Bioengineered Recombinant FVIII

描述(申请人提供)：重组凝血因子VIII的制造是一项复杂的、劳动和资本密集型的工作。在目前市场上销售的重组蛋白中，凝血因子VIII的生产效率最低，是迄今为止单位质量基础上最昂贵的，药价为每克10,000,000美元。目前应用的目标是开发一种能够提高第八因子生物制品的生产量和药价的产品技术和制造工艺。Expression Treateutics已经开发并获得了两项技术专利，一项是转基因生物工程因子，其表达水平是标准人类因子的100倍；另一项是高性能的婴儿地鼠肾来源细胞系，命名为BHK-MS，可以在悬浮生物反应器条件下的无血清介质中以高密度无限培养和扩增。在目前的申请中，我们建议完成临床前制造、产品表征以及提交研究性新药申请所需的药理学和毒性研究。

DESCRIPTION (provided by applicant): The manufacturing of recombinant coagulation factor VIII is a complex, labor and capital intensive endeavor. Of the recombinant proteins currently marketed, coagulation factor VIII is manufactured with the lowest efficiency and is by far the most expensive on a per unit mass basis with a pharmacy price of $10,000,000 per gram. The goal of the current application is to develop a product technology and a manufacturing process that can improve the production volume and pharmacy price of factor VIII biologics. Expression Therapeutics has developed and patented two technologies, a bioengineered factor VIII transgene that demonstrates 100-fold greater expression than standard human factor VIII, and a high performance baby hamster kidney-derived (BHK) cell line, designated BHK-MS, that can be cultured and expanded indefinitely and at high density in serum-free medium under suspension bioreactor conditions. In the current application, we propose to complete pre-clinical manufacturing, product characterization and pharmacology and toxicity studies necessary to submit an Investigational New Drug application.