EFPIA Biomanufacturing Satellite Session: Concept Paper 2020 Updates

The EFPIA MQEG Biomanufacturing sub-team has published a number of position papers and is currently progressing industry consensus activities on other key topics. Updates will be given on the progress of recent position papers relating to certain key topics such as drug device combination products, antibody drug conjugates and polysorbates. A subsequent session comprising of presentations followed by a panel discussion will be dedicated to AAV: Current Status, Manufacturing Challenges and Opportunities. The number of clinical studies in which adeno-associated viral (AAV) vectors are used for in vivo gene transfer is steadily increasing. Excellent safety profile and high transduction efficiency for numerous target tissues speak for AAV vectors in in vivo gene therapy in diseases such as coagulation disorders, hereditary blindness and neurodegenerative diseases. Gene therapies are predicted to have a similar increase kinetics as has been the case for monoclonal antibodies since the beginning of the 1980s. However, there are still a number of hurdles to be overcome in the field of manufacturing and control before a similar market penetration as for monoclonal antibodies can be envisaged. Especially the complexity of manufacturing and analytics restrict a broad application of the technology. The current main problem for gene therapy with AAV is the low yields and small manufacturing scales. Depending on the selected manufacturing technology (suspension/adherent cell culture, flatware/fixed bed, etc.), the manufacturing units may have to be significantly multiplied for larger numbers of patients, which can only be limited by significantly intensifying the process. This is also currently being pursued for the production of monoclonal antibodies. This satellite symposium will use case studies of successful AAV-based therapies to familiarize participants with the basic therapeutic mechanisms but also shed light on manufacturing-related challenges and opportunities in particular whilst also giving an overview of the regulatory challenges of AAV therapeutics.