A NOVEL ELECTROPORATION SYSTEM FOR GENE THERAPY WITH EPO

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We propose to use a proprietary TriGrid Electroporation System (TES) for the delivery and prolonged expression of erythropoietin (EPO). Recombinant forms of EPO are commercially available as treatment for anemias associated with bone marrow depletion from HIV treatment and chemotherapy, and chronic renal failure. However, on average, this treatment must be delivered three times a week. Electroporation of EPO into mouse muscle tissue by simple needle electrodes has been described by others to be effective in increasing the level of EPO and hematocrit in blood for up to 6 months, suggesting potential viability as an alternative to standard EPO therapies. We propose to demonstrate feasibility of TES in delivering a reporter gene and EPO into muscle tissue and the advantages TES has over current modes of EPO delivery, particularly in the sustained expression of EPO to stimulate red blood cell production. We expect the development of TES in the delivery of EPO will provide a more convenient and less expensive treatment for patients undergoing current EPO therapy. The future of TES may not only be for the delivery of genes for replacement therapy, but also to deliver DNA vaccines to stimulate host immune responses for HIV and cancer treatment. PROPOSED COMMERCIAL APPLICATIONS: The estimated sales of Epogen, a human recombinant form of erythropoietin (EPO) manufactured by Amgen Pharmaceuticals, are above $500 million annually. The development of a treatment which allows the body to manufacture its own erythropoietin (EPO) to stimulate red blood cell production as a response to anemia caused by disease therapy, is very enticing. By introducing and expressing the EPO gene in muscle using Ichor's electroporation array, the body would be able to produce endogenous EPO for a more extended amount of time and would decrease the number of treatments patients would require to treat their anemia, thus decrease the overall cost of treatment. The already high cost of care for AIDS and cancer patients often makes this treatment to improve their quality of life unaffordable.