GMP Cell Therapy and Vector Core

该计划项目资助（PO 1）探索通过在临床前研究和临床试验中采用脐带血干/祖细胞和T细胞的体外扩增来增强脐带血移植。所有四个项目将在研究过程中进行临床试验。这些依赖于GMP生产设施的可用性，用于制备细胞治疗产品和相关病毒载体。项目1和4将使用MD安德森癌症中心（MDACC）的药品生产质量管理规范（GMP）设施，项目2和3将依赖于细胞和基因治疗中心贝勒医学院（Baylor College of Medicine）的GMP设施和临床载体生产设施。位于上海市浦东新区和上海浦东新区的GMP设施已经运行了10多年。这两个机构的细胞处理设施在制备各种细胞产品方面具有相当丰富的经验，包括本申请项目所需的所有产品。这两个设施都由HEPA过滤器组成。10，000级空间分为多个细胞制备实验室、低温储存区、细胞分选和分析实验室、大型设备区和中央供应设施。这两个工厂都配备了良好的GMP条件下运行，广泛的文件系统，条形码，环境监测和质量保证，控制和改进计划。核心的其他组成部分是位于ESTA和MDACC的质量控制实验室，其对细胞产品（和载体）进行内部检测，并负责对药品生产质量管理规范进行常规监测;以及质量保证组，以确保符合GMP，并对生产和放行的所有方面提供独立的概述。临床载体生产设施也是核心的一部分，已经为地方、国家和国际研究生产了30多个临床级腺病毒载体和逆转录病毒载体。两个机构的GMP工作人员具有丰富的监管经验，这将有助于将实验室研究转化为临床试验。总之，GMP实验室核心是P01的重要组成部分，将为项目1、2、3和4中临床研究的实施提供必要服务。

This Program Project Grant (PO1) explores the enhancement of cord blood transplantation by employing ex-vivo expansion of cord blood stem/progenitor and T cells in preclinical studies and clinical trials. All four projects will undertake clinical trials during the course of the studies. These rely on the availability of a GMP manufacturing facility for the preparation of the cellular therapy products and associated viral vectors. Projects 1 and 4 will use on the Good Manufacturing Practice (GMP) Facility at MD Anderson Cancer Center (MDACC) and Projects 2 and 3 will rely on the GMP facility and the Clinical Vector Production Facility at the Center for Cell and Gene Therapy Baylor College of Medicine (BCM). The GMP Facilities at BCM and MDACC have been in operation for more than 10 years. The Cell Processing Facilities at both institutions have considerable experience in the preparation of a wide variety of cellular products, including all that would be required for the Projects in this application. Both facilities consist of HEPA filtered. Class 10,000 space divided into multiple cell preparation laboratories, low temperature storage areas, cell sorting and analysis laboratories, large equipment areas and central supply facilities. Both Facilities are well equipped to operate under GMP conditions, with extensive documentation systems, barcoding, environmental monitoring and quality assurance, control and improvement programs. Additional components of the Core are the Quality Control Laboratories at BCM and MDACC, which perform in-house testing of cellular products (and vectors), and are responsible for routine monitoring of Good Manufacturing Practices; and the Quality Assurance Group to ensure compliance with GMP and provides independent overview of all aspects of manufacturing and release. The Clinical Vector Production Facility at BCM, which is also a part of the Core, has produced more than 30 clinical grade adenovectors and retroviral vectors for local, national and international studies. The GMP staff at both institutions have extensive regulatory experience that will facilitate the translational of laboratory studies into clinical trials. In summary, the GMP Laboratory Core is a vital component of the P01 that will provide essential services to the implementation of the clinical studies in Projects 1, 2, 3 and 4.