临床级逆转录病毒载体储备液在细胞工厂中生产的包装细胞清除率的扩大生产规模及验证。
Production scale-up and validation of packaging cell clearance of clinical-grade retroviral vector stocks produced in cell factories.
作者信息
Przybylowski M, Hakakha A, Stefanski J, Hodges J, Sadelain M, Rivière I
机构信息
Gene Transfer and Somatic Cell Engineering Facility, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
出版信息
Gene Ther. 2006 Jan;13(1):95-100. doi: 10.1038/sj.gt.3302648.
The clinical implementation of gene therapy requires large-scale production of viral vector stocks (VS) derived from packaging cell lines. Upon scaling-up, maintenance of high viral titers and filtration of the VS become significantly challenging. Thus, production schemes amenable to straightforward validation must be developed. To this end, we have established a semi-closed process to manufacture batches of 7 l or more of clinical-grade oncoretroviral VS using 10-tray Cell Factories. Using a peristaltic pump, the VS are collected on 3 consecutive days, filtered, pooled and stored frozen. To ensure the absence of viable vector-producing cells (VPCs) from each VS unit-dose, we undertook an orthogonal log-removal validation study to demonstrate the ability of both the filtration system to remove viable cells and the VS freezing process to inactivate them. We demonstrate a total VPC-reduction of 11.6 log, thus insuring the absence of contaminating VPCs in transduced clinical samples. We also show that this production process generates stable VS that can be stored at -80 degrees C for more than 3 years. Importantly, this relatively simple and affordable process can be customized to generating large volume of VS for small animal or non-human primate studies. This methodology is not limited to the generation of cell-free clinical oncoretroviral VS, and can be applied to other types of vectors produced in packaging cell lines, such as lentiviral vectors.
基因治疗的临床应用需要大规模生产源自包装细胞系的病毒载体储备液(VS)。在扩大生产规模时,维持高病毒滴度以及对VS进行过滤变得极具挑战性。因此,必须开发易于直接验证的生产方案。为此,我们建立了一种半封闭工艺,使用10盘细胞工厂生产7升或更多的临床级嗜肝性逆转录病毒VS批次。使用蠕动泵,在连续3天收集VS,进行过滤、合并并冷冻保存。为确保每个VS单位剂量中不存在有活力的载体生产细胞(VPC),我们进行了一项正交对数去除验证研究,以证明过滤系统去除有活力细胞的能力以及VS冷冻过程使其失活的能力。我们证明VPC的总减少量为11.6个对数,从而确保转导的临床样本中不存在污染性VPC。我们还表明,这种生产过程产生的稳定VS可以在-80摄氏度下储存超过3年。重要的是,这种相对简单且经济实惠的工艺可以定制,以生产用于小动物或非人类灵长类动物研究的大量VS。这种方法不仅限于生产无细胞的临床嗜肝性逆转录病毒VS,还可应用于在包装细胞系中生产的其他类型载体,如慢病毒载体。