Targeted Therapy for Neoplastic Diseases

Identification of effective treatments is urgently needed to provide patients with better options in treating ovarian cancer, which afflicts 25,580 Americans annually. Ovarian cancer is caused by genetic alternations; therefore, gene therapy offers a promising approach for the treatment of ovarian cancer. Transgenex Nanobiotech (TGN) Inc has developed a nanoencapsulated transposon system (NTS) for treatment of cancers using its proprietary drug candidate, TGN208. In phase I, TGN has identified nanoparticles that can specifically target to ovary, and optimized the nanotransposon system and identifieded the antineoplastic mechanisms of TGN208. Herein, in phase II TGN proposes to further its R & D on this NTS to pursue the safety and efficacy studies in mouse and rhesus monkey models. The specific technical objectives are: aim #1) to develop DNA constructs for enhanced integration of transposase–tagged transgene and ovary specific expression, aim #2) to identify the most effective transgene for ovarian cancer in mouse models and test the safety, toxicity and pharmacokinetics of the transgene expression and aim #3) to evaluate the untoward effects of TGN208 NTS in nonhuman primate model. These studies are expected to lead the filing of an IND and progress to phase-III involving human studies.