Clinical Testing of the First Suspension BHK-M Cell Platform Derived Biotherapeutic

第一个悬浮 BHK-M 细胞平台衍生的生物治疗药物的临床测试

基本信息

批准号：
9759962
负责人：
Gabriela Denning
金额：
$ 75.21万
依托单位：
EXPRESSION THERAPEUTICS
依托单位国家：
美国
项目类别：
财政年份：
2012
资助国家：
美国
起止时间：
2012-08-01 至 2022-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9759962
关键词：
Antibodies Biological Response Modifier Therapy Biological Sciences Biomanufacturing Biomedical Engineering Bioreactors Blood Coagulation Factor Businesses Canada Capital Caring Cell Line Cells Chemistry Clinical Complication Consumption Contracts Country Data Analyses Development Development Plans Documentation Dose Economics F8 gene Factor VIII Family suidae Feedback Goals Half-Life Hamsters Hemophilia A Hemorrhage Human Immune Tolerance Infusion procedures Insurance International Investments Iowa Kidney Label Laboratories Legal patent M cell Materials Testing Methods Multicenter Trials National Heart, Lung, and Blood Institute Neoadjuvant Therapy Patients Persons Pharmaceutical Preparations Pharmacology and Toxicology Phase Policies Population Prevention Process Production Productivity Proteins Protocols documentation R-factor Recombinants Records Recovery Reporting Research Resources Running Safety Serum Services Small Business Innovation Research Grant Suspensions System Technology Technology Transfer Teenagers Testing Therapeutic Treatment Efficacy Treatment Factor Western Europe Work barrier to care cell bank clinical development clinical material commercialization cost design first-in-human improved inhibitor/antagonist innovation meetings novel off-patent open label preclinical development primary endpoint programs recombinant antihemophilic factor VIII research clinical testing safety study stability testing therapy design

项目摘要

PROJECT SUMMARY/ABSTRACT The long-term objective of current project is to commercialize a novel coagulation factor product manufactured using a novel production cell line, designated ET3i/BHK-M with a primary indication of treatment of anti-drug antibodies that develop following the treatment of congenital hemophilia A with factor VIII (fVIII) replacement products. Expression Therapeutics has developed multiple technologies that, when integrated, provide a highly efficient biomanufacturing platform designed for improved volumetric productivity. In the phase I SBIR project periods, Expression Therapeutics generated and validated a research cell bank demonstrating the highest levels of recombinant factor VIII reported to date, completed upstream and downstream process development, and successfully transferred the technology to a contract manufacturing organization for production of phase I clinical test material. The current application seeks to complete all manufacturing, nonclinical, clinical planning, and regulatory activities required to conduct a phase I first-in-man trial of ET3i/BHK-M, with the primary endpoint of safety and tolerability.

项目概要/摘要当前项目的长期目标是将生产的新型凝血因子产品商业化使用命名为 ET3i/BHK-M 的新型生产细胞系，其主要适应症为抗药物治疗使用因子 VIII (fVIII) 替代疗法治疗先天性 A 型血友病后产生的抗体产品。 Expression Therapeutics 开发了多种技术，这些技术集成后可提供高度高效的生物制造平台，旨在提高产量。在第一阶段SBIR项目中期间，Expression Therapeutics 生成并验证了一个研究细胞库，展示了最高水平迄今为止已报告的重组因子 VIII，已完成上游和下游工艺开发，以及成功将技术转让给合同制造机构进行I期临床生产测试材料。当前的申请旨在完成所有制造、非临床、临床规划和进行 ET3i/BHK-M I 期首次人体试验所需的监管活动，主要终点为安全性和耐受性。