ESTABLISHMENT OF A NATIONAL GENE VECTOR LABORATORY

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。描述(由申请人提供)：本申请建议在金砖四国建立一个两用NGVL“媒介生产设施”。该设施将作为生产基于质粒DNA的载体的主要场所，并且在不需要额外的NGVL资源的情况下，为基于腺病毒的载体提供后备生产能力和能力。该设施已经生产了FDA审查的质粒材料，已用于人类临床研究，目前还有另外三种质粒产品在生产中。员工在生产符合当前良好制造规范(CGMP)的生物制品方面拥有丰富的经验和专业知识，供人类临床使用。主任、副主任和质量保证主任具有生物技术行业的背景，具有监管事务、产品和工艺开发、制造、质量系统和分子生物学、病毒学、血液学、细胞生物学和流式细胞术方面的专业知识。在卫生部的资助下，目前正在扩大现有流程。该设施按照cGMP运行，用于生产第一阶段/第二阶段的生物制品，各种设施系统受到已实施的和正在进行的主验证计划的约束。拟议中的NGVL将设在COH的生物医学和遗传学中心(CBG)，该中心目前生产重组单抗、质粒DNA、病毒载体和体外操纵的细胞产品。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. DESCRIPTION (provided by applicant): This application proposes the establishment of a dual-purpose NGVL "vector production facility" at the BRICOH. The facility will serve as the primary site for production of plasmid DNA-based vectors and, without additional NGVL resources, provide back-up production capacity and capability for adenovirus based-vectors. The facility has produced FDA-reviewed plasmid material that has been used in human clinical studies and currently has three additional plasmid products in production. The staff has significant experience and expertise in the production of current Good Manufacturing Practices (cGMP)-compliant biologics for human clinical use. The Director, Associate Director and QA Officer have backgrounds in the biotechnology industry with expertise in regulatory affairs, product and process development, manufacturing, quality systems and molecular biology, virology, hematology, cell biology and flow cytometry. Scale-up of current processes is under way with funding from the COH. The facility is operated in accordance with cGMP for production of Phase I/II biologics and the various facility systems are subject to an implemented and ongoing Master Validation Plan. The COH's Center for Biomedicine and Genetics (CBG), where the proposed NGVL will be located, currently produces recombinant monoclonal antibodies, plasmid DNA, viral vectors and ex-vivo manipulated cell products.