CSL Limited Annual Report 2022/23 36 6 Powered by Innovation Our strategic scientific platforms To ensure a robust and diverse innovation pipeline based on a foundation of scientific excellence, CSL continues to strengthen its therapeutic area focus underpinned with robust technical development platforms. CSL uses its strategic technical platforms of plasma protein technology, recombinant protein technology, cell and gene therapy, and vaccines technology to support continued innovation and continually refine ways in which products can address unmet medical needs, help prevent infectious disease to protect public health, and help patients lead full lives. Plasma Protein Technology Plasma is a valuable resource for many current and potentially new biological therapies. We rely upon our donors to provide this life-saving resource and as such, CSL Behring has an obligation to maximise the value of each plasma donation through the development and delivery of important therapies for the benefit of patients. CSL’s yield and reliability programs for donated plasma continue to be an important, strategic area of focus for CSL as we strive to be the industry leader in plasma-derived therapies. Recombinant Protein Technology 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 optimise the route and frequency of delivery. CSL’s garadacimab program highlights the value of monoclonal antibodies, 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 Therapy 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, either by removing the patient’s cells and modifying them or, as with HEMGENIX®, by using molecular machinery derived from viruses to deliver the therapeutic gene to the desired cell type within the patient’s body. Vaccines Technology CSL Seqirus is a global leader in influenza vaccine technologies for prevention and control of seasonal disease, and a transcontinental partner in pandemic preparedness. Our egg-based and cell-based manufacturing capabilities in three continents produce more than 100 million doses of influenza vaccines annually. Together with our 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 our innovative vaccines pipeline, which includes next-generation technologies such as next-generation mRNA and recombinant antigen production, to address emerging and present viral threats to human health.
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