ESTABLISHMENT OF A NATIONAL GENE VECTOR LABORATORY

国家基因载体实验室的建立

• 批准号: 7286405
• 负责人: LARRY A COUTURE
• 金额: $ 51.1万
• 依托单位: BECKMAN RESEARCH INSTITUTE/CITY OF HOPE
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-09-30 至 2007-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7286405
• 关键词: ESTABLISHMENT; NATIONAL; GENE; VECTOR; LABORATORY

DESCRIPTION: State the application's broad,TffTt|-term objectives and specific aims, making reference^Fthe health relatedness of the project. Describe concisely the research design and methods for achieving these goals. Avoid summaries of past accomplishments and the use of the first person. This description is meant to serve as a succinct and accurate description of the proposed work when separated from the application. If the application is funded, this description, as is, will become public information. Therefore, do not include proprietary/confidential information. DO NOT EXCEED THE SPACE PROVIDED. This application proposes the establishment of a dual-purpose NGVL "vector production facility" at the Beckman Research Instituteof the City of Hope (COH). The facility will serve as the primary site for production of plasmid DNA- based vectors and, without additionalNGVL 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 additionalplasmid products in production. The staff has significant experience and expertise in the production of cGMP-compliant biologies 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/I1 biologies 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. The CBG is a purpose-built, two-story 20,000 sq. ft. building on the COH campus with approximately 9,000 sq. ft. of classified manufacturing space. The remaining space includes the departmental offices. QC labs and facility systems. The CBG staff completely manages and operates this $12-milHon facility. The NGVL support requested here will provide the resources to produce up to 15 clinical grade plasmid products and associated master cell banks per year plus a capacity of up to 3-4 adenoviral vector products per year. The COH is currently funding the establishment of 293 master and working cell banks that will be made available for NGVL production activities. All activitieswithin the CBG are monitored and documented through a thorough Quality System according to cGMP. The CBG supports the translation of academic-based technologies into the clinical setting and has made a significant commitment to the facility to support the development and production of recombinant proteins, viral and non-viral vectors, vaccines and ex-vivo manipulated cell products for phase I/II clinical studies. PERFORMANCE SITE(S) (organization, city, state) Beckman Research Institute of the City of Hope 1450 East Duarte Road Duarte, CA 91010-3000 KEY PERSONNEL. See instructions on Page 11. Use continuation pages as neededto provide the required information in the format shown below. Name Organization Role on Project Larry A. Couture, Ph.D. Beckman Research Institute of COH Director - PI David L. DiGiusto, Ph.D. Beckman Research Institute of COH Associate Director - Co-Pi PHS 398 (Rev. 4/98) Page 2 BB Number pages consecutively at the botton throughout the application. Do not use suffixes such as 3a, 3b. CC Principal I^fcjgator/Program Director (Last, first, middle): Type the name of the principal investigator/program director at the top of each printed page and each^Pitinuation page. (For type specifications, see instructions on page 6.) RESEARCH GRANT

描述：说明应用程序的广泛目标和具体目标，并参考项目的健康相关性。描述 简明地阐述了实现这些目标的研究设计和方法。避免总结过去的成就和使用第一人称。这个描述 与应用程序分开时，旨在作为对拟议工作的简洁而准确的描述。如果申请获得资助，此说明， 照原样将成为公共信息。因此，请勿包含专有/机密信息。请勿超出所提供的空间。 该申请建议在 Beckman 建立一个双用途 NGVL“载体生产设施” 希望之城研究所 (COH)。该设施将作为生产质粒 DNA 的主要场所 基于载体，无需额​​外的NGVL资源，即可提供备用生产能力和能力 基于腺病毒的载体。该设施生产了经 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正在生产。员工拥有丰富的经验 以及生产人类临床使用的符合 cGMP 的生物制品方面的专业知识。董事、副董事 和质量保证官员拥有生物技术行业背景，在监管事务、产品和流程方面拥有专业知识 开发、制造、质量体系和分子生物学、病毒学、血液学、细胞生物学和流程 细胞计数术。在 COH 的资助下，目前工艺的规模正在扩大。该设施运营于 符合生产 I/I1 期生物制剂的 cGMP，并且各种设施系统均受到 已实施并正在进行的主验证计划。 COH 的生物医学和遗传学中心 (CBG) 拟议的 NGVL 将落户，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。 CBG 是 COH 园区内一栋专门建造的两层建筑，面积 20,000 平方英尺 拥有约 9,000 平方英尺的分类制造空间。剩余空间包括部门 办公室。 QC 实验室和设施系统。 CBG 员工完全管理和运营这个耗资 1200 万美元的设施。这 此处请求的 NGVL 支持将提供生产多达 15 种临床级质粒产品的资源， 每年相关的主细胞库加上每年多达 3-4 个腺病毒载体产品的容量。 COH 是 目前正在资助建立 293 个主细胞库和工作细胞库，供 NGVL 使用 生产活动。 CBG 内的所有活动均通过全面的质量体系进行监控和记录 根据cGMP。 CBG 支持将学术技术转化为临床环境，并已 对该设施做出了重大承诺，以支持重组蛋白、病毒的开发和生产 用于 I/II 期临床研究的非病毒载体、疫苗和离体操作细胞产品。 绩效站点（组织、城市、州） 希望之城贝克曼研究所 东杜阿尔特路1450号 杜阿尔特, CA 91010-3000 关键人员。请参阅第 11 页上的说明。根据需要使用后续页面以如下所示的格式提供所需信息。 名称 组织在项目中的角色 拉里·A·库图尔博士COH贝克曼研究所所长-PI 大卫·L·迪朱斯托博士COH 贝克曼研究所副所长 - Co-Pi PHS 398（修订版 4/98）第 2 页 BB 在整个应用程序的底部连续编号页码。请勿使用 3a、3b 等后缀。 CC校长I^fcjgator/项目总监（最后、第一个、中间）： 在每个打印页和每个 ^Patinuation 页的顶部键入主要研究者/项目主管的姓名。 （有关类型规格，请参见 第 6 页上的说明。） 研究资助