CHARACTERIZATION & SAFETY TESTING OF CLINICAL RETROVIRUS

特征描述

• 批准号: 2664177
• 负责人: JEFFREY M OSTROVE
• 金额: $ 15万
• 依托单位: MICROBIOLOGICAL ASSOCIATES, INC.
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-04-01 至 2000-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2664177
• 关键词: Retroviridae; cell bank /registry; microorganism culture; microorganism resource /registry; tissue /cell culture; transfection /expression vector

The main objective of this contract project is to provide clinical grade retrovirus vector supernatant that will be used for targeting somatic human cells ex vivo in trials of gene therapy. This will involve the production of a master cell banks of the retrovirus producer lines, cryopreservation storage of such banks, in some cases production of working cell banks and cryopreservation storage of such banks, production of multi-liter quantities of clinical grade retrovirus supernatant and its storage by cryopreservation. An integral part of this objective is performance of a variety of safety testing on the cell lines which produce the retrovirus vector supernatant, the retrovirus supernatant itself, and on the human cells that are transduced with this retrovirus vector in culture. Such safety testing would include, but is not restricted to verification of the source of producer lines, detection of many types of contaminating pathogenic organisms, and detection of replication competent retrovirus. Such safety testing will be performed in a manner which will verify that the retrovirus supernatant and transduced cell products to be used in the clinical trial conform to the safety standards set by regulatory requirements of the United States Food and Drug Administration. One of the target diseases for such gene therapy trials is the inherited immune system deficiency known as chronic granulomatous disease (CGD). In the clinical trial for CGD the blood stem cells derived from patients with CGD will be exposed in culture to the clinical grade retrovirus provided by this contract. The actual culture and transduction of stem cells is not part of this contract project, but the safety testing of the transduced stem cell product is an objective of the contract project.

该合同项目的主要目标是提供临床级 将用于靶向人类体细胞的逆转录病毒载体上清液 基因治疗试验中的离体细胞。 这将涉及到生产 逆转录病毒生产系主细胞库的冷冻保存 储存此类细胞库，在某些情况下生产工作细胞库和 此类库的低温保存，多升的生产 临床级逆转录病毒上清液的数量及其储存 冷冻保存。 这一目标的一个组成部分是执行 对产生逆转录病毒的细胞系进行各种安全测试 载体上清液、逆转录病毒上清液本身以及人类 在培养物中用该逆转录病毒载体转导的细胞。 这样的 安全测试包括但不限于验证 生产线的来源，多种污染物的检测 病原生物，以及复制能力逆转录病毒的检测。 此类安全测试的执行方式将验证 逆转录病毒上清液和转导细胞产物用于 临床试验符合监管机构制定的安全标准 美国食品和药物管理局的要求。 中的一个 此类基因治疗试验的目标疾病是遗传性免疫系统 缺乏被称为慢性肉芽肿病（CGD）。 在临床上 CGD 试验将从 CGD 患者身上提取的血液干细胞 在培养物中暴露于由此提供的临床级逆转录病毒 合同。 干细胞的实际培养和转导不属于 这个合同项目，但是转导干细胞的安全性测试 产品是合同项目的目标。