utilizing a cell surface receptor targeting ligand, such as cytokine or chemokine, and a domain that binds an inhibitory oligonucleotide, to efficiently deliver the inhibitory oligonucleotide to the cell that expresses the cell surface receptor targeting ligand. Chemokine receptors are differentially expressed on various cells, including tumors; hence this technology allows targeting siRNA to aberrant cells. Gene silencing can also be achieved in variety of immune cells by targeting cytokine receptors. This technology has great potential for developing into a safe and effective means of delivering therapeutic siRNAs.

Potential Commercial Applications: Competitive Advantages:
  • Leukemia
  • General oncology
  • Autoimmune disease
  • Simple method for linking siRNA to polypeptides to create non-covalent or covalent complexes
  • In vivo targeted delivery of inhibitory RNAs into cells rather than systemically
  • Delivery of multiple inhibitory RNAs to target multiple genes
  • Long term repression of target gene expression through RNAi phenomenon

Development Stage:
  • In vitro data available
  • In vivo data available (animal)
  • In situ data available


Bira Arya (NIA)  ➽ more inventions...

Purevdorj Olkhanud (NIA)  ➽ more inventions...

Juan Espinoza (NIA)  ➽ more inventions...

Intellectual Property:
U.S. Pat: 8,703,921 issued 2014-04-22
U.S. Pat: 9,415,116 issued 2016-08-16
US Application No. 12/988,148
US Application No. 15/204,789
US Application No. 14/220,726

Biragyn A, et al. PMID 23603860

Collaboration Opportunity:

The National Institute on Aging, a division of NIH, seeks statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize chemokine-based siRNA/shRNA technology for treatment of cancers and autoimmune diseases, e.g., to control expression of immunomodulatory cytokines and other factors that facilitate tumor escape. In addition, NIA would be open to entering into collaborative research relationships whereby resources such as intellectual property can be pooled and applied to the development of therapies with respect to leukemia and other oncological conditions. Please contact John D. Hewes, Ph.D. at 240-276-5515 or for more information.

Licensing Contact:
Surekha Vathyam, Ph.D.
Phone: 240-276-6865

OTT Reference No: E-051-2008/0
Updated: Oct 10, 2017