CORE--GENE & VIRAL THERAPY

核心基因

基本信息

批准号：
7618238
负责人：
Mark J Federspiel
金额：
$ 21.01万
依托单位：
MAYO CLINIC ROCHESTER
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/7618238
关键词：
Advisory Committees Animal Model Biological Products Clinic Clinical Clinical Protocols Clinical Trials Collaborations Consultations Data Development Discipline Drug Industry Equipment Figs - dietary Genes Human Resources Investigation Investigational New Drug Application Laboratories New Agents Other Therapy Pharmaceutical Preparations Pharmacology and Toxicology Phase Procedures Process Production Purpose Quality Control Recombinant DNA Research Ethics Committees Research Personnel Resource Sharing Resources Safety Testing Therapeutic Training Translating Translations United States Food and Drug Administration United States National Institutes of Health Viral Viral Genes Viral Vector Virus Work animal care authority design gene therapy manufacturing facility manufacturing process novel pre-clinical preclinical study programs quality assurance small molecule vector

项目摘要

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.

这种共享的资源满足了促进从我们的基本基础研究中出现的有前途的基因和病毒疗法的转化，以进入临床前和临床试验。它旨在满足食品药品监督管理局的所有严格要求，包括提供足够的临床级材料来确定候选剂的疗效，毒理学和药理特征的能力，以及具有监管过程的所有步骤的专业知识。实现这一目标的专业知识和设施经常在学术环境中找到，从而在引入新的创意和有前途的疗法的关键过程中导致延误或实际障碍，除了由制药行业得到很好支持的小分子药物。基因和病毒疗法支持资源具有三个组件，分别是病毒媒介生产实验室，毒理学药理学实验室和质量保证，它们与每个研究人员合作以无缝的方式合作，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，用于使用良好的制造实践（GMP）的大规模病毒生产，过程开发以及临床级产品的生产。毒理学药理学实验室拥有专门的设备和受过训练的人员，用于在动物模型中使用良好的实验室实践（GLP）在AAAALAC批准的Mayo梅奥诊所动物护理设施中设计和进行新型生物学产品的毒理学和药理特征。已经成立了一个质量保证单元，以确保基因和病毒疗法的过程和结果的质量支持资源。