EXPRESSION THERAPEUTICS LLC SBIR Phase II Award, August 2018

PROJECT SUMMARY ABSTRACT The long term objective of current project is to commercialize a novel coagulation factor product manufactured using a novel production cell linedesignated ET i BHK M with a primary indication of treatment of anti drug antibodies that develop following the treatment of congenital hemophilia A with factor VIIIfVIIIreplacement productsExpression Therapeutics has developed multiple technologies thatwhen integratedprovide a highly efficient biomanufacturing platform designed for improved volumetric productivityIn the phase I SBIR project periodsExpression Therapeutics generated and validated a research cell bank demonstrating the highest levels of recombinant factor VIII reported to datecompleted upstream and downstream process developmentand successfully transferred the technology to a contract manufacturing organization for production of phase I clinical test materialThe current application seeks to complete all manufacturingnonclinicalclinical planningand regulatory activities required to conduct a phase I first in man trial of ET i BHK Mwith the primary endpoint of safety and tolerability PROJECT NARRATIVE Despite many advances toward the control and prevention of bleeding in hemophilia A through coagulation factor VIII infusion therapyof severe patients develop anti drug antibodies termedinhibitorsthat block treatment efficacyIn the USCanada and Western Europethe majority of inhibitor patients are treated off label with highermore frequent administration of expensive coagulation factor VIII products in protocols collectively referred to asImmune Tolerance InductiontherapyThe goal of the current project is to commercialize the first product using our novel commercial cell production systemwhich will be a coagulation factor VIII product with a primary label indication for the treatment of fVIII inhibitors through Immune Tolerance Induction therapy