TO6 - GENERATING MONOCLONAL CELL LINES, MONOCLONAL ANTIBODIES, AND LABELED MONOCLONAL ANTIBODIES AGAINST HUMAN AND MOUSE COLQ

NCATS需要单克隆抗体用于开发生物分析方法，用于AAV8-COLQ的表达、毒代动力学和药代动力学研究以及免疫原性评估，AAV8-COLQ是一种基于平台的治疗先天性肌无力综合征（CMS）的基因治疗候选药物。该任务顺序定义了生产单克隆细胞系的任务，这些单克隆细胞系产生与COLQ的PRAD结构域或Human COLQ的Human表位有亲和力的抗体，以及为本项目纯化的单克隆抗体制剂，以及适合在临床前和临床试验中使用这些抗体制剂的文件。

NCATS needs Monoclonal antibodies for bioanalytical method development for expression, toxicokinetic and pharmacokinetic studies and immunogenicity assessment of AAV8-COLQ, a platform-based gene therapy drug candidate for the treatment of Congenital Myasthenic Syndrome (CMS). This task order defines the tasks which will be undertaken to produce monoclonal cell lines that generate antibodies with affinity to either the PRAD domain of COLQ or the Human epitope of Human COLQ, and purified monoclonal antibody preparations for this project and documentation suitable for use of these antibody preparations in regulated preclinical and clinical assays.