Producing humanized therapeutics in quail egg white

用鹌鹑蛋清生产人性化疗法

• 批准号: 6735891
• 负责人: STEPHEN H PARKER
• 金额: $ 9.17万
• 依托单位: SYNAGEVA BIOPHARMA CORPORATION
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-06-01 至 2005-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6735891
• 关键词: CD40 molecule; antiantibody; bioreactors; biotechnology; biotherapeutic agent; cell line; complementary DNA; egg food product; enzyme linked immunosorbent assay; genetically modified animals; immunoglobulin G; immunologic substance development /preparation; lysozyme; microinjections; monoclonal antibody; quail; technology /technique development; transfection

DESCRIPTION (provided by applicant): This project is focused on the development of a novel transgenic avian production platform that will provide human therapeutics in a rapid, cost-effective fashion. Specifically, we intend to create transgenic Japanese quail (Coturnix Coturnix sp. japonica) that produce humanized monoclonal antibody (huMAb) within the albumen (white) portion of their laid eggs. The anticipated demand for therapeutic huMAbs will require a production capacity greater than what is currently available in the worldwide industry. The rapid onset of sexual maturity in quail will result in the production of huMAb in a fourth of the time required by other transgenic platforms (e.g., chickens, mammals and plants). The primary goal of Phase I is to produce huMAb in quail egg white in sufficient quantities for subsequent detailed characterization. This will be accomplished by first identifying DNA constructs that express huMAb in vitro in a cell specific manner. Transgenic G1 hens will be generated to produce huMAb in quantities sufficient for glycosylation and avidity analyses to be initiated in Phase II of the project. Phase II will conclude with the production of a transgenic flock capable of generating quantities of huMAb required for clinical trials. The development of quail as a bioreactor platform will decrease the time required to evaluate potential huMAb therapeutics and other biologics.

描述（由申请人提供）：该项目的重点是开发一个新型的转基因鸟类生产平台，该平台将以快速，具有成本效益的方式提供人类治疗剂。具体而言，我们打算在其蛋鸡的蛋白（白色）部分中创建产生人源化的单克隆抗体（Humab）的转基因鹌鹑（Coturnix coturnixsp。Japonica）。 预期的对Humab的预期需求将需要比全球行业目前可用的生产能力更大。鹌鹑性成熟度的迅速发作将导致其他转基因平台（例如鸡，哺乳动物和植物）所需的时间的四分之一。第一阶段的主要目标是用足够数量的鹌鹑蛋白生产Humab，以进行详细的表征。这将通过首先识别以细胞特异性方式在体外表达Humab的DNA构建体来实现。将生成转基因G1母鸡以产生足以在项目的II期中启动糖基化和亲戚分析的量。第二阶段将以生产能够产生临床试验所需的Humab数量的转基因羊群的结论。鹌鹑作为生物反应器平台的发展将减少评估潜在的Humab疗法和其他生物制剂所需的时间。