R&D Capabilities

Plasma is a valuable resource for many current and potentially new biological therapies. We rely upon our donors to provide this lifesaving resource and as such, CSL has an obligation to maximize the development and delivery of important products from this vital resource for the benefit of patients. Maximizing patient benefit through our yield and reliability programs for donated plasma continues to be an important, strategic area of focus for CSL as we strive to be the industry pacesetter.

The capability to develop and manufacture recombinant proteins facilitates the ability to manipulate the sequence of naturally occurring proteins 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. Monoclonal antibodies are a specific subset of recombinant proteins that are developed to have a highly specific targeting to block or enhance certain biologic or immune processes which lead to disease states – the specificity of the targeting of monoclonal antibodies ensures very high efficacy with minimal side-effects.

Cell and gene therapies 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 cell and gene therapies is that the patient’s own cells are manipulated to produce the disease-correcting protein, rather than the traditional approach of manufacturing the protein and then periodically administering it to the patient.

CSL Seqirus is a global leader in seasonal influenza prevention and control and a transcontinental partner in pandemic preparedness. Our broad range of vaccines – egg-based and cell-based products, seasonal, pre-pandemic and pandemic influenza vaccines – meets the needs of different populations around the world. CSL’s commitment to population protection is evidenced through our innovative vaccines pipeline, which includes next generation technologies such as aQIVc and self-amplifying mRNA.

Our efforts in this area focus on providing trusted products and technologies to serve patients with a range of serious immunologic and neurologic diseases, including primary and secondary immunodeficiencies (PID and SID) and chronic inflammatory demyelinating polyneuropathy (CIDP). We are optimizing patient experience and convenience through more flexible ways to dose and administer our existing immunoglobulin products. We are also progressing key recombinant assets in early development such as our anti-G-CSFR monoclonal antibody, anumigilimab (CSL324), in certain neutrophilic dermatoses. We continue to build on our strong 40-year legacy in hereditary angioedema (HAE) as we look to expand on our current medicines to provide optimal treatments for the full range of HAE patients, including our recombinant monoclonal antibody garadacimab, which is currently in Phase III development.

CSL remains focused on easing the burden of disease and improving the lives of patients with rare bleeding disorders. We have made major advances in hemophilia A and B in recent years with the launch of our novel recombinant coagulation factor medicines and through the acquisition of exclusive global license rights to commercialize etranacogene dezaparvovec, an AAV5 (adeno-associated virus) gene therapy for the treatment of hemophilia B, which is currently under regulatory review. Additionally, we are undertaking exciting research and development efforts to explore new indications in hematology as well as novel therapeutics in hemostasis and thrombosis. This includes planning for an important global Phase III study to evaluate the early administration of KCENTRA® (4-factor prothrombin complex concentrate) on survival in trauma patients suffering life-threatening bleeding.

In addition to our existing product ZEMAIRA®/RESPREEZA® for patients with alpha 1 antitrypsin deficiency, CSL is investigating new clinical treatments for respiratory diseases using novel recombinant monoclonal antibodies and plasma-derived therapies to address this need. Trabikibart (CSL311), our anti-beta common monoclonal antibody, is being investigated for the treatment of severe uncontrolled asthma and severe chronic obstructive pulmonary disease (COPD). In idiopathic pulmonary fibrosis (IPF), a severe debilitating disease, we have started a clinical development program with garadacimab, the first of our compounds being explored in this disease area. CSL787, our plasma-derived, inhaled immunoglobulin is being investigated for patients with bronchiectasis and severe COPD patients.

The cardiovascular and metabolic therapeutic area is focused on improving and extending the lives of patients with cardiovascular disease (CVD) and diabetes. CSL112, apolipoprotein A-I (human), is being developed to reduce the risk of recurrent cardiovascular events during the 90-day high-risk period following a heart attack, the period when the majority of first-year recurrent cardiovascular events occur. If successful, CSL112 will be the first therapy to demonstrate cardiovascular risk reduction through the novel apoA-I mechanism and will transform how acute myocardial infarction patients at high-risk of recurrent cardiovascular events are treated.

In kidney transplant recipients, antibody-mediated rejection (AMR) is a leading cause of allograft loss, and there is significant unmet need for effective treatments. Clazakizumab, our anti-interleukin-6 (IL-6) monoclonal antibody, is currently being investigated in a Phase III clinical trial (IMAGINE) for the potential treatment of chronic active antibody-mediated rejection. In haematopoietic stem cell transplantation, acute graft-versus-host disease (GvHD) is a life-threatening type of rejection where the donor cells attack the recipient; it is 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.

With a focus on influenza, developing new and better vaccines across all age groups in expanded markets is a strategic priority for CSL Seqirus, including further advancing our cell-based manufacturing technology and our MF59® adjuvant and developing our self-amplifying messenger RNA (sa-mRNA) technology, to enhance the immune response of those particularly vulnerable to influenza such as children and older adults. We are also investigating a quadrivalent adjuvanted cell culture influenza vaccine (aQIVc) which combines FLUCELVAX® antigen with MF59® adjuvant, an additive that acts to strengthen the immune response to vaccination.

The acquisition of Vifor Pharma Ltd expands CSL’s leadership across an attractive portfolio focused on Renal Disease and Iron Deficiency and, enhances CSL’s patient focus and ability to protect the health of those facing a range of rare and serious medical conditions. The Vifor Pharma portfolio of products across Nephrology, Dialysis and Iron Deficiency therapies complements CSL’s existing therapeutic focus areas including Hematology and Thrombosis, Cardiovascular-Metabolic, and Transplant, and high-quality pipeline. The acquisition expands CSL’s portfolio breadth with the addition of 10 commercialised products including FERINJECT®/ INJECTAFER®, VENOFER®, VELTASSA®, and soon KORSUVA®, adding leadership positions across multiple franchises. In addition, CSL’s global scale, R&D capabilities and resources augment the delivery of Vifor Pharma’s products to patients globally.