Gene and Virus Therapy Program

To permit translation of a promising therapy from the research laboratory to a human clinical trial, the Food and Drug Administration (FDA) requires efficacy data, toxicological and pharmacological characterization of the drug, clinical grade drug manufacturing, a well-designed clinical trial protocol, and appropriate institutional approvals. The purpose of the Gene and Virus Therapy Support Resource (GVTSR) is to provide to Mayo Clinic investigators with promising virus-based therapies the resources and expertise in manufacturing and animal testing of recombinant viruses to meet these FDA requirements. The translation of new drugs, including promising virus-based therapies, from the cycles of preclinical research to clinical trials requires expertise and facilities not often found in an academic setting. Thus, the initiation of the clinical trial process can be significantly delayed or completely blocked for the academic researcher /clinician. The Mayo Clinic has established the necessary core resources on-site, the GVTSR, to translate gene and virus research at the Mayo Clinic to Phase l/ll clinical trials. The GVTSR has three components, the Viral Vector Production Laboratory (WPL), the Viral Toxicology Pharmacology Laboratory (VTPL) and Quality Assurance (QA), which work in collaboration with each investigator to fulfill the unique requirements of each project. The WPL 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 VTPL 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. The QA unit assures the quality of the procedures and results of the GVTSR. Over the past five years, the GVTSR has been directly involved in the translation of four new MCCC research projects to Phase I clinical trial and the support of four other Phase II clinical trials originally translated with GVTSR support .