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Advancing science where it’s needed most

Scientific & Medical Research

There continue to be many areas in hemophilia where innovation is needed. We are focused on advancing science and programs to address serious unmet needs for people with hemophilia. To see a full list of our clinical trials, click here.


Development of Inhibitors

The development of inhibitors, or antibodies, to factor replacement therapy is one of the most serious complications for people with hemophilia, occurring in ~30% of people with severe hemophilia A.1 Bleeding is very hard to control in someone with hemophilia who develops inhibitors,2 and immune tolerance induction (ITI) therapy using factor-based products is considered to be the only way to eliminate inhibitors.3 

With immune tolerance induction (ITI) therapy, factor concentrate is given regularly over a period of time to train the immune system to accept the factor treatment without reacting to it. Improvement from ITI has typically been seen within 12 months, but more difficult cases can take two years or longer.2 This is a significant burden for patients and their caregivers.

Based on the considerable unmet need, we are conducting a study to determine whether Fc-based recombinant factor VIII can rapidly tolerize inhibitors. 

For information on this study and our other ongoing research with rFVIIIFc on the development of inhibitors and management of patients with inhibitors, please click here.


Joint Health

Debilitating joint disease is one of the most common and challenging complications for people with hemophilia, and we are committed to understanding and researching ways to improve long-term joint health.

We have conducted long-term studies showing improved joint health when our extended half-life therapies are used prophylactically, and formed a novel, advanced imaging collaboration focused on improving the management of joint health. Through a first-of-its-kind initiative, we are using imaging to study the role and importance of factor IX distribution to tissue in long-term joint health. We are also working to advance the adoption of ultrasound imaging to identify and monitor joint bleeds since clinical examinations lack the ability to tell joint bleeds from other joint conditions.


Women and Girls with Hemophilia

We are a founding member and the sole corporate sponsor of My Life, Our Future, a collaboration with the American Thrombosis and Hemostasis Network, Bloodworks Northwest, and the National Hemophilia Foundation.

Through My Life, Our Future, nearly 650 women and girls have been confirmed to carry a defective clotting factor gene and added to the world’s largest hemophilia research repository. This knowledge can help women and girls better manage their own health and help improve scientific understanding of hemophilia.

Read our interview with Dr. Robert Sidonio, Assistant Professor of Pediatrics, Associate Director of Hemostasis and Thrombosis at Children’s Healthcare University, Emory University, to learn more about the often overlooked needs of women and girls with bleeding disorders, including women and girls with factor VIII and factor IX deficiency.


Improving Treatment in the Developing World

We believe that all people with hemophilia should have access to proper treatment, regardless of where they live. Together with Swedish Orphan Biovitrum AB, we have committed to the single largest donation of factor therapy, up to one billion international units (IUs) of factor therapy over 10 years, beginning with the World Federation of Hemophilia’s (WFH) Humanitarian Aid Program receiving 500 million IUs over five years. 

Learn more about our donation, which has already helped treat over 12,300 people in 41 countries, and doubled the percentage of children receiving treatment from 14 to 28 percent.

 

1. Witmer and Young, Therapeutic Advances in Hematology. 2013 Feb; 4(1): 59-72.
2. World Federation of Hemophilia. Accessed June 16, 2017
3. Hay and Dimichele. Blood. 2012