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.
The long-term commercial objective of the current project is to commercialize a novel coagulation factor product for the treatment of persons with hemophilia A of which 75% currently do not receive factor products due to product cost and availability. Expression Therapeutics has developed multiple technologies that, when integrated, provide a highly efficient biomanufacturing platform designed for improved volumetric productivity of recombinant coagulation factor VIII, a complex and hard to manufacture biologic. In the phase I SBIR component of this project, Expression Therapeutics demonstrated proof-of-concept of the efficiency and scalability of this novel recombinant factor VIII product and manufacturing process platform. During the phase II period, preclinical safety, efficacy and product identity activities will be executed with the goal of an Investigational New Drug application submission.