GENE THERAPY DEVICE FOR TREATMENT OF HEMOPHILIA BETA

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DESCRIPTION (Unedited Applicant's Abstract): The goal of this proposal is to develop a novel gene therapy delivery device for treatment of the genetic disorder. Hemophilia B. a devastating and costly (over a billion dollar market) disease characterized by spontaneous internal bleeding. The planned experiments will provide a cell therapy delivery vehicle for implantation into a large vessel of an animal. and eventually, a patient suffering from this genetic defect. Specific Aim 1 plans to test two IVD prototype fabrications for the ability to support adequate myoblast cell number and FIX secretion rates to achieve clinically significant FIX levels. Specific Aim 2 plans to optimize the in vitro cFIX production rates of the selected D/D prototype. Specific Aim 3 plans to introduce this IVD prototype with autologous canine myocytes transduced with hFIX gene into a normal dog where it will be secured in the vena cava by a Greenfield filter anchor (GFA). The hemocompatibility of this device within the vena cava, and myocytes viability will be assessed over various periods of time (up to 6 weeks). Specific Aim 4 plans to test the cFIX production rates and biological activity of cFIX transduced autologous canine myoblasts in the cell IVD prototype in a Hemophilia B dog model. The successful completion of these studies will provide 'proof of concept' for utilization of the IVD as a gene therapy' treatment of hemophilia B. The ultimate goal is to develop a novel cell and gene therapy delivery system (comprised of a cell-filled IVD anchored by means of a Greenfield filter) that is introducable and retractable via a percutaneous catheter insertion through the femoral vein into the inferior vena cava. The IVD may be used to deliver any desired compound via cell or gene therapy. This approach may be the key enabling technology for both the gene therapy and cell therapy industries, providing a safe means to implant a retrievable device to introduce gene products directly into the systemic circulation of an individual with a genetic disorder. PROPOSED COMMERCIAL APPLICATION: Not Available