EIC and ERC first joint workshop on cell and gene therapy — recordings, presentations and what it means for European research to innovation pathways

Brussels, October 27th 2021
Summary
  • The European Innovation Council and the European Research Council held a joint workshop on cell and gene therapy on 29 June 2021; recordings and slide decks are now available.
  • Organisers say the event aimed to bridge curiosity-driven ERC research and EIC innovation support to accelerate translation into clinical and commercial outcomes.
  • Speakers covered organoids, extracellular vesicles, CAR-T and other cell therapies, vector design and manufacturing, mRNA platforms, gene editors and delivery systems.
  • The EIC is running dedicated Pathfinder, Transition and Accelerator funding lines and a Challenger call on emerging technologies in cell and gene therapy; application rules, budgets and selection criteria are detailed in the EIC work programme.
  • Practical bottlenecks repeatedly flagged were manufacturing scale, regulatory pathways, immunogenicity and capital gaps between discovery and commercial deployment.
  • The workshop underlined the need for active portfolio management, IP clarity and ecosystem supports but also highlighted persistent challenges that funding alone cannot quickly fix.

EIC and ERC first joint workshop on cell and gene therapy

On 29 June 2021 the European Innovation Council (EIC) and the European Research Council (ERC) ran their first joint thematic workshop, focused on cell and gene therapy. The meeting brought together ERC grantees working at the frontier of relevant science, principal investigators involved in EIC projects, EIC Board members and ERC Scientific Council representatives. The purpose was stated as exploring emerging scientific trends and the practical routes by which breakthroughs at the bench could be moved toward clinical development and market implementation.

Why the EIC and the ERC ran this workshop

The event was explicitly framed as an attempt to reduce the gap between frontier, curiosity-driven research financed by the ERC and innovation and scaleup support offered by the EIC. Organisers argued that targeted, coordinated action could help convert EU excellence in fundamental research into competitive technology, companies and health benefits. That framing reflects a widely acknowledged tension in European research and innovation policy: excellent science is necessary but not sufficient for market impact. The workshop was also used to highlight EIC instruments that are intended to support the pathway from idea through demonstration to scaling.

Role of the ERC:The ERC funds bottom-up frontier research with a primary aim of supporting scientific excellence and breakthrough discoveries rather than direct market translation. ERC support frequently generates ideas with later commercial value but the ERC’s remit is research excellence rather than innovation policy.
Role of the EIC:The EIC is designed to support translation and scaleup across the innovation spectrum, including Pathfinder grants for early concepts, Transition grants for maturation toward application, and the Accelerator for company scaleup and market entry. The EIC also operates an investment vehicle (the EIC Fund) to provide equity style finance to selected companies.

Event outputs — recordings, slides and statements

Recordings of the panels and presentations are available and the organisers have posted the slide decks used by presenters. The programme included plenaries and panels spanning fundamental and translational topics from extracellular vesicles to organoids, genome editing scaffolds and manufacturing platforms. Several senior figures made public statements that were circulated with the event materials.

Selected statements appearing with the workshop materials:

Jean - Pierre Bourguignon, ERC president ad interim, stressed the importance of curiosity driven research in producing future innovation and said the EIC and ERC should join efforts to bridge the gap between frontier research and market translation.

Mark Ferguson, President of the EIC Board, emphasised the EIC’s role supporting transformative technologies and the need to build a continuum of instruments so excellent science can translate into impactful applications.

Iordanis Arzimanoglou, EIC programme manager for health and biotechnology, provided a reflective statement after the workshop noting the strong scientific base in Europe and the recurring obstacles—particularly lack of risk capital and gaps in the ecosystem that he said the EIC should help address.

Presentations — who spoke and on what

The workshop featured a broad set of technical talks. Below is a curated list of slide decks and presenters that were made available after the event. These reflect the range of technical approaches discussed and provide primary sources for those wanting to dig into the science and engineering presented at the event.

