FVIII Infusion Therapy

Agarose gel electrophoresis of a DNA restriction digest used to ensure quality

Currently, hemophilia A is treated by prophylactic or on-demand intravenous administration of either plasma-derived or recombinant factor VIII products. Current limitations of treatment include the development of humoral anti-factor VIII immune responses and the cost of treatment. The cost of hemophilia A treatment is a function of the manufacturing costs related to low-level expression of recombinant human factor VIII. In addition to being a significant economic factor in health care costs in developed countries, the cost of factor VIII precludes factor VIII replacement therapy in most hemophilia A patients in developing countries.

The same bioengineered factor VIII transgene that demonstrates high-level expression in the gene therapy setting also generates 100-fold greater expression from recombinant protein manufacturing systems. By reducing manufacturing costs, our recombinant factor VIII product will have a significant impact on decreasing the cost of factor VIII replacement therapy, thereby advancing the treatment of hemophilia A worldwide.