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Two female researchers testing vials in a lab)

R&D Haematology


CSL remains focused on easing the burden of care and improving the lives of patients with rare bleeding disorders. We have made major advances in haemophilia A and B in recent years with the launch of our novel recombinant coagulation factor medicines and through the acquisition of exclusive global licence rights to commercialise etranacogene dezaparvovec, uniQure’s AAV5 (adeno-associated virus) gene therapy for the treatment of haemophilia B. Additionally, we are undertaking exciting research and development efforts to explore new indications in haematology as well as novel therapeutics in haemostasis and thrombosis. Our haemophilia research strategy is based on maximising 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 continuing to work towards improving the quality of life for Haemophilia B patients with extended dose options for IDELVION®. This product is CSL Behring’s long-acting recombinant albumin fusion protein for treating haemophilia B. The routine prophylaxis dosing regimen has been updated to extend the treatment interval up to 21-day dosing for appropriate adult patients.

Our commitment to remain at the forefront of innovation is stronger than ever. Through the acquisition of Calimmune Inc., CSL is focused on the development of ex vivo haematopoietic stem cell (HSC) gene therapy which has the potential to offer a significant advantage to patients suffering from currently incurable genetic diseases. Cell and gene therapies are highly innovative, next-generation products that, after decades of research and development, are now starting to positively impact the lives of patients with serious diseases. For diseases with few effective therapeutic options, such as certain blood cell cancers, or where successful therapy has required a lifetime of regular symptomatic treatment, such as rare inherited genetic deficiencies, they offer the promise of a long-term cure. The technology has the potential to be used in treatments for a wide range of rare diseases that would complement our business, including those within the company’s current product portfolio.

CSL889 is a form of plasma derived Hemopexin, an important naturally occurring protein produced in the body whose levels are decreased in patients with sickle cell disease (SCD). CSL889 is being developed for the treatment of acute vaso-occlusive crisis, a severe and painful complication in SCD patients.