Advanced Therapies 2023 Congress
by Iheoma Anosike, Consultant
at Scendea
Introduction
Scendea were thrilled to attend the two-day Advanced Therapies 2023 Congress, held at ExCel London, on 14 and 15 March. The event is Europe’s largest cell and gene conference and exhibition, and brings together experts in the field of advanced therapy medicinal products (ATMPs) who are at the forefront of efforts aiming to improve patient access to quality, safe and effective cell and gene therapies.
Day 1
A presentation on novel approaches to overcome manufacturing challenges typically encountered in advanced therapy development kicked off the event, and included a discussion on the significance of computational fluid dynamics as a useful tool to facilitate the accurate scale up and production of novel cell and gene therapy products. A panel discussion on the key factors to consider in order to aid the improvement of patient outcomes during the clinical development of autologous and allogeneic cell therapies then followed. The conversations covered the implications of donor health on cell therapy potency particularly for autologous cell therapies, and the potential for biomarkers to be leveraged as a tool to indicate donor and cell health.
A plethora of bitesize presentations from small-medium sized biotechnology and start-up companies, large pharmaceutical companies, clinicians, researchers and academics were held during the afternoon of Day 1, and carried on throughout the second day of the conference. The presentations spanned various topics relating to areas such as gene therapy development, cell therapy manufacture and viral vector manufacture and attendees were able to choose sessions of interest to attend.
The key issues to consider in order to overcome challenges often posed by cell therapy manufacture was aptly illustrated by an interesting case study on human ventricular progenitor cells undergoing development as a regenerative cell therapy for heart failure. The importance of shifting away from manual and open manufacturing processes which are commonly associated with difficulties relating to product contamination, towards modular, closed and automated workflow systems instead, was discussed.
An additional, highly informative presentation explained the benefits of multi-dimensional mass spectrometry as a method allowing for the extended characterization of adeno-associated virus (AAV) vector capsids. Mass spectrometry can be used as an effective identity test during the release testing of AAV vectors. The method allows for the characterisation of virus capsid proteins VP1, VP2 and VP3, and identification of impurities such as host cell proteins.
Day 2
The morning of the second and final day of the conference started with a discussion panel including stakeholders from the US Food, Drug and Administration (FDA) and Pharmaceuticals and Medical Devices Agency (PMDA) to discuss potential revisions to the ATMP Regulation 1394/2007 aimed to help promote the development of ATMPs and improve patient access. The panel considered international efforts that are currently being made to harmonise requirements for the development and approval of ATMPs in order to improve worldwide access to promising advanced therapies. The significance of the costs associated with these therapies was deliberated, and it was agreed that health technology assessment models would need to be revisited in order to help inform decisions regarding paying for these products. It was concluded that in the lead up to any future revisions to the ATMP legislation, the potential of the current and existing framework should be maximised.
A detailed discussion on regulatory complexities associated with allogeneic cell therapies saw the safety risks associated with novel allogeneic cell therapy products explained, including the potential for cell differentiation and associated tumorigenicity risks. Risk reduction strategies were discussed, including initiating trials with autologous cells before transitioning to allogeneic cells, the use of immunosuppressants and/or rescue medications and safety/suicide gene incorporation in cell therapies. Manufacturing challenges associated with allogenic cell therapies were also considered, including difficulties with human leukocyte antigen (HLA) typing, HLA mismatch and the characterisation of donor cells/starting material, including donor to donor variability. Differences between the US and EU with regards to adventitious agent testing requirements for donor cells were also presented. In the US for example, an FDA cleared test for anti-cytomegalovirus (CMV) antibodies must be carried out, whereas in the EU, this is dependent on the donor history.
An exciting presentation on novel non-viral vectors for gene therapies rounded off the afternoon, which discussed the clinical utility of extracellular vesicles. A case study on extracellular vesicles derived from allogeneic mesenchymal stem cells was presented. These extracellular vesicles are used in a cystic fibrosis transmembrane conductance regulator (CFTR) mRNA gene therapy and are administered to patients via aerosol delivery to target pulmonary and immune cells affected by cystic fibrosis. The vesicles are pegylated to facilitate penetration across the airway mucus gel layer, which is often a key barrier to successful inhaled gene therapy.
Conclusion
The discussions and presentations delivered at the Advanced Therapies 2023 conference illustrate that significant progress has been made in terms of the current understanding of approaches to improve the development, manufacture and eventual commercialisation of advanced therapy medicinal products. Globally, regulatory agencies are committed to fostering a harmonised approach to create a more favourable landscape to facilitate the development of advanced therapy medicines, whilst also ensuring that safety, quality and efficacy standards are not compromised. It is hoped that these efforts will contribute to increasing patient access to these innovative and promising therapies.
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