Chemical Modification of Polysaccharides for Efficient Delivery of RNA and DNA Therapeutics
Background Nucleic acid-based therapeutics are rapidly becoming the next generation of drugs, offering promising potential for a variety of diseases. Antisense and small interfering RNAs (siRNA) "knock down" (dampen or decrease) the expression of specific, disease-related gene targets and have applications in cancer, cardiovascular, metabolic, and neurological diseases. On the other hand, DNA is used for gene therapy or as vaccines against infectious diseases and cancers. The challenges of using nucleic acids as drugs in patients include susceptibility to nucleases in the tissue, rapid clearance from blood, inefficient cellular uptake, and targeting to the appropriate cells and sub-cellular compartments.
UT Austin researchers have developed an invention to deliver RNA or DNA drugs to cells in vitro and in vivo. The technology demonstrates a favorable efficiency and toxicity profile compared to competitive technologies on the market.
Invention Description Researchers at The University of Texas at Austin have developed an original method to overcome the limitations for intracelllular delivery that are found with polysaccharides such as chitosan. This is achieved by the conjugation of secondary and tertiary amines with a small molecular modifier to biocompatible polysaccharides (i.e., sugar-type polymers). These modified polysaccharides can be used to produce nanoparticles with nucleic acids and other molecules for enhanced intracellular delivery with minimal cytotoxicity.
This novel invention can be used to improve delivery of gene therapy and DNA-based vaccines for numerous diseases. It can also be used for delivery of siRNA and miRNA or antisense oligos for disease-specific and organ-specific gene knockdown. The nanoparticles are effective through a variety of routes including intravenous, intranasal, and oral routes. Interested companies will benefit from the fact that the researchers have demonstrated the potential of this technology through in vitro and in vivo studies in mice.
Enhanced delivery of nucleic acids (RNA and DNA) in vitro and in vivo Biocompatibility Enhanced solubility of the polysaccharide, leading to easier formulation process and scale-up Effectiveness through various routes of administration
Market Potential/Applications Companies based in the pharmaceutical, chemical, nucleic acid, or nanotechnology industries will have interest in this technology.
Development Stage Proof of concept
IP Status One PCT patent application filed
UT Researcher Krishnendu Roy, Ph.D., Biomedical Engineering, The University of Texas at Austin Sudhir Pai Kasturi, Biomedical Engineering, The University of Texas at Austin Bilal Ghosn, Biomedical Engineering, The University of Texas at Austin
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