Bioengineered Recombinant FVIII

DESCRIPTION provided by applicant 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 $ 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 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 PUBLIC HEALTH RELEVANCE 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 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