PresenterTitle or topicPosted
Antonella BongiovanniFrom cell to cell: extracellular vesicles as delivery platform16 Sep 2021
Pieter VaderExtracellular vesicles for therapeutic RNA delivery8 Oct 2021
Hans CleversOrganoids to model, diagnose and treat human diseases20 Sep 2021
Giulio CossuCell therapy for Duchenne muscular dystrophy (DMD)8 Oct 2021
Michele De LucaEpithelial Stem Cells in Cell and Gene Therapy20 Sep 2021
Halas O.Immune Niches for Cancer ImmunoTherapy Enhancement20 Sep 2021
Zsuzsanna IzsvákNon-viral, Sleeping Beauty transposon based gene therapy8 Oct 2021
Roman JeralaCoiled-coil mediated tethering of CRISPR/Cas and exonucleases for enhanced genome editing8 Oct 2021
Prisca LiberaliRegeneration in multicellular systems8 Oct 2021
Matthias P. LutolfEngineering next-generation organoid models8 Oct 2021
Luigi NaldiniGenetic Engineering of Hematopoiesis to Treat Inherited Diseases and Cancer8 Oct 2021
Sven PankeNEWmRNA – Synthetic biology approaches for mRNA8 Oct 2021
Francesca SpagnoliEngineering pancreatic tissue: cells in time and space26 Oct 2021
Thorsten StafforstSite-directed RNA base editing – a marketable innovation25 Oct 2021
Christine MummeryHuman pluripotent stem cell models and vascular topics8 Oct 2021

All slide decks are downloadable from the EIC pages. The set of talks intentionally ranged from discovery science to practical platform technologies and manufacturing, reflecting the EIC and ERC intention to examine both scientific trajectories and translation barriers.

Technical themes and pragmatic challenges surfaced at the meeting

The technical talks covered many approaches. Below are concise explanations of the most repeatedly discussed concepts. These inlinetitle/inlineparagraph pairs are intended to clarify the terminology used across the presentations and to connect the science with translation challenges.

Organoids:Lab-grown three-dimensional structures derived from stem cells that recapitulate features of an organ. Presenters argued organoids are powerful model systems for disease biology, patient-specific testing and drug screening but scaling, reproducibility and regulatory acceptance for use in diagnostics or therapeutic decision making remain barriers.
Extracellular vesicles (EVs):Membrane-bound particles naturally released by cells that carry proteins, RNAs and lipids and can be engineered as delivery vehicles. Advantages include low immunogenicity and native targeting mechanisms. Challenges include heterogeneity, loading efficiency, manufacturing consistency and regulatory classification between biological therapeutics and drug delivery systems.
CAR-T and adoptive cell therapy:Autologous or allogeneic T cells engineered with receptors to target cancer. They have shown clear successes in haematological cancers. Key open problems discussed were extending efficacy to solid tumours, control of off-target toxicity, reducing cost and complexity, and supply chain issues for autologous products.
Sleeping Beauty transposon and non-viral gene delivery:Transposon systems such as Sleeping Beauty mediate stable DNA integration without viral vectors. They can reduce some costs and complexity of viral manufacture but current limitations include efficiency, insertional specificity and delivery to desired cell types in vivo.
mRNA therapeutics (NEWmRNA concepts):mRNA-based modalities rose to prominence following SARS-CoV-2 vaccines. Speakers stressed the difference between chemical synthesis of short RNAs and enzymatic production of long mRNA and discussed routes to diversify the mRNA 'chemical space' and improve manufacturing, stability, capping strategies and immunogenicity.
RNA base editing and site-directed RNA editing:RNA editing approaches, including recruitment of endogenous ADAR or engineered editors, aim to alter RNA transcripts transiently without altering the genome. Proponents highlight dosability and reversibility compared with DNA editing, but challenges include delivery, off-target edits and duration of effect.
Genome editing enhancement strategies (e.g. CC-Exo):Approaches such as tethering exonucleases to CRISPR/Cas via designed coiled-coil peptides can improve knockout efficiencies or delete defined DNA segments. These are still preclinical but illustrate how engineering at the protein or complex level aims to increase activity and specificity.

