CSL’s strategic scientific platforms CSL is dedicated to maintaining a strong innovation pipeline grounded in scientific rigour by focussing on strategic therapeutic areas and leveraging its technical development platforms. CSL defines a “strategic platform” as an area where it has the expertise to manufacture a new therapy or vaccine at every stage in its lifecycle, such as plasma protein technology, recombinant protein technology, cell and gene therapy, and vaccines technology. These platforms facilitate ongoing innovation in the development of products to address unmet medical needs, prevent infectious disease and help patients lead full lives. Plasma protein technology Plasma is a valuable resource for many current and potentially new biological therapies. CSL relies upon donors to provide this life-saving resource and as such, CSL has an obligation to maximise the value of each plasma 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 as we strive to be the industry leader in plasma-derived therapies and increase patient access to CSL’s life-changing products. CSL continues to focus on initiatives to increase immunoglobulin (Ig) yield: Horizon 1 improvements include 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 further leverage this remarkable natural resource and its pluripotential opportunities for patients. 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. 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. 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, cell and gene therapies 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. 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. Vaccines technology CSL is a global leader in influenza vaccine 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 technologies such as sa-mRNA and recombinant antigen production, to address emerging and present viral threats to human health. 27
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