This technology is a stable, non-toxic, non-viral transfection agent based on positively-charged polymers composed of cyclopropenium ions.
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
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.
Patent Pending (WO/2018/035435)
IR CU16329
Licensing Contact: Cynthia Lang