Across the talks the technical optimism about capability advances was tempered by repeated, practical caveats: manufacturing and quality control at clinical and commercial scales, immune responses to vectors, standardisation and qualification of in vitro models, the difficulty of moving from promising small-scale results to robust clinical grade processes, and the complexity and cost of regulated pathways to patients.

Policy, funding instruments and where these activities sit in the EU innovation architecture

The workshop was also a platform to connect detailed technical themes with the mechanics of EU research and innovation funding. Key funders and instruments were discussed and the EIC used the event to highlight its instruments and associated eligibility rules and evaluation approach.

ERC investment in cell and gene therapy:The ERC reported it funded more than 150 projects in cell and gene therapy since 2014 with a combined ERC contribution in excess of €300 million. ERC grants are competitive and curiosity-driven; the ERC Proof of Concept mechanism is a separate scheme to test commercial potential of ERC-funded results.
EIC instruments overview:The EIC operates three core direct funding streams relevant here: Pathfinder for high-risk science-towards-technology (proof of principle), Transition for validating and maturing technologies into demonstrators and market plans, and the Accelerator for company support and scaleup combining grants and an investment component through the EIC Fund. The EIC also provides Business Acceleration Services including coaching and investor matchmaking.

Below are practical numbers, deadlines and summary selection arrangements pulled from the EIC Work Programme and the call materials published for 2021. These are important for applicants but also indicate how the Commission intends to allocate resources across different stages of translation.

Instrument / callWho can applyTypical funding and formKey 2021 timelines and indicative budgets
EIC Pathfinder (Open)Collaborative consortia (min three legal entities across MS/AC) or single entities in some callsGrants for R&I actions to reach proof of principle. Typical request up to EUR 3M (EUR 4M for some challenges). 100% funding of eligible costs.Call deadline 19 May 2021. Indicative budget EUR 168M for open call. Separate Pathfinder Challenge calls 27 Oct 2021 with EUR 132M across challenges.
EIC Transition (Open)Single applicants (SME or research organisations) or small consortia (2-5 partners) building on eligible Pathfinder, FET or ERC Proof of Concept resultsGrants up to EUR 2.5M typically. 100% funding to raise TRL to ~5-6 and prepare market readiness.Call deadline 22 Sep 2021. Indicative budget EUR 59.6M for open call; additional challenge budgets EUR 40.5M.
EIC Accelerator (Open, blended)Single startups/SMEs (or in exceptional cases small mid-caps). Natural persons may apply intending to form companiesBlended finance: grant component up to EUR 2.5M (reimburses eligible R&D/demo costs up to 70%) and an investment component (equity/quasi-equity) EUR 0.5M up to EUR 15M. Investment terms negotiated by EIC Fund.Short applications any time. Full applications cut-offs 9 June 2021 and 6 Oct 2021. Indicative budget EUR 592.5M for the Accelerator programme in 2021, portion from NextGenerationEU for recovery-related priorities.
ERC Proof of ConceptPIs with an existing ERC grantLump sum EUR 150,000 for up to 18 months to explore commercial or societal potential.ERC PoC runs with cut-off dates and two-stage evaluation. See ERC webpages for deadlines and ethics/evaluation details.

The EIC also launched a dedicated Pathfinder Challenge on emerging technologies in cell and gene therapy. Challenge calls are intended to form managed portfolios of projects that together target specific strategic outcomes and are overseen by EIC Programme Managers charged with active portfolio management.

Selection and evaluation — how proposals are assessed

The EIC uses tailored evaluation approaches for each instrument. The following summaries come from the 2021 EIC Work Programme and the related call documentation. Applicants should always consult the relevant call text for the final rules and thresholds.

Pathfinder evaluation (open and challenge)

Pathfinder proposals are peer assessed by external experts and through an evaluation committee. The three evaluation criteria and thresholds used in 2021 were:

CriterionThresholdWeight / focus
Excellence (vision, novelty, plausibility, interdisciplinarity)4/560%
Impact (innovation potential, exploitation, communication and open science)3.5/520%
Quality and efficiency of implementation (consortium, work plan, resource allocation)3/520%

Successful Pathfinder Challenge proposals are also selected in the context of building a coherent portfolio, so the evaluation committee considers not only scores but also how proposals contribute to the Challenge’s strategic objectives.

Transition evaluation

Transition proposals (which must build on eligible Pathfinder, FET or ERC PoC results) are assessed in two phases: an initial remote expert evaluation and, for top-ranked applicants, a face to face interview with an EIC jury that applies a GO / NO-GO decision. Criteria and thresholds used in 2021 included technological breakthrough, business and market fit, team and milestones, with thresholds of roughly 4/5 for the principal criteria.

Accelerator evaluation

The Accelerator uses a short-application screening followed by full applications for successful short applicants. Full applications are remotely evaluated; those passing are invited to face to face jury interviews. For Accelerator selection the jury makes a GO / NO-GO recommendation. The Accelerator criteria mix technology and market assessment, team and investment risk. Important features are:

Seal of Excellence:In the Accelerator, a high-quality proposal that meets excellence and impact thresholds but cannot be funded due to budget constraints may receive a Seal of Excellence. Holders of the Seal are intended to receive help accessing other national or regional funding sources and Business Acceleration Services.

The Accelerator offers tailored coaching and an AI-based IT platform to help applicants prepare full proposals and to support investor matchmaking.

EIC Fund and blended finance — practical notes and caveats

The EIC Fund handles the investment component of Accelerator blended finance and is responsible for negotiating investment terms after an initial GO decision. In 2021 EIC material stated leverage objectives for Fund investments and claimed the Fund helps attract additional private capital into supported companies. Investment decisions require due diligence and Board approval at the Fund and the Commission retains oversight. Applicants should note several practical points raised in the workshop and documents:

EIC Fund investment scope and constraints:The investment component is typically equity or quasi-equity and ranges from EUR 0.5M up to EUR 15M under the 2021 framework. For small mid-cap companies (up to 499 employees) only the investment component is available; grant-only or grant-first options exist for some applicants, but these require specific evidence of ability to finance deployment or milestone-based progression to investment.

Negotiations and due diligence can extend the timeline. Grant agreements for the grant component may be signed earlier while investment terms are negotiated in parallel. The EIC Fund seeks to crowd in follow-on investors but applicants should not treat the EIC decision as a guaranteed or immediate financing line without understanding the commercial due diligence that follows.

ERC Proof of Concept, ethics and review

The ERC Proof of Concept (PoC) grant is a separate mechanism for ERC grantees to investigate commercial or societal potential of ERC-funded projects. Important features:

ERC PoC funding and eligibility:PoC grants are awarded to principal investigators who already hold an ERC frontier research award. The 2026 work programme (and previous arrangement) envisages a lump sum of EUR 150,000 for up to 18 months. Eligibility is limited to PIs whose proposals derive substantially from their ERC research.
Ethics review for PoC and Horizon Europe funded projects:Ethics screening and assessment are mandatory and structured in multiple stages including pre-screening, screening by independent reviewers and full ethics assessment for complex cases. Activities involving human embryonic stem cells carry additional legal requirements under EU rules.

Practical items applicants repeatedly asked about

Workshop materials and EIC documentation clarify procedural items that commonly concern applicants. Below are practical details pulled from the 2021 documents:

TopicPractical detail
Application portalsSubmissions use the EU Funding & Tender Opportunities Portal and EIC AI-assisted IT platform for Accelerator full proposals.
Deadlines and cut-offsShort applications to the Accelerator are accepted any time; full continuous and cut-off schedules are in the EIC work programme. Main 2021 full application cut-offs were 9 June and 6 Oct 2021.
Resubmission rulesThe Accelerator implements limits on resubmission frequency; specific rules depend on whether rejection occurs at short-application, remote full evaluation or post-interview stages.
CoachingCompanies awarded a GO at the short stage receive coaching days; applicants can also select a business coach from a pre-qualified pool.
Open Science and IPPathfinder requires open access publications while allowing IP protection; Transition and Accelerator expect a clear IP strategy and formal protection where appropriate.

Data protection, third party processors and retention

The EIC and the Executive Agency responsible for implementation (EISMEA) publish specific data protection notices for the Accelerator. Key operational points that applicants should note:

Legal basis and controllers:Processing is carried out under Regulation (EU) 2018/1725. The European Innovation Council and SMEs Executive Agency (EISMEA) and the EIC Fund are joint data controllers for Accelerator processing related to submissions, selection and management.
Third party processors involved:The data protection notice identifies several entities that process data on behalf of the controllers for investment due diligence and operational tasks, including the European Investment Bank (EIB), Alter Domus Luxembourg S.à r.l., and Dealflow.eu BV. Applicants giving consent should note these named processors and their roles in due diligence, investor matchmaking and paymaster functions.
Retention:Retention periods disclosed in EISMEA notices included: personal data of selected experts 7 years; funded Accelerator applications retained for 10 years after programme closure; unsuccessful applicants retained up to 5 years. Limited categories of data may be kept for research or statistical purposes up to 25 years, subject to safeguards.

What the workshop highlighted about the translational gap — a pragmatic critique

The EIC-ERC workshop models a sensible policy approach: match frontier discovery and innovation funding, surface cross-cutting technical bottlenecks and create managed portfolios to align research with later-stage development. The scientific talks showed Europe has deep competence across multiple approaches. But the meeting also made clear that funding alone will not automatically deliver broad clinical or commercial rollout. Practical obstacles repeatedly surfaced:

1) Manufacturing and scale. Several speakers warned that many technologies are promising at small scale but face a step change in complexity, expense and regulatory burden when moved to GMP manufacturing and clinical supply. This is not just money, it is process control, quality systems and trained workforce.

2) Regulatory and evidentiary expectations. Translational plans require early conversations with regulators and realistic timeframes for safety, immunogenicity and long-term follow up. For some modalities, the regulatory path is still evolving.

3) Ecosystem and capital. Multiple presentations and discussants flagged a shortage of patient risk capital and ecosystem connectors in Europe compared with the US. EIC instruments and the EIC Fund aim to address this but investors remain cautious at the preclinical/ early clinical stages.

4) Standardisation and qualification of models. Organoids and organs-on-chip show promise for personalised testing and disease modelling, but the field lacks widely accepted standards and validated qualification pathways for regulatory use or to replace animal models.

Next steps and where to find materials

The EIC and ERC indicated they plan to continue collaboration. The workshop materials — video recordings and slide decks — were published across late summer and autumn 2021 on EIC and ERC websites. Interested researchers and innovators should consult the EIC funding portal, the ERC Proof of Concept pages, and the EIC Work Programme for precise application rules and deadlines.

Readers who want to go deeper should download the individual slide decks referenced in the presentation table above, review the EIC work programme sections on Pathfinder, Transition and Accelerator, and read the ERC Proof of Concept guidance if they are ERC grantees considering commercial follow-up.

Concluding note — promise and realism

The EIC-ERC workshop underscored that Europe hosts a rich ecosystem of discovery and early innovation in cell and gene therapy. The combined approach — aligning frontier science with targeted innovation instruments, active portfolio management and investor engagement — is the right direction for policy makers who want more breakthroughs to reach patients and companies. However, the workshop also reinforced that strategic funding must be combined with investments in manufacturing capacity, clinical development infrastructure, regulatory pathways and ecosystem building to turn scientific promise into sustainable patient benefit and market impact. Policymakers and funders should avoid overly optimistic narratives and instead plan for multi-year, multidisciplinary programmes that link science, process engineering, regulation and capital.

Selected references and where to read official materials

Primary sources for the statements, call details and slide decks used above are the EIC website pages for the workshop and the EIC Work Programme 2021, the ERC Proof of Concept guidance pages, and the EISMEA data protection notices. For grant rules applicants should always consult the official call texts on the EU Funding & Tenders Portal.