Leaders in their fields, our scientists are deeply committed to the discovery and development of new medicines to address areas of serious need for patients. Leveraging our deep understanding of the biology of haemostasis gained over 15 years of research, we plan to accelerate the development of our innovative pipeline of programmes in haemophilia, cold agglutinin disease, sickle cell disease, beta thalassemia, and other blood disorders.
Our pipeline includes haemophilia programmes that have been designed to provide less-frequent prophylactic dosing for haemophilia A and subcutaneous dosing for haemophilia B, and gene therapy programmes for haemophilia A and B.
It also includes programmes to address cold agglutinin disease, a rare and chronic autoimmune haemolytic condition for which there are no approved therapies. BIVV009 (formerly TNT009) is the only therapy in development that is designed to selectively inhibit the classical complement pathway of the immune system. BIVV009 targets C1s, thereby impacting the central mechanism of this disease.
We are also pursuing several approaches that seek to target the root cause of sickle cell disease, a profoundly debilitating disease that is linked to a shorter life expectancy and has few treatment options. Just as we have done in haemophilia, we hope to bring forward new medicines that meaningfully advance the treatment of people with sickle cell disease.