Case Number 103020 - A Family of Therapeutic Delivery Systems

Contact: Jill Uhl
Phone: 513-558-5621

Description:  The present invention is in the fields of molecular biology, biochemistry and pharmaceuticals. In general, the invention provides compositions for the cellular delivery of nucleic acids, polypeptides and/or molecular complexes comprising nucleic acids and polypeptides, and methods of making and using such compositions. The present invention provides a new class of non-viral transduction vectors that can be used for both in vivo and in vitro applications. This includes unique polycationic polymers that can associate with many suitable bioactive molecules, including proteins and other compounds that posses multiple cationic sites. The polymer can act as a delivery vehicle for the associated bioactive molecule, in vivo or in vitro, to the cells of interest for the bioactive molecule. In one embodiment, the present invention provides for a new series of polyamides for use as gene delivery agents. Also disclosed are methods of using the polymers to bind products, e.g., oligonucliotides, and facilitate cellular uptake. In one embodiment, the invention provides for the in vitro delivery of plasmid DNA into cells. The present also provides for the use of these polymers for the delivery of a nucleic acid is biologically active into a cell.
The polymers are nontoxic and suitable for (highly efficacious ) drug delivery, efficient intracellular gene delivery, as well as delivery of DNA, RNA, SiRNA and oligonucleotides. One family of polymers is completely biodegradable, yielding glucose and an oligoamine monomer that is similar in chemical structure to that found in the body. The polymers are suitable for noncovalent attachment of targeting agents for cell-type specific delivery.
Areas of Application
Both the polymers and the oligonucleotide decoys have clinical applications, either separately or in combination (i.e., with the polymer delivering the decoys into cells). The polymers also have applications as reagents for in vivo and in vitro delivery of bioactive molecules. Favorable in vitro test results have been achieved in a variety of primary cell lines. The oligonucleotides, both separately and with the polymers as a delivery vehicle, are being studied for treatment of myocardial ischemia/reperfusion.

The following three PCT Applications have been filed:
  1. Polyamides and Polyamide Complexes and Methods of Use - Inventor: Theresa M. Reineke
  2. Oligonucleotide Decoys and Methods of Use - Inventor: W. Keith Jones
  3. Polyamides and Polyamide Complexes for Delivery of Oligonucleotide Decoys - Inventors: Theresa M. Reineke and W. Keith Jones