EIC convenes Cell and Gene Therapy community to address manufacturing, vectors and translation barriers

Brussels, November 30th 2023

Summary

›On 27 October 2023 the EIC held a Cell and Gene Therapy Symposium in Brussels as part of the ESGCT Congress to showcase its Pathfinder CGT portfolio and build connections across research, industry and policy.
›The CGT Pathfinder portfolio groups nine projects into two subportfolios: cell therapy manufacturing and gene delivery systems, with coordinated portfolio activities focused on technology, regulation and exploitation.
›Speakers and attendees highlighted persistent bottlenecks in vector manufacturing, scaling, standardization and regulatory clarity that constrain clinical translation and commercial roll out.
›EIC emphasised proactive portfolio management rather than isolated project funding and published a strategic plan for the CGT portfolio to capture shared opportunities and tackle crosscutting challenges.
›The symposium produced resources and a recording and sits against a backdrop of major public and private investments in biomanufacturing globally that increase pressure to translate technical advances into industrial capacity and patient access.

EIC Cell and Gene Therapy Symposium: purpose and framing

On 27 October 2023 the European Innovation Council organised a Cell and Gene Therapy (CGT) Symposium in Brussels. The event was held as an extended activity of the EIC Pathfinder Challenge portfolio on CGT. It was organised by Dr. Iordanis Arzimanoglou, then Programme Manager for Health and Biotechnology at the EIC, and convened researchers, industry representatives, policy makers and EIC-funded project teams. More than 100 participants attended across industry, public sector bodies and research institutions. The meeting aimed to explain the EIC's portfolio approach, demonstrate the value of proactive portfolio management, and to strengthen links across actors involved in CGT.

Event details and participants

The symposium took place within the context of the 30th Annual Congress of the European Society of Gene and Cell Therapy. It featured three keynote speakers who are established figures in the CGT field: Professor Hans-Peter Kiem, Dr. Michael Wenger and Professor Seppo Ylä-Herttuala. High level policy makers also joined the meeting. The session was positioned as the capstone activity of an EIC 'CGT campaign month' which included communication outputs such as an EIC podcast episode titled 'Fighting cancer with Cell and Gene therapy'. A recording of the symposium and supporting documents were published by the EIC.

Why the EIC organised a portfolio rather than single-project effort

The EIC has been explicit that funding individual projects is not enough to address systemic barriers facing advanced therapies. The EIC's CGT strategy frames the portfolio as a coherent collection of projects that share components or address overlapping building blocks. The intention is to create critical mass so that crosscutting problems can be surfaced and tackled, for example manufacturing scale up, vector quality, regulatory uncertainty, and exploitation pathways toward the clinic and market.

Shared component model:The EIC used a 'shared component' logic when selecting projects under the Horizon EIC Pathfinder 2021 challenge. Proposals were mapped to building blocks and projects that converged on common technical or translational components were grouped into a portfolio. The approach produced two subportfolios aligned to tangible bottlenecks in the field.

The CGT Pathfinder portfolio: subportfolios and projects

Nine projects selected under the 2021 Pathfinder Challenge were organised into two subportfolios. The grouping was intended to concentrate know how and surface common needs for technology development, regulatory pathways and exploitation.

Subportfolio	Project acronym	Project focus
Advancing cell therapy manufacturing and products to a clinical stage	AiPSC	AI-powered platform for autologous iPSC manufacturing
Advancing cell therapy manufacturing and products to a clinical stage	SMARTER	Smart manufacturing for autologous cell therapies via biomonitoring and advanced process control
Advancing cell therapy manufacturing and products to a clinical stage	MUTAVAC	Mutanome based stem cell vaccine for cancer
Advancing cell therapy manufacturing and products to a clinical stage	T-FITNESS	Fine-tuning T cell networks of exhaustion with synthetic sensors
Advancing cell therapy manufacturing and products to a clinical stage	X-PAND	Ex vivo expansion and multiomics profiling for safer hematopoietic stem cell gene therapies
Advancing cell therapy manufacturing and products to a clinical stage	PAT4CGT	Automated online monitoring and control for cell and gene therapy manufacturing
Improving gene delivery systems (vectors)	EdiGenT	Prime editing and non-viral delivery strategies for gene therapy
Improving gene delivery systems (vectors)	AAVOLUTION	Next generation AAV vectors for liver-directed gene therapy
Improving gene delivery systems (vectors)	NOVISTEM	Non-viral gene modified stem cell therapy

Portfolio level priorities

The EIC described three crosscutting target areas for coordinated work across the CGT portfolio. These are technological development, regulatory preparedness, and exploitation pathways to clinical application and commercial uptake.

Technology:Focus on improving manufacturing processes, quality control, process monitoring, scalable production of vectors and cell therapies, non-viral delivery systems, and process automation. The projects include work on AI-assisted iPSC manufacture, online PAT tools, and next generation AAV vectors.

Regulatory:Work to reduce regulatory uncertainty by creating shared evidence packages, aligning analytics and assays, and surfacing issues that consistently slow approval pathways. The portfolio model is meant to generate lessons that regulators and sponsors can use to harmonise expectations.

Exploitation:Activities to improve translation include investor engagement, business coaching, intellectual property and licensing strategies, and connecting projects to national funding bodies and networks that can support later stage development and manufacturing.

EIC activities and outputs from the symposium

The symposium served several practical purposes. It presented the CGT Pathfinder portfolio to the ESGCT community, demonstrated the EIC's proactive portfolio management through a panel of EIC-funded projects, and created networking opportunities across the CGT ecosystem. The event concluded the EIC CGT campaign month and the EIC published supporting documents and recordings.

