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

项目摘要/摘要 目前项目长期目标是将生产的一种新的凝血因子产品商业化 使用一种新的生产细胞系，命名为ET3I/BHK-M，具有初步的抗药物治疗指征 用第VIII因子(FVIII)替代治疗先天性血友病A后产生的抗体 产品。Expression Treeutics开发了多种技术，当集成时，提供了高度的 高效的生物制造平台，旨在提高体积生产率。在第一阶段SBIR项目中 期间，表达治疗公司产生并验证了一个研究细胞库，证明了最高水平 迄今报告的重组因子VIII中，已完成上游和下游过程开发，以及 成功地将技术转移到一家合同制造组织，用于生产I期临床 测试材料。目前的应用程序寻求完成所有制造、非临床、临床规划和 进行ET3i/BHK-M第一阶段人内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.