Excellence in Research & Development
CSL Behring's mission is to discover, develop, and deliver our innovative therapies that improve the patients' quality of life. Our scientists and researchers are recognized for their contributions to developing breakthrough medicines that have the potential to help millions of patients worldwide.
We are grateful to the patients, volunteers, and researchers that participate in the clinical trials for testing our potential biotherapies. All contributions have helped better our understanding of diseases.
CSL Behring's R&D capabilities are focused in three major areas:
CSL Behring’s clinical research activities make products available to patients worldwide and identify novel therapeutic uses for plasma proteins.
As a leading manufacturer and developer of therapeutics derived from human plasma, CSL Behring is committed to maintaining the highest product safety standards and to continually improving manufacturing effectiveness. CSL Behring's research programs are focused on developing novel plasma proteins with improved efficacy and enhanced convenience.
We have extensive experience in the production, clinical development and launch of recombinant coagulation factors including IDELVION® and AFSTYLA®.
We are also focused on the development, production and testing of novel monoclonal antibodies (MAbs) to treat inflammatory diseases (CSL324), hereditary angioedema (CSL312) and target fatty acid metabolism (CSL346).
In collaboration with Momenta Pharmaceuticals, we are also developing recombinant Fc multimer proteins to control inflammation associated with autoimmune diseases.
Through the recent acquisition of Calimmune Inc., CSL is now focused on the development of ex vivo hematopoietic stem cell (HSC) gene therapy which has the potential to offer a significant advantage to patients suffering from currently incurable genetic diseases.
The acquisition of Calimmune introduced CAL-H/CSL200 to our pipeline, an HSC gene therapy for the treatment of sickle cell disease, which complements our current product portfolio and deep expertise in hematology.
The technology also has the potential to be used in treatments for a wide range of other rare diseases that would complement our business, including those within the company’s current product portfolio.
These capabilities manifest themselves in our innovative immunoglobulins, specialty plasma products, hemophilia products, breakthrough medicines and treatments for transplant.
R&D provides support to CSL Behring's world-leading immunoglobulin (Ig) franchise. Our efforts in this area are focused on new indications for our existing intravenous and subcutaneous products such as PRIVIGEN® and HIZENTRA®, exploring new molecules and new technologies for delivery options, focusing on new indications such as Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), and refining our formulation and purification processes.
We have established an exclusive research collaboration and worldwide license agreement with Momenta Pharmaceuticals, Inc. to develop and commercialize recombinant Fc multimer proteins for use in controlling inflammation. The agreement includes Momenta’s novel M230/CSL730 product which has been shown to match the potency and efficacy of intravenous Ig at significantly lower doses in animal models of autoimmune disease. Clinical trials with M230/CSL730 are now underway with the potential to further grow our long-term global leadership in helping patients with autoimmune diseases that are treated with immunoglobulins.
CSL Behring recently launched the world’s first and only subcutaneous preventative treatment for patients with Hereditary Angioedema (HAE). HAEGARDA represents a new standard of care for HAE patients, reducing HAE attacks by 95% and the need for rescue medication by 99%.
In order to remain at the forefront of innovation in HAE treatment, CSL has signed an exclusive license agreement with CEVEC Pharmaceuticals to develop highly differentiated recombinant C1 Esterase Inhibitor (C1-INH) proteins. Building on our deep knowledge and expertise of HAE and plasma derived C1-INH, we aim to leverage CEVEC’s expertise in the production of recombinant C1-INH using their proprietary CAP®Go technology. The technology provides the opportunity to develop innovative proteins with improved half-life and more convenient administration, further improving the quality of life for patients with HAE.
CSL is committed to developing therapies for patients with rare and life-threatening conditions. We are currently developing new products in solid organ transplant, an area of unmet clinical need. Organ transplants often fail and the organ is eventually rejected, even though the transplant has been successful in the first instance.
Antibody-mediated rejection (AMR) is a major cause of kidney transplant failure and is often associated with the activation of complement, a set of proteins that work with antibodies and play a role in the development of inflammation and tissue damage. C1-INH present in human plasma regulates the complement pathway. Administering additional C1-INH after solid organ transplantation is expected to reduce the action of the complement system, therefore reducing the likelihood of the transplanted organ being rejected. We have initiated a clinical trial to evaluate the efficacy and safety of plasma derived C1-INH (CSL842) in the treatment of refractory AMR in renal allograft recipients.
We recently entered into a strategic collaboration with Vitaeris Inc. to expedite the development of clazakizumab (an anti-IL6 MAB, formerly ALD518) as a therapeutic option for solid organ transplant rejection. Vitaeris' transplant rejection program is complementary to CSL's current development activities in solid organ transplant. Our expertise in immunology, our pipeline and strategic partnerships are full of promise to address unmet needs in the transplant community.
Our commitment to remain at the forefront of innovation is stronger than ever. The acquisition of Calimmune Inc., introduced a new ex vivo haematopoietic stem cell gene therapy (CAL-H/CSL200) for the treatment of sickle cell disease into our Breakthrough Medicine pipeline. Calimmune’s proprietary platform technologies have the potential to develop new breakthrough medicines for a wide range of other rare diseases that complement CSL’s product portfolio and expertise.
We continue to make strong progress in our Breakthrough Medicine portfolio with three of our novel monoclonal antibodies (mAb) in clinical trials. CSL312 is an anti-factor XIIa mAb in development for multiple indications including HAE. CSL324 neutralises G-CSF activity and may provide a new treatment for rare inflammatory diseases associated with overactive neutrophils (white blood cells). CSL346 targets VEGF-B and could potentially be used to control glucose absorption in Type 2 diabetes by targeting fatty acid metabolism.
The largest clinical trial ever undertaken by CSL Behring is now underway with CSL112, a novel plasma derived apolipoprotein A-1 infusion therapy that has been shown to have an immediate and significant impact on the removal of cholesterol from arteries. CSL112 has the potential to reduce early recurrent cardiovascular events, experienced by nearly one in five survivors of acute myocardial infarction or heart attack. If successful, CSL112 will be a transformative growth driver for CSL and will help to address one of the world’s most prevalent and devastating diseases.
CSL Behring remains focused on easing the burden of care and improving the lives of haemophilia patients. R&D has contributed to major advancements in recent years with the launches of CSL Behring's internally developed family of novel recombinant coagulation factor medicines including AFSTYLA® and IDELVION®. Our hemophilia research strategy is based on: maximizing the value and performance of our existing coagulation therapies, developing new protein-based therapies for treating bleeding disorders, and addressing inhibitors -- all with a major focus on increasing the patient quality of life.