Documents and outputs published by the EIC included a presentation by Dr. Iordanis Arzimanoglou and a symposium brochure. A recording of the session and a podcast episode were made available to increase visibility of the work.

Broader EIC CGT community across EIC instruments

Beyond the Pathfinder projects, the EIC highlighted a larger community of related projects funded across EIC instruments including Accelerator, Transition and Pathfinder projects that intersect with CGT themes. These projects cover clinical validation, oncolytic viruses, allogeneic cell therapies, RNA therapeutics, novel delivery carriers, manufacturing automation and gene editing technologies.

Programme	Selected projects mentioned
EIC Accelerator and Transition	CD20CARNAPT - iTANK, CELLULARITY, EuroXpand, FIGHT-DM1, I-CREATE, LAKHESYS, Smart ProTcell, TREG, FIBREX, TAONas-LUAD, MACOV, TRACTION, CARxALL, TraffikGene-Tx
EIC Pathfinder	AiPSC, X-PAND, MUTAVAC, SMARTER, PAT4CGT, T-FITNESS, EdiGenT, AAVOLUTION, NOVISTEM, INCITE, ULISES, I-GENE, iAds, NANO-ENGINE, HyperTargIPS-NK, CAR T-REX

Technical concepts explained

AAV vectors and production challenges:Adeno-associated virus vectors are commonly used for in vivo gene delivery. Producing AAV at clinical scale is technically demanding. Common issues include low yields, heterogeneous particle quality, impurities and empty capsids that lack therapeutic DNA. Downstream purification and assays to characterise potency are also immature relative to other biologics. These bottlenecks drive cost and limit patient access.

Non-viral delivery and prime editing:Non-viral delivery aims to avoid limitations of viral vectors by using synthetic carriers, nanoparticles or physical methods to deliver nucleic acids. Prime editing is an advanced gene editing technique that can make precise sequence changes without creating double strand breaks. Combining non-viral delivery with prime editing could reduce immunogenicity and manufacturing complexity but faces efficiency and safety barriers for in vivo use.

Autologous versus allogeneic cell therapies:Autologous therapies use a patient’s own cells and are inherently personalised but require bespoke manufacturing and logistics. Allogeneic 'off-the-shelf' approaches aim to deliver standardised products at scale but face immune compatibility and persistence challenges. Both approaches need improvements in manufacturing control, potency assays and cost reduction to become widely accessible.

Market and policy context highlighted at the event

Speakers and slides at the symposium stressed a persistent gap between scientific advances and broad patient access. The market for gene therapies remains small relative to the potential. For example, regulatory approvals for gene therapies have been limited and many candidate therapies are still in clinical trials. Manufacturing capacity, standardisation of assays, supply chains and regulatory frameworks were singled out as critical constraints.

The EIC also positioned the portfolio against global moves to expand biomanufacturing. The symposium materials referenced initiatives and investments such as US federal funding for national biomanufacturing, regional manufacturing investments in Europe and private foundation commitments. These build pressure to translate university and early stage advances into industrial capacity.

Analytical takeaways and caveats

The EIC’s portfolio approach is a logical step to tackle problems that single projects cannot fix alone. There are advantages to concentrating expertise and sharing lessons on assays, analytics and regulatory interaction. The Pathfinder portfolio is well targeted to technical bottlenecks that frequently block clinical translation.

However several risks remain. Pathfinder funding is primarily early stage and may not be sufficient to carry technologies through the costly late stage development, manufacturing scale up and regulatory filing. The translation gap between proof of concept and market entry often requires additional capital and manufacturing partnerships that are not guaranteed. Regulatory harmonisation across EU member states remains work in progress which can slow multicountry clinical trials and reimbursement planning. Finally, rising public and private investments raise expectations for rapid progress. That increases pressure on grant programmes to provide predictable follow-on funding and to coordinate with national initiatives and industrial partners.

Practical implications for stakeholders

For researchers and SMEs: portfolio activities can provide exposure to regulators, investors and industrial partners. Teams should plan for manufacturing strategies early and prioritise analytics that are likely to be accepted by regulators. For funders and policy makers: bridging instruments that link Pathfinder discoveries to industrial scale and regulatory readiness will be necessary. For patients and payers: the core issues determining access will be manufacturing cost, supply reliability and value frameworks used in reimbursement decisions.

Next steps and signals to watch

Key items to monitor following the symposium include implementation of the EIC CGT strategic plan, concrete portfolio activities that produce harmonised assays or shared manufacturing pilots, progress in securing investment partners for EU funded equity components, and whether national and European industrial initiatives align with the technologies emerging from the Pathfinder projects. The EIC has published materials and a recording from the symposium which can be used to track commitments and follow up activities.

Resources and further reading

The EIC published a recording of the Cell and Gene Therapy CGT community symposium and made available presentation slides and a brochure dated 27 November 2023. The EIC podcast episode 'Fighting cancer with Cell and Gene therapy' formed part of a visibility campaign in support of the CGT portfolio. Relevant EIC pages contain lists of Accelerator, Transition and Pathfinder projects working in related areas.

About the organiser

Dr. Iordanis Arzimanoglou, who organised the symposium, served as EIC Programme Manager for Health and Biotechnology from June 2020 to May 2024. He led the CGT portfolio work and has a background in molecular genetics and academic leadership. During his tenure he coordinated strategic portfolio activities including workshops on licensing, IP, clinical translation and proactive engagement with national and regional innovation actors.