Pharmaceuticals have developed via three manufacturing revolutions. The first arose in the 19th century from the ability to chemically synthesise drugs previously only obtainable from natural sources (aspirin, quinine). Next in the 1970's the biotechnology revolution enabled proteins such as insulin, clotting factors or antibodies to be developed into safe widespread treatments. Currently we are in the midst of the cell therapy revolution where the ability to grow human cells outside the body is being exploited to create cell based treatments. More generally, artificial cell culture is a widespread and rapidly expanding technology with applications in medicine, bioprocessing, crop science, drug development and clinical research. In recent years the idea of cell based therapies has moved from mere possibility to actual treatments for conditions such as leukaemia, stroke, blindness and arthritis. These require not only that cells can be grown outside the body but that they can be multiplied and modified before reintroduction into the patient. In many cases the body's immune system restricts cell therapies to autologous forms where the original cells are obtained from the patient, grown and or modified and then reintroduced. However several allogeneic treatments, where a commercial cell line is used to treat many patients, are also in development for conditions such as stroke or inherited blindness. Much work depends upon growing stem cells which are a "raw material" that can be transformed into a wide range of tissue types for medical applications. Growing sufficient numbers of stem cells to satisfy the needs of various treatments is still a significant challenge. Cells used in research laboratories are often selected for their ability to grow rapidly and indefinitely on plastic surfaces but cells for therapy need life like environments and grow in a highly regulated manner. Currently, cells are cultured on surfaces that largely fall into two groups; low cost, bulk materials, exemplified by plastic dishes, or high cost, low volume biological matrices which recreate the conditions found within the body and are increasingly important as more demanding or fragile cell types are used. This project seeks to use a recently developed and patented industrial process to overturn this product landscape by manufacturing engineered protein polymers with advanced cellular functions at low cost. By bridging the gap between traditional polymer science and protein biochemistry we can create a range of matrices to assist the growth of cells for many downstream applications. The 18 month project, supported by the Cell and Gene Therapy Catapult will start by developing one lead product for use in the rapidly expanding stem cell industry. This uses simple coating of plastic surfaces by our protein polymer and has already shown significant advantages over rival technologies in stem cell culture in our hands. Independent validation will enable us to embark on its commercial exploitation to reduce costs and increase efficiency of the whole cell therapy sector. We then intend to further demonstrate its wider applicability for work on muscle, nerve and cartilage by collaboration with leading research groups in the field and with industry. We will also test its usefulness in recreating even more realistic 3 dimensional environments for cell and tissue culture. Finally by exploiting a recent development by us to include large protein modules within the polymer we will create a matrix which can be decorated with any number of cell modifying molecules which are found in natural extracellular environment. This offers an unprecedented opportunity to create bespoke complex cell growth environments in the "test tube" Using both readily commercialisable products and the new intellectual property we intend to move decisively toward either spin out company or licensing agreement at the end of this project.

The European Consortium for Communicating Gene and Cell Therapy Information (EuroGCT) unites 49 partner organisations and institutions across Europe, including the major European advanced therapies learned societies, with the common goal of providing reliable and accessible information related to cell and gene therapy development to European stakeholders. EuroGCT has two major objectives: • To provide patients, people affected by conditions, healthcare professionals and citizens with accurate scientific, legal, ethical and societal information and with engagement opportunities, and thus to support better informed decision-making related to cell and gene-based therapies. • To facilitate better decision-making at key points in development of new therapies and thus enable improved product development, by providing the research community and regulatory and healthcare authorities with an information source on the practical steps needed for cell and gene therapy development. To achieve our aims, EuroGCT will adopt a highly structured system for coordinated management of information related to cell and gene therapy development and, from this, will implement an ambitious programme of online and direct stakeholder information provision and engagement. All outputs will be delivered in 7 European languages, to ensure broad accessibility, and will be rigorously evaluated against measurable objectives throughout the project duration. The proposed consortium comprises leading cell and gene therapy-related organisations and basic and clinical research labs across Europe, including new member states; together with experts in product development, ethical, legal and societal issues, and in evaluating clinical outcomes; patient representatives; and science communicators. It thus is uniquely placed to develop a world-leading cell and gene therapy information resource and to meet the challenge outlined in Topic SC1-HCO-19-2020.

Autologous immunotherapies have revolutionised cancer treatment providing impressive survival benefits in patients with blood cancers. The next generation of personalised immunotherapies using tumour-infiltrating lymphocytes (TIL) aims to overcome efficacy limitations of CAR-T therapies in the treatment of solid tumours. Lack of effective, fast, adaptive, controllable, and scalable manufacturing process remains one of the critical bottlenecks for clinical adoption of such complex personalised cell therapies. In the SMARTER project, Achilles Therapeutics UK Limited, a clinical-stage company developing autologous cell therapies, partners with the centre of excellence for Cell and Gene Therapy Catapult and academic experts in process biomarker discovery (Instituto de Investigation Sanitaria La Fe) and bioprocess sensor development (Leibniz Universitas Hannover). The consortium aims to develop a first-in-class, smart bioprocessing manufacturing platform for personalised autologous cell therapies, implementing for the first time in-line process analytical technologies and smart process control systems. The project exploits breakthrough discoveries of novel T cell expansion process biomarkers and development of new fluorescence spectroscopy sensors for real-time monitoring of critical process parameters, toa enable adaptive process control of the precision TIL biomanufacturing process. After the project, the prototype R&D platform will be ready for follow-up development of the commercial scale bioreactor in GMP environment. The SMARTER platform will critically improve production efficiency, reduce overall costs-of-goods, shorten manufacturing cycle times (shorter vein-to-vein time), decrease batch failures and lead to more consistent and predictable cell therapy product quality. Finally, the innovations will enable clinical implementation of a potential breakthrough personalised adoptive cell therapy for hardest-to-treat solid tumours such as lung cancer and melanoma.

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Awaiting Public Project Summary