CSL Ltd Annual Report 2021

Our strategic scientific platforms To ensure a robust and diverse innovation pipeline based on a foundation of scientific excellence, CSL has evolved and strengthened its therapeutic area focus. We will continue to use our four strategic scientific platforms of plasma fractionation, recombinant protein technology, cell and gene therapy, and cell-based and egg-based vaccines to support continued innovation and continually refine ways in which products can address unmet medical needs, help prevent infectious disease and protect public health, and help patients lead full lives. Plasma Fractionation Plasma is a valuable and natural but limited source of many current and potentially new biological therapies and we rely upon our donors to provide this lifesaving resource. As such, CSL Behring has an obligation to maximise the development and delivery of important products from this vital resource for the benefit of patients. Maximising patient benefit from as much of the donated plasma as possible is a critical area of focus as we strive to be the industry pacesetter. Recombinant Protein Technology The capability to develop and manufacture both recombinant proteins and monoclonal antibodies enables efficiency in manufacturing. It also facilitates the ability to manipulate the sequence of naturally occurring proteins to achieve desired therapeutic goals, such as the ability to selectively target specific biological mechanisms, enhanced potency and improved pharmacokinetics, resulting in more effective, highly differentiated medicines with the potential to optimise the route and frequency of delivery. Cell and Gene Therapy 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. Vaccines Seqirus is a transcontinental partner in pandemic preparedness and a major contributor to the prevention and control of influenza globally. Our broad range of influenza vaccines – egg-based and cell-based products, seasonal, pre-pandemic and pandemic influenza vaccines – meets the needs of different populations around the world. In Australia and the Asia Pacific region, Seqirus is a leading provider of in-licensed vaccines and specialty pharmaceuticals. It is also the world’s only supplier of a unique range of products made in the national interest for the Australian Government, including antivenoms and Q fever vaccine. Focus on influenza vaccine technologies The core focus for Seqirus R&D is the development of improved and innovative solutions for global influenza protection. For more than 50 years, influenza vaccines have been manufactured using chicken eggs to grow virus, which is then extracted, inactivated and purified. Seqirus has pioneered the modernisation of influenza vaccine technologies to improve the effectiveness of influenza vaccines, such as the development of FLUCELVAX ® QUADRIVALENT, a four-strain vaccine manufactured using state-of-the-art cell technology in our plant in Holly Springs, North Carolina, US. Cell-based manufacturing has a variety of potential advantages, including greater efficiency of production and improved matching of the virus strains included in the vaccine to those recommended by the World Health Organization (WHO). One of our most important new product development projects is our adjuvanted quadrivalent cell culture influenza vaccine (aQIVc), which combines cell-culture vaccine with MF59 ® adjuvant and will be targeted for use in older adults and children. Seqirus has also been researching self-amplifying mRNA for influenza, the next generation of mRNA technology with the potential to prevent influenza more effectively and consistently. When administered, self-amplifying mRNA has the capacity to replicate (or amplify) itself. As a result, far less mRNA may be required in the vaccine formulation to generate equivalent antigen production and an effective immune response. This has been verified in published preclinical studies where lower doses of a self-amplifying mRNA vaccine generate an equivalent or even stronger antibody and cellular immune response compared with current first-generation mRNA vaccines. CSL Limited Annual Report 2020/21 28 7 Powered by Innovation