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Bioengineered Recombinant FVIII

生物工程重组 FVIII

基本信息

批准号：
8796739
负责人：
Gabriela Denning
金额：
$ 67.36万
依托单位：
EXPRESSION THERAPEUTICS
依托单位国家：
美国
项目类别：
财政年份：
2013
资助国家：
美国
起止时间：
2013-02-01 至 2017-01-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8796739
关键词：
Adolescence Anabolism Animal Model Biochemical Biological Products Biomanufacturing Biomedical Engineering Bioreactors Blood Coagulation Factor Breathing Capital Cell Culture Techniques Cell Line Cells Clinical Complex Development Development Plans Disease Documentation Dose Endoplasmic Reticulum Ensure Factor VIII Family suidae Feedback Fermentation Glycoproteins Goals Golgi Apparatus Growth HIV Half-Life Hamsters Health Healthcare Hemophilia A Human Intellectual Property Investigational Drugs Investigational New Drug Application Kidney Kilogram Legal patent M cell Mammalian Cell Marketing Messenger RNA Methods Modeling Performance Persons Pharmaceutical Preparations Pharmacology Pharmacology and Toxicology Pharmacy facility Phase Plasma Post-Translational Protein Processing Preparation Price Process Production Productivity Property Proteins Recombinant Proteins Recombinants Records Recovery Reference Standards Regulatory Affairs Replacement Therapy Rodent Running Safety Serum Serum-Free Culture Media Small Business Innovation Research Grant Suspension substance Suspensions System Technology Technology Transfer Testing Therapeutic Toxic effect Transgenes Virus base biophysical techniques commercialization cost cost effective density design experience human F8 protein improved innovation manufacturing process manufacturing process development meetings mortality novel operation pre-clinical preclinical safety recombinant antihemophilic factor VIII response scale up technology development

项目摘要

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.

描述（由申请人提供）：重组凝血因子VIII的生产是一项复杂的劳动和资本密集型工作。在目前上市的重组蛋白中，凝血因子VIII的生产效率最低，并且是迄今为止单位质量基础上最贵的，每克药价为1000万美元。目前申请的目标是开发一种产品技术和制造工艺，以提高因子VIII生物制剂的产量和药品价格。Expression Therapeutics开发了两项技术并获得了专利，一项是生物工程因子VIII转基因，其表达量比标准人因子VIII高100倍，另一项是高性能的幼鼠肾源性（BHK）细胞系，称为BHK- ms，可以在悬浮生物反应器条件下在无血清培养基中以高密度无限期培养和扩增。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新药申请所需的药理学和毒性研究。