A Novel Electroporation System for Gene Therapy with EPO

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(Provided by the applicant): We propose to develop a clinical non-viral therapy for treatment-induced anemia in AIDS/HIV and chemotherapy patients using a proprietary TriGrid electroporation system (EPT). Anemia is currently treated with repeated administration of recombinant erythropoietin (EPO), which is expensive and not readily available to these patients due to the already elevated cost of treatment for their primary disease. However, correction of this anemia will not only improve quality of life, but may also have the potential to improve these patients' probability of survival. Thus, development of a more cost effective and equally efficacious method of EPO therapy such as EPT-delivery of an Epo plasmid, is clearly justified.Ichor's Phase I work has shown the feasibility of delivering DNA plasmids containing genes such as erythropoietin (Epo), into skeletal muscle of small and large rodents using EPT. Phase II work will involve a comprehensive dose response analysis, and characterization of treatment toxicity and vector biodistribution. Using a clinically relevant plasmid construct, initial experiments will include testing in normal and diseased rodent models to evaluate biologic response and establish efficacy and dosing. A large animal study will be conducted to verify dose scale up and identify potential toxicity and safety issues associated with the therapy. PROPOSED COMMERCIAL APPLICATION: The estimated sales of Epogen and Procrit, recombinant forms of erythropoietin (EPO), are above $2 billion annually. The development of a treatment which allows the body to manufacture its own EPO to stimulate red blood cell production in response to treatment- induced anemia, is very enticiting. By introducing and expressing the EPO gene in muscle using Ichor's electroporation technology, the body would be able to produce EPO for a more extended amount of time and would significantly decrease the number of treatments patients would require to treat their anemia, thus decreasing 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 and perhaps their likelihood for survival, unaffordable.