Two female researchers testing vials in a lab)

R&D Hematology

Hematology

CSL Behring remains focused on easing the burden of care and improving the lives of patients with rare bleeding disorders. Additionally, we are undertaking exciting research and development efforts to explore new indications in hematology as well as novel therapeutics in hemostasis and thrombosis. We have made major advancements in hemophilia A and B in recent years with the launches of our internally developed family of novel recombinant coagulation factor medicines. Our hemophilia research strategy is based on maximizing the value and performance of our existing coagulation therapies and developing new protein-based therapies to treat bleeding disorders and increase the quality of life for patients.

Hand holding a pipet cropped

We are working towards improving the quality of life for Hemophilia B patients by evaluating the possibility of extending the dosing intervals from 14 to 21 days. A Phase 3 extension study (PROLONG-9FP) is underway and global regulatory submissions to gain approval for the extended dosing regimen are planned for 2019.

Our commitment to remain at the forefront of innovation is stronger than ever. The acquisition of Calimmune Inc., introduced a new ex vivo hematopoietic stem cell gene therapy (CAL-H/CSL200) for the treatment of sickle cell disease (SCD) into our hematology and thrombosis pipeline. Calimmune’s proprietary platform technologies also have the potential to develop new medicines for a wide range of other rare diseases that complement CSL’s product portfolio and expertise. Complementing the CSL200 program is another exciting program in SCD, CSL889. The CSL889 is plasma derived Hemopexin, an important endogenous molecule whose levels are decreased in patients with sickle cell disease, for treatment of acute vaso-occlusive crisis, a severe and painful complication in SCD patients. Clinical trials using CSL200 and CSL889 are anticipated to start in 2019 and 2020 respectively.