R&D Capabilities

Plasma is a valuable resource for many current and potentially new biological therapies.
CSL relies on our plasma donors to provide this lifesaving resource and as such, we operate under the belief that we have an obligation to maximize the benefit of each donation. The pursuit of state-of-the-art technologies to improve yield and reliability processes for donated plasma continue to be an important, strategic area of focus for CSL therefore we strive to be the industry leader in plasma-derived therapies and increase patient access to these life-changing products. CSL continues to focus on initiatives to increase immunoglobulin (Ig) yield including enhancing the current Ig process robustness and recoveries through data analytics, process optimisation, plasma allocation and operational excellence. In addition to yield, we also focus on new delivery vehicles, new formulations and the development of new plasma medicines to make the most of this natural resource for patients.

Recombinant protein technology uses cells, grown in large batches, each as an individual protein production factory. This allows product supply to be reliably scaled (compared to plasma collection), ensuring a robust and resilient supply of products to patients. The capability to further manipulate the sequence of recombinant proteins permits a responsiveness to achieve desired therapeutic goals, such as the ability to replace a patient’s own deficient or inactive protein, selectively target specific biological mechanisms, enhance potency and improve pharmacokinetics, resulting in more effective, highly differentiated medicines with the potential to optimize the route and frequency of delivery. Today, recombinant proteins make up a significant portion of CSL’s early development portfolio and a differentiating aspect of our newest pipeline product, garadacimab, will be its patient-friendly characteristics.

Genetic medicines such as genome editing, RNA editing and RNA mediated protein replacement, are highly innovative, next-generation products that, after decades of research and development, are now starting to improve 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 fundamental differentiating characteristic of genetic medicine is that the patient’s own cells are manipulated to produce the disease-correcting protein. Building on the success of HEMGENIX^®, CSL continues to develop technologies to make therapeutic gene delivery more precise and durable, so that CSL can deliver on the potential of a long-term cure.

CSL is a global leader in influenza vaccines technologies for prevention and control of seasonal disease, and a transcontinental partner in pandemic preparedness. CSL’s egg-based and cell-based manufacturing capabilities in three continents produce more than 100 million doses of influenza vaccines annually. Together with CSL’s MF59^® adjuvant, our influenza vaccines help to meet the needs of different populations around the world.
CSL’s ongoing commitment to population protection is evidenced through CSL’s innovative vaccines pipeline, which includes next generation technologies such as self-amplifying mRNA and recombinant antigen production, to address emerging and present viral threats to human health.

Our efforts are dedicated to providing trusted products and technologies for patients with serious immunologic and neurologic diseases, such as primary and secondary immunodeficiencies (PID and SID) and chronic inflammatory demyelinating polyneuropathy (CIDP). In addition, we are assessing the use of inhaled immunoglobulin for patients with bronchiectasis to enhance our respiratory portfolio, alongside our existing product ZEMAIRA^®/RESPREEZA^® for alpha-1 antitrypsin deficiency. Building on our legacy of delivering reliable, life-sustaining products, the Immunoglobulin TA is committed to advancing integrated and convenient approaches, guided by the voices of our patients.

CSL remains focused on easing the burden of disease and improving the lives of patients with rare bleeding disorders and benign hematological conditions. Significant progress was achieved in recent years in the treatment of haemophilia A and B through the introduction of innovative recombinant coagulation factors. More recently CSL gained approval of the first gene therapy for haemophilia B, HEMGENIX^®. CSL has deployed research efforts to develop therapeutic options for patients with sickle cell disease, a patient population with a high unmet medical need. We are focused on both acute treatment of vaso-occlusive crises and effective prophylaxis to reduce the frequency of sickle-cell related events. Additionally, we are undertaking exciting research efforts to advance innovative therapeutics in benign hematology conditions with unmet medical need, specifically in the areas of hemostasis and thrombosis.
There is a worldwide trend towards reasonable patient blood management (PBM) that includes the replacement of allogeneic blood product transfusions by coagulation factor concentrates wherever they are available. In keeping with CSL’s vast history in the area of coagulation factor concentrates, efforts are ongoing to ensure availability of fibrinogen and prothrombin factor concentrates during surgical procedures with high risk of major bleeding. In addition, the PBM includes focus on IV iron therapy for pre-/post-operative anemia management.

The cardiovascular and renal therapeutic area is focused on improving and extending the lives of patients with cardiovascular and renal diseases through our marketed products and development programs. Cardiovascular disease (CVD) is the primary cause of morbidity and mortality in patients with chronic kidney disease, particularly those with advanced CKD stages (4-5) who have a markedly elevated risk for CVD. The 3-year rate of major adverse cardiac events (cardiovascular death, myocardial infarction, stroke) in patients undergoing hemodialysis is approximately 25%, almost 3-fold higher than observed in patients with cardiovascular disease not on dialysis. CSL is developing Clazakizumab (an anti-IL6 antibody) for the prevention of cardiovascular morbidity and mortality in patients with end stage renal disease. In addition, there is an urgent need to develop novel therapies that preserve kidney function and delay, or avoid, dialysis. We are proud to offer TAVNEOS^® and FILSPARI^® to help patients with rare renal diseases.

We continue to build on our strong 40-year legacy in hereditary angioedema (HAE) as we work to expand our current medicines to provide optimal treatments for the full range of HAE patients, including our recombinant monoclonal antibody garadacimab.
Our efforts also focus on developing treatments for select autoimmune indications of high unmet need. Autoimmune diseases are chronic, complex conditions that are a result of a loss of self-tolerance. CSL is committed to build and grow the autoimmune portfolio to bring innovative treatments to patients with autoimmune diseases.
Despite advances in transplantation improving short-term survival, transplant rejection is one of the greatest limitations to long-term graft and patient survival for both solid organ and haematopoietic stem cell transplant recipients.
In kidney transplant recipients, antibody-mediated rejection (ABMR) is a leading cause of allograft loss, and there is significant unmet need for effective treatments. CSL is focused on developing therapies to address these conditions that may lead to transplant organ failure.
In haematopoietic stem cell transplantation, acute graft-versus-host disease (GvHD) is a life-threatening type of rejection and a leading cause of mortality and morbidity following transplant. There is a significant unmet need for more effective, less toxic therapies for GvHD. We are investigating alpha 1 antitrypsin (AAT, ZEMAIRA^®) for the prevention and treatment of acute GvHD in two Phase III studies.

Advancing vaccines for the benefit of public health is a strategic priority for CSL. With a focus on expanding beyond influenza and progressing our current technologies, our first non-influenza vaccine, the sa-mRNA COVID-19 vaccine, KOSTAIVE^® was recently approved in Japan.

Additionally, CSL is further advancing cell based manufacturing technology in products such as aTIVc (adjuvanted trivalent influenza vaccine), vaccines containing CSL’s MF59^® adjuvant as well as developing the messenger RNA (mRNA) platform, targeting viruses of both seasonal and pandemic potential.