This invention utilizes a delivery system that allows for a biologically relevant moiety to be delivered to a desired cell by utilizing a transporting agent that binds two receptors expressed by that cell. This decreases the toxicity that is shown by the active compound by delivering it only to a targeted cell.
The current invention utilizes an endosome disrupting agent that is appended to one targeting moiety and a biologically-active agent that is appended to a second targeting moiety. The endosome disrupting moiety allows the biologically active elements on the second targeting moiety to be released from endosomes into the cytosol. The dual targeting technology can consist of aptamers, cholesterol like moieties, DNA, and/or proteins such as antibodies. The targeting nature of the appended moieties allow for therapeutic agents to be used without the side effects of killing normal, healthy cells.
The targeting agents can also be amended by adding imaging agents to the chemical moiety. This approach enhances the specificity of the compounds for a specific cellular target.
The current invention allows for the targeted uptake and cellular release of the therapeutic agents that can be used in chemotherapy, anti-viral therapy, cellular imaging, and other biological functions.
The current invention targets receptors such as those overexpressed in cancer cell lines. By selectively targeting these receptors, the exposure of therapeutic agents to diseased cells is increased.
By increasing the availability of the therapeutic agents to diseased cells, the overall dosage can be decreased. The decrease in dosage is allowed because of the increased efficiency in getting the agent to the desired target.
The current invention is advantageous when compared to current targeted therapy because it has a dual targeting system. Current targeted therapies target a single receptor which can also be expressed in healthy cells. By creating a separate moiety for a different target the efficacy of drug delivery is increased.
In addition to chemotherapeutic activity, the current invention can also be used as a delivery molecule for imaging agents and/or anti-viral agents.