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EIC Pathfinder challenge: Emerging technologies in cell and gene therapy

Details

Status
Open
Reference
HORIZON-EIC-2021-PATHFINDERCHALLENGES-01-03
Publication date
Opening date
Deadline model
Single-stage
Deadline date
Funding programme
Horizon Europe – the Framework Programme for Research and Innovation

Description

Cell and gene therapy (CGT) are widely accepted as top biomedical trends for over three years now and continue to evolve in their use for treating human diseases. CGTs are expected to increasingly shape the medical treatment and diagnosis, as we are approaching the era of precision medicine. Cell-based therapy is a promising strategy for effective treatment across a wide range of diseases though the focus so far, has primarily been on cancer, e.g. Chimeric Antigen Receptor T-cell (CAR-T) therapy made from removing T cells from individual patients, engineer them to be able to recognize and kill cancer cells before re-administer them to the same patient. CAR-T cell therapy is widely regarded as having revolutionised the treatment of some blood cancers. Recent research evidence suggests that cell therapy can effectively apply to solid cancers as well.

Gene therapy, on the other hand, is yet far from having revealed its full potential and, therefore, innovative gene therapies remain 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 easy. The reality, however, is that the whole process from concept to commercialisation, from research to commercial grade viral vector under GMP standards, is a very demanding one, with the constant need for technological improvements to successfully overcome challenges such as increasing accuracy/specificity and scaling up the production or the release of tests that must be completed before use in patients. Finally, combined cell- and gene-based approaches in preclinical studies, is a relatively new bio-trend that is increasingly gaining the interest of cell and gene therapy scientists worldwide.

With this Pathfinder Challenge, EIC strategically aims at reinforcing critical components of the European cell and gene therapy community, such as focused research consortia, start-ups and spinoffs, in their ability to compete and sustain in this fiercely competitive field, full of challenges and obstacles all along the way from discovery to the manufacturing step. Proposals submitted to this call should effectively address exactly that, by proposing convincing technological solutions and/or new breakthrough concepts that go far beyond the current state-of-the-art.

Proposers are invited to submit disease-specific or non-disease-specific proposals, focused on emerging technologies or technological solutions aimed to overcome the current cell and gene therapy challenges in one or several the areas listed below, but without being restricted only to these areas.

Advancing cell therapy manufacturing and products to a clinical stage

Advanced technological solutions that can effectively support the GMP manufacturing step of cell therapy e.g. in terms of speed and cost effectiveness.

 Novel cell therapy products, targeted to frequent diseases such as cancer and organ failure but also to less frequent diseases like immunodeficiency disorders, that can be used by clinical stage biopharmaceutical companies.

Cell therapy technological solutions that can improve important constraints in handling highly concentrated and complex formulations of recombinant biologics, such as controlled release of therapeutics and injectability.

Improving adoptive cell therapies (CAR-T, TCR, TIL)

New technological solutions that would help to improve current adoptive cell therapy approaches: i) by lowering the high cost and complexity of the procedure; ii) by overcoming the long known rejection problem observed in the off-the-shelf or allogeneic CAR-T cell therapies; iii) by targeting the CAR into one location, which would take away the variability problem (the CAR randomly goes into the genome of cells resulting in variable levels of potency) and iv) by developing CAR-T based new immunotherapeutic approaches against solid tumours with the use of monoclonal antibodies.

Identifying next generation cell therapies for cancer

New technological platforms that can contribute to identifying next-generation cell therapies for cancer (finding new targets for the engineered immune cells to home in on, or novel source of cells for new therapeutic approaches