Multi-functional Nanotherapeutics for Targeted Drug and Gene Therapy

Market Overview:

This multi-functional polymeric micelle nanocarriers serve as a platform technology for combinatorial delivery of multiple bioactive molecules for the treatment of pathological diseases or repair of injured tissues. With a growing population of cancer patients and the increasing quest for an effective cure, gene therapy represents a $100 billion cancer treatment market. The most commonly used treatment for cancer patients is chemotherapy. However, the efficacy of anticancer drugs has been limited by their toxic side effects in normal cells and drug resistance acquired by cancer cells. Current gene therapy methods involve nucleic acid therapeutics; however, the naked DNA/RNA used in these treatments are rapidly degraded by DNase/RNase in the blood or cellular lysosome and cannot pass through the cell membrane sufficiently due to its negative charge. To overcome these challenges, Clemson University researchers developed a multi-functional nanoparticle to be used as the drug/gene delivery vehicle for combinatorial therapy. This multi-functional polymeric micelle nanotherapeutic is capable of delivering multiple therapeutic agents within a single formulation and therapeutic intervention.


Application                                                                           Stage of Development                                                                     

Non-viral gene delivery;                                                        Validated Prototype; In vivo studies and rat studies completed

Tissue and organ specific cancer treatment,

neural regeneration          



• Allows for any hydrophobic drugs to be loaded in the nanoparticle core while any therapeutic nucleic acids can be simultaneously loaded in the shell, resulting in effective delivery of drugs and nucleic acid for the combinatorial therapy

• Improves efficiency with lower cytotoxicity in the presence of serum, providing an advantage over commercially available non-viral vectors

• Utilizes non-viral gene delivery, creating a safer delivery mechanism than traditional viral vector delivery


Technical Summary

This invention consists of polymeric micelle nanoparticles for targeted drug and nucleic acids delivery. Three key features of this successful nanoparticle include: a hydrophobic core that provides a reservoir for loading of the hydrophobic drug, targeting moieties for tissue/cell specific treatment, and a hydrophilic shell that delivers therapeutic nucleic acids. These features are achieved by the composition composed of an amphiphilic graft copolymer, poly (lactide-co-glycolide)-graft-polyethylenimine (PLGA-g-PEI) that spontaneously self-assemble in aqueous solution to form micelles. The hydrophobic PLGA core provides a reservoir for loading of hydrophobic drugs with limited water solubility, while the PEI hydrophilic shell can electrostatically bind nucleic acids such as plasmid DNA, antisense oligonucleotides, or siRNAs. The PEI shell contains primary amines to which a variety of targeting ligands can be covalently conjugated.  This Clemson University invention allows for the concomitant delivery of chemotherapeutic and MDR knockdown gene, overcoming drug resistance and increasing therapeutic efficiency. 


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Inventors:                        Jeoungsoo Lee


Application Type:            Utility


Serial Number:                14/966,614


CURF Reference:            2014-027


Patent Information:
For Information, Contact:
Chris Gesswein
Director of Licensing
Clemson University Research Foundation
Jeoung Soo Lee
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