Sanofi R&D teams aim to transform the standard of care for people who are affected by rare blood disorders. The company's unique R&D program builds on a legacy of innovation, stemming from the development of the first extended half-life clotting factors for treating hemophilia A and B, branching out to the first approved treatment for acquired thrombotic thrombocytopenic purpura (aTTP), and cutting-edge research in cold agglutinin disease.
Rare Blood Disorders Pipeline
Sanofi researchers are working closely with patient communities to set priorities and harnessing pioneering technology platforms to develop innovative treatments. Since 2018 Sanofi has welcomed innovative companies Bioverativ, Principia, and Ablynx, and leveraged technology from strategic partner Alnylam, to reinforce its foundations in rare blood disorders R&D. With elegant molecular designs, TAILORED COVALENCY®, NANOBODY® molecules, small interfering RNA (siRNA) technology, and a genomic medicine program, Sanofi's R&D teams are working to change expectations for how these conditions could be managed in the future.
Hemophilia, a rare, genetic blood disorder that affects blood clotting, is the cornerstone of Sanofi's Rare Blood Disorders program. To address common limitations experienced by patients, our scientists are pursuing two approaches to hemophilia management that are now in late-stage clinical trials. One investigational treatment is designed to provide extended protection for people with hemophilia A or B, with and without inhibitors (antibodies against factor therapy), designed as a subcutaneous dose. Sanofi is also working in partnership with biopharmaceutical company Sobi to develop a separate investigational treatment designed to provide high, sustained factor activity levels for people with hemophilia A.
Sanofi believes all patients should have access to therapy. Together with Sobi, the company expanded its support of the World Federation of Hemophilia Humanitarian Aid Program in 2021 by pledging to donate up to 500 million international units (IUs) of clotting factor for five years, fulfilling their 2014 pledge to donate up to 1 billion IUs of factor for humanitarian use. This is an important step toward providing a predictable supply of therapies to people in need.