GENE THERAPY DEVICE FOR TREATMENT OF HEMOPHILIA B

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The goal of this proposal is to develop a novel gene therapy delivery device for the 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 the first steps to develop 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 optimize the isolation of high yield, pure human myoblasts. Specific Aim 2 plans to develop a canine FIX retroviral vector system and optimize transduction of human myoblasts in preparation for Phase Il preclinical dog trials. Specific Aim 3 plans to optimize the transduction of human myoblasts with an already existing human FIX retroviral vector. Specific Aim 4 plans to optimize both FIX transduced myoblast packing into hollow fibers and FIX secretion rates. The successful completion of these studies sets the stage for Phase Il to scale up these cells in a cell cartridge containing the necessary number of hollow fibers containing transduced human muscle cells and implant this device in a large animal model. This approach may be the key enabling technology for the gene therapy industry, providing a safe means to introduce an implantable and retrievable device to introduce gene products into the systemic circulation of an individual with a genetic disorder. PROPOSED COMMERCIAL APPLICATIONS: This proposal is the first step in the development of an implantable device into the systemic circulation for the delivery of proteins via gene therapy to treat genetic diseases. This plan is focused on the treatment of Hemophelia B, a devastating and costly (over a billion dollar market) genetic disease characterized by spontaneous internal bleeding. This approach could be the enabling technology for the gene therapy industry.