ESTABLISHMENT OF A NATIONAL GENE VECTOR LABORATORY

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。描述（由申请人提供）：本申请建议在BRICOH建立一个两用NGVL“载体生产设施”。该设施将作为生产基于质粒DNA的载体的主要场所，在没有额外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。该工厂生产了FDA审查的质粒材料，已用于人类临床研究，目前有三种额外的质粒产品在生产中。员工在生产符合现行药品生产质量管理规范（cGMP）的人类临床用生物制剂方面具有丰富的经验和专业知识。总监、副总监和质量保证官具有生物技术行业的背景，拥有法规事务、产品和工艺开发、制造、质量体系和分子生物学、病毒学、血液学、细胞生物学和流式细胞术方面的专业知识。目前正在卫生组织办公室的资助下扩大现有进程。该设施按照cGMP运行，用于生产I/II期生物制剂，各种设施系统均符合已实施和正在进行的主验证计划。COH的生物医学和遗传学中心（CBG），其中拟议的NGVL将位于，目前生产重组单克隆抗体，质粒DNA，病毒载体和离体操作的细胞产品。

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.