Globally, we are helping to lead the transformation of hemophilia treatment in the developing world, where people with severe hemophilia often don’t survive to adulthood. Together with Sobi, we have pledged the single largest donation of hemophilia factor therapy in history: up to one billion IUs of clotting factor over 10 years, beginning with up to 500 million IUs to the World Federation of Hemophilia Humanitarian Aid Program over a period up to five years. This is an important first step toward providing a predictable and sustainable supply of therapy to those most in need.
Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Beginning in 2018, we launched the first approved treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a rare, life-threatening, autoimmune-based, blood disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to a very low platelet count, a loss of red blood cells and restricted blood supply to parts of the body.
Poised for Growth
In addition to our current portfolio, we have a strong pipeline focusing on areas of unmet need in the rare blood disorder community, including hemophilia, cold agglutinin disease, sickle cell disease, and beta thalassemia.
- Cold Agglutinin Disease
We are currently conducting late-stage clinical trials of a breakthrough investigational therapy for the treatment of cold agglutinin disease (CAD), a chronic and severe rare blood disorder with no approved treatments.
- Sickle Cell Disease and Beta Thalassemia
In partnership with Sangamo, we are developing gene-edited cell therapies with the aim of providing a one-time, lasting treatment for people living with sickle cell disease or beta thalassemia. These lifelong rare blood disorders are triggered by genetic mutations. Currently, patients have few treatment options.
Learn about treatment options: