The application of promising gene therapy strategies to clinical trials can occur only after extensive preclinical testing of the substrates used for gene transfer which often are recombinant viruses. The development and production of these clinical reagents must occur in highly specialized laboratories that can handle biohazardous material and can function in accordance with Food and Drug Administration (FDA) regulations under Good Manufacturing Practices (GMP). We have established a biohazard GMP facility called the Human Applications Laboratory dedicated to the production of gene transfer reagents for clinical trials. This newly renovated facility is housed in 2,500 sq ft of laboratory space and has 5 self-contained manufacturing modules, each capable of the production and isolation of different viruses.
The specific aims of the laboratory will be to provide the facilities and expertise to: (1) Establish protocols and standard operating procedures (SOP) for all aspects of human applications of gene therapy that will facilitate FDA certification and assure safety of clinical trials proposed by all investigators; (2) Develop and produce recombinant retroviruses and recombinant adenoviruses that are acceptable to the FDA and Recombinant DNA Advisory Committee (RAC); (3) Isolate, cultivate, genetically modify and harvest human cells for preclinical studies documenting the feasibility and safety of the therapeutic approach; (4) Return to the investigator genetically modified cells in a form suitable for direct administration to patients.
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