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

点击翻译按钮获取中文摘要

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.