This core will function as a shared resource representing about 25% of the activity of the Gene Therapy Vector Production Laboratory which is part of the Baylor College of Medicine Center for Gene Therapy. The purpose of the core will be to enable the production of gene therapy vectors for testing in human clinical trials. A core is necessary for this function due to the high cost of the infrastructure and expertise necessary to satisfy FDA requirements for the approval of biologicals in clinical trials. The physical characteristics of the facility will help assure sterile vector production. These include seamless flooring, epoxy resin wall and ceiling covering, positive pressure air flow HEPA filtered air, HEPA filtered biosafety hoods, certified equipment and a gowning/airlock secured double door entrance room. All equipment and supplies used at the facility will be approved for the production of biologicals used in human trials. This includes certification by the suppliers, quality assurance by the facility, and standard operating procedures (SOPs) for the receipt, inventory, expiration and disposal of all material. All operations and procedures in the laboratory will be governed by SOPs approved by the Center for Biologics Evaluation and Review (CBER). Personnel involved in the production of vectors will be trained in Good Laboratory Practices (GLP) and Good Manufacturing Practices (GMP) as required by statute. The Core will be responsible for the production of master cell banks and working cell banks necessary for the production of the vectors at sufficient scale under controlled conditions to allow their use in Phase I clinical trials. Adenovirus vectors will be grown in the helper human cell line 293. The Gene Therapy Vector Production Laboratory will also produce master cell banks for retroviral packaging cell lines, although this will only be a small component of the activities for this Program Project. The Core will be responsible for rigorous quality control to ensure the safety of the materials that will be administered to the patients and to ensure the safety of the personnel involved in the production of the vectors. This will involve detailed record keeping, in- house quality assurance and the oversight of subcontracted quality control tests. The operational director of the laboratory will be responsible for personnel training and compliance with FDA requirements. He has scientific interests in viral vector development and has been trained in the procedural requirements for production of biologicals for the enactment of investigational new drug (IND) applications. He will be an important resource for all independent investigators initiating clinical protocols, as a consultant in the early phases of the project and in the preparation and presentation of materials to the NIH Recombinant DNA Advisory Committee and FDA.
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