FEASIBILITY ANALYSIS OF A NOVEL SYSTEM FOR GENE DELIVERY

Gene therapy strategies based on plasmid DNA (pDNA) delivery have been proposed for a variety of applications including vaccines and endogenous production of therapeutic proteins. Unfortunately, clinical implementation of these approaches has been hampered by the lack of clinically viable gene delivery systems. Recent studies suggest that in vivo etectroporation can dramatically improve the delivery of pDNA. However, the current methods and devices are inappropriate for widespread clinical application. Since lchor has already developed clinical electroporation technology for pharmaceutical delivery, the company is well positioned to develop the technique for pDNA delivery. A critical step is the development of safe, reliable, and effective means of administration. It must also be simple to apply and result in minimal discomfort for the patients. In light of these criteria, Ichor proposes to develop an integrated administration device to enable clinical implementation of electroporation mediated pDNA delivery. During Phase I, Ichor will demonstrate basic feasibility for its integrated pDNA administration system. The scope of the research includes design and development of a prototype unit and testing to verify safety and efficacy in small animals. Successful completion of Phase I will lead to development of devices appropriate for application in larger animals and eventually, humans.