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

项目总结/摘要 本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的新生产细胞系，其主要适应症为抗药物治疗， 用因子VIII（fVIII）替代治疗先天性血友病A后产生的抗体 产品. Expression Therapeutics开发了多种技术，当这些技术整合在一起时， 高效的生物制造平台，旨在提高体积生产率。在SBIR项目第一阶段， 期间，Expression Therapeutics生成并验证了一个研究细胞库，该细胞库显示出最高水平 已完成上游和下游工艺开发，以及 成功地将该技术转让给合同生产组织，用于生产I期临床 试验材料。当前应用程序旨在完成所有制造、非临床、临床计划和 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。

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.