CORE--GENE & VIRAL THERAPY

这种共享资源满足了促进从我们的基本实验室研究中出现的有前途的基因和病毒疗法的翻译进入临床前和临床试验的关键需求。其设计符合美国食品药品监督管理局的所有严格要求，包括能够提供足够的临床级材料以确定候选药物的疗效、毒理学和药理学表征，以及监管过程所有步骤的专业知识。实现这一目标的专业知识和设施在学术环境中并不常见，导致在引入新的创造性和有前途的疗法的关键过程中出现延误或实际障碍，而不是制药行业大力支持的小分子药物。基因和病毒治疗支持 资源有三个组成部分，病毒载体生产实验室，毒理学药理学实验室和质量保证，以无缝的方式与每个研究人员合作，以满足每个项目的独特要求。病毒载体生产实验室拥有专门的设施和人员，用于大规模病毒生产、工艺开发和使用药品生产质量管理规范（GMP）生产临床级产品。毒理学药理学实验室拥有专业设备和经过培训的人员，可在AAALAC批准的马约诊所动物护理机构中使用药物非临床研究质量管理规范（GLP）设计和进行动物模型中新型生物制品的毒理学和药理学表征。成立了质量保证部门，以确保基因和病毒治疗的程序和结果的质量。 支持资源。

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.