European Innovation Council’s strategic actions: A game-changer for cell and gene therapy
Authors: Iordanis Arzimanoglou
Journal: Molecular Therapy (2022)

Cell and gene therapy (CGT) continues to evolve as we enter the era of precision medicine to treat human disease. Cell-based therapies have revolutionized the treatment of some blood cancers, but new evidence is required to demonstrate effectiveness in the treatment of solid cancers as well. Gene therapy, on the other hand, has yet to reach its full potential and therefore remains a top priority in genomic medicine. Some companies believe that, after having achieved a robust proof-of-concept, clinical development and downstream interaction with regulatory agencies will be a straightforward process. The reality, however, can be quite different. The entire process from concept to clinical grade viral vector under good manufacturing practice standards is a very demanding one. There is a constant need for technological improvements to overcome challenges in maintaining productivity, scaling up production, or undergoing tests using advanced analytics to meet regulatory requirements. The European Innovation Council (EIC) in the European Commission aims to support the CGT journey from concept to implementation in the clinic. In late 2020, as newly appointed program manager responsible for health and biotechnology, I began my mission to ensure that the EIC strategically and systematically directs thematic support toward projects and companies working in CGT, due to the increasing importance of this field for Europe. In this context, the European Research Council (ERC) portfolio for CGT projects was screened, and the findings were presented to a working group of ERC Scientific Council and EIC Board members. It was agreed that this dynamic field of research should be further explored, with the topic selected for the first-in-a-series of joint EIC-ERC events exploring emerging scientific trends with high innovation potential. As a result of that, the first ever EIC-ERC joint CGT event became a reality, thanks to the generous support of specific ERC Scientific Council and EIC Board members who comprised an organizational committee, as well as the commitment of EIC and ERC project officers. The recordings and presentations of this event, which attracted the elite of European scientists and innovators in the field and had over 240 attendees, are available (https://eic.ec.europa.eu/news/cell-and-gene-therapy-first-eic-erc-workshop-recordings-and-presentations-available-now-2021-10-27_en), as well as a summary (https://eic.ec.europa.eu/statement-eic-programme-manager-iordanis-arzimanoglou_en). In parallel, the EIC issued a call on emerging technologies in CGT targeted at developing more effective CGT treatments and at overcoming critical challenges faced by researchers and innovators (https://eic.ec.europa.eu/eic-funding-opportunities/calls-proposals/eic-pathfinder-challenge-emerging-technologies-cell-and_en). As documented by the 2021 EIC Work Program (WP), a relevant “challenge call” was designed to specifically target the following CGT areas: (1) advancing cell therapy manufacturing and products to a clinical stage, (2) improving adoptive cell therapies, (3) identifying next-generation cell therapies for cancer, (4) applying cell therapy to treat cancer patients in a personalized manner, (5) improving the effectiveness and lowering the risks of gene delivery systems (vectors), and (6) improving gene therapy manufacturing processes and production. In response to this call, 134 eligible proposals were received, which underwent remote evaluation by external experts, followed by the selection of the proposals, which will eventually form the CGT portfolio, by the evaluation panel using a strategic intelligence approach. We were delighted to see two distinct patterns emerge in the 12 top-ranked proposals, which now comprise two distinct sub-portfolios. Interestingly, the first corresponds to areas (1) and (4) while the second to areas (5) and (6), as described above. The portfolio-building exercise enabled us to understand that Europe already has strength in these two areas, and that we at the EIC strive to support them in their effort to achieve critical mass and global excellence. Earlier this year, the EIC published a second “challenge call” on RNA-based therapeutics and diagnostics, focused on tackling challenges relating to mRNA-based systems, such as accurate delivery, and on newer types of RNA including tRNA.1Dolgin E. tRNA therapeutics burst onto startup scene.Nat. Biotechnol. 2022; 40: 283-286Crossref PubMed Scopus (4) Google Scholar Furthermore, our WP has foreseen targeted support, for start-ups and SMEs in the field, with EIC Accelerator grants, equity investments, and business acceleration services. The aim is to develop strategies and new concept-based solutions to mitigate longstanding obstacles at the phase 1 trial level, such as in vivo administration and safety, as well other key issues related to post-COVID supply and distribution of CGT therapeutics. This is only the beginning of the CGT innovation journey, as part of ongoing activities and strategic considerations leading to value creation in the CGT field with a potential pan-European impact. We envisage that 5 years from now the EIC, with its targeted funding and strategic intelligence-based non-funding support, will contribute to Europe becoming a global player in the CGT field. This is part of a broader EIC strategy whereby program managers select and recommend focused thematic areas with high disruptive potential for targeted funding, using the power of strategic intelligence and after following the global industry trends in their thematic sector. This can potentially lead to the establishment of the EIC as the most effective catalyst of breakthrough science to disruptive innovation.