Non-viral gene delivery agents

This technology is a stable, non-toxic, non-viral transfection agent based on positively-charged polymers composed of cyclopropenium ions.

Unmet Need: Stable, non-toxic, cationic polymer capable of crossing cell membranes

Gene therapy is a promising technology that allows therapeutics to modulate genetic expression by introducing, activating, or disabling genes. However, most cells have a bilayer membrane that blocks certain molecules, including nucleic acids, from entering the cell. Cellular transfection agents allow materials to be introduced into cells by disrupting this membrane; however, there is currently a paucity of compounds that can effectively bypass the membrane without causing cellular toxicity

The Technology: Polymer-based transfection agent that binds DNA and easily penetrates cell membranes

This technology describes the synthesis of various polymers incorporating cyclopropenium ions and their use as cellular transfection agents for gene delivery. Post-polymerization functionalization allows the incorporation of a positively-charged ion into the polymer structure in a highly controlled, selective, and predictable fashion. Due to the positive charge characteristics, this technology readily forms complexes with the negatively-charged phosphate backbone of DNA, enabling its use as a potent transfection agent. Additionally, the nature of the polymer and the cyclopropenium ion can be tuned to optimize transfection efficiency. As such, this technology presents a highly efficient and tunable method for delivery of genetic material into cells.

Applications:

• Delivery vehicle for gene therapy
• Transfection reagent for research applications
• Drug delivery

Advantages:

• Non-viral
• Stable
• Non-toxic
• Tunable chemical functionality
• Non-pH dependent
• Formal charge lies on carbon atoms, rather than heteroatoms

Lead Inventor:

Luis Campos, Ph.D.

Patent Information:

Patent Pending (WO/2018/035435)

Related Publications:

• Freyer JL, Brucks SD, Gobieski GS, Russell ST, Yozwiak CE, Sun M, Chen Z, Jiang Y, Bandar JS, Stockwell BR, Lambert TH, Campos LM. “Clickable Poly(ionic liquids): A materials platform for transfection.” Angew Chem Int Ed Engl. 2016 Sep; 55(40): 12382-12386.

• Yoon H, Dell EJ, Freyer JL, Campos LM, Jang WD. “Polymeric supramolecular assemblies based on multivalent ionic interactions for biomedical applications.” Polymer. 2014 Jan; 55(2): 453-564.

Tech Ventures Reference:

• IR CU16329

• Licensing Contact: Cynthia Lang