Restoring Balance in the Blood

Families managing hemophilia are seeking treatments that prevent bleeds and joint damage and require less frequent administration. That’s why we’ve been pursuing a new kind of extended half-life factor VIII replacement therapy, now in late-stage clinical trials. Based on advanced protein engineering, this elegantly engineered molecule is designed to sustain high protection from bleeds in people with hemophilia A by keeping factor levels in the normal to near-normal range for most of the week. 

NANOBODY® technology is behind the first approved treatment for aTTP, a life-threatening, immune-mediated blood disorder. The problem: small blood clots form throughout the body. The cause: a defective enzyme prevents a protein called von Willebrand factor (vWF) from working properly: large forms of this “sticky” protein remain in the blood too long, attracting platelets.

By attaching a tiny fragment of an antibody – a NANOBODY® molecule – to the vWF, we can stop it acting on platelets. In combination with plasma exchange and immunosuppressive therapy, this leads to fewer platelets caught by vWF. Platelet levels in the blood can rise, rebalancing the blood.

In people with WAHA, antibodies and other immune cells destroy healthy red blood cells faster than bone marrow can produce them. Monoclonal antibodies could give us a way to disrupt this autoimmune response. To help decommission auto-antibodies in WAHA, we're investigating a mAb designed to bind with a protein on the surface of cells that are involved in activating the autoimmune response.