CORE--GENE & VIRAL THERAPY

这种共享资源填补了促进从我们的基础实验室研究中出现的有前途的基因和病毒疗法进入临床前和临床试验的关键需求。它的设计符合食品和药物管理局的所有严格要求，包括能够提供足够的临床级材料来确定候选药物的功效、毒理学和药理学特征，以及监管过程的所有步骤的专业知识。在学术环境中往往找不到实现这一目标的专业知识和设施，这导致在引入新的创造性和有前途的治疗方法的关键过程中出现延误或实际阻碍，而制药行业良好支持的小分子药物除外。基因和病毒治疗支持

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.