This shared resource fills a critical need to facilitate the translation of promising gene and viral therapies emerging from our basic bench investigations to enter into preclinical and clinical trials. It has been designed to meet all the rigorous requirements of the Food and Drug Administration, including the ability to provide adequate clinical grade material to determine the efficacy, toxicological, and pharmacological characterization of the candidate agent, as well as expertise with all steps of the regulatory process. The expertise and facilities to achieve this are not often found in an academic setting, resulting in delays or actual blocks in the critical process of introducing new creative and promising therapies, other than small molecule drugs that are well supported by the pharmaceutical industry. The Gene and Virus Therapy Support Resource has three components, the Viral Vector Production Laboratory, the Toxicology Pharmacology Laboratory and Quality Assurance, which work together in a seamless manner in collaboration with each investigator to fulfill the unique requirements of each project. The Viral Vector Production Laboratory has the dedicated facilities and personnel for large-scale virus production, process development, and the manufacture of clinical grade product using Good Manufacturing Practices (GMP). The Toxicology Pharmacology Laboratory has specialized equipment and trained personnel to design and conduct toxicological and pharmacological characterization of novel biological products in animal models using Good Laboratory Practices (GLP) in the AAALAC-approved Mayo Clinic animal care facilities. A Quality Assurance unit has been formed to assure the quality of the procedures and results of the Gene and Virus Therapy Support Resource.
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