The Genprex immunogene therapy platform consists of anti-cancer genes encapsulated in nanovesicles delivered intravenously. The Oncoprex TUSC2 gene is encapsulated in a positively charged nanovesicle that binds to actively replicating (and, therefore, negatively charged) cancer cells, and then enters the cancer cell through selective endocytosis.
The particle size is small enough to allow Oncoprex to cross tight barriers in the lung, but large enough to avoid accumulation or clearance in the liver, spleen and kidney. The cationic charge of the nanovesicle targets cancer cells, and direct nanovesicle fusion avoids target cell endocytosis. A Phase I clinical trial showed that intravenous Oncoprex therapy was proven to selectively and preferentially target primary and metastatic tumor cells, resulting in clinically significant anticancer activity. The nanovesicles are non-immunogenic, allowing repetitive therapeutic dosing and providing extended half-life in the circulation.
Our nanovesicle delivery platform is applicable to delivery of a range of therapeutic and prophylactic plasmid DNAs and RNA interference constructs. The nanovesicle manufacturing methods we and our collaborators have developed have been optimized and are useful for a wide array of disease treatments. Our nanovesicles are clinically proven to deliver molecular kinase inhibitors effectively. Clinical outcomes demonstrated that the delivery system used in Oncoprex is well tolerated in humans and can safely deliver high therapeutic doses.