Bioengineered Recombinant FVIII

项目摘要/摘要 重组凝血因子VIII的生产是一项复杂的、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的生产成本最低。 效率高，是迄今为止单位质量基础上最昂贵的，药房价格为每桶1000万美元 奶奶。本申请的目标是开发一种产品技术和制造方法， 可提高因子VIII生物制品的产量和药价。Expression Treeutics有 开发并获得专利的两项技术，一种生物工程第八因子转基因，证明了100倍于 比标准的人类因子VIII表达更多，并获得高性能的小仓鼠肾脏 (BHK)细胞系，命名为BHK-MS，可在 悬浮生物反应器条件下的无血清培养。在当前的应用程序中，我们建议完成 提交必要的临床前制造、产品特性和药理及毒性研究 一项研究性新药申请。

PROJECT SUMMARY/ABSTRACT 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.