ONCOPREX® Nanoparticle Delivery System

Our oncology platform utilizes our non-viral ONCOPREX® Nanoparticle Delivery System. Using this system, plasmids containing tumor suppressor genes, which are deleted early in the development of cancer, are encapsulated within lipid nanoparticles and administered intravenously to the patient to re-express the deleted tumor suppressor genes. This platform, originally developed through collaborative research between the University of Texas MD Anderson Cancer Center and the National Institutes of Health, has been optimized to work with our initial product candidate, REQORSA® immunogene therapy.

Designed to Deliver Cancer-Fighting Genes Systemically

Our oncology platform consists of anti-cancer genes expressing DNA plasmids contained in non-viral lipid nanoparticles delivered intravenously. REQORSA utilizes the ONCOPREX® Nanoparticle Delivery System to encapsulate the TUSC2 gene in positively charged nanoparticles that bind to negatively charged cancer cells, and then enter the cancer cell through selective endocytosis, a process by which cells take in substances from outside the cell by engulfing them in a vesicle. The nanoparticles in our system differ significantly from liposomes historically used for drug delivery in that they are true particles encapsulating the therapeutic payload within a bilamellar lipid coat.

Safety and Efficacy

We have administered REQORSA to more than 50 patients in Phase 1 and 2 clinical trials using our systemic, proprietary, non-viral delivery system.

A Phase 1 clinical trial showed that systemic, intravenous therapy using the ONCOPREX Nanoparticle Delivery System was shown to selectively and preferentially target primary and metastatic tumor cells, resulting in clinically significant anticancer activity. The nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and providing extended half-life in the circulation.

Our earlier clinical trials have also shown that the ONCOPREX Nanoparticle Delivery System is well tolerated in humans and can safely deliver high therapeutic doses. We believe the ONC-001 clinical trial was the first systemic gene therapy clinical trial using a nanoparticle delivery system to deliver a tumor suppressor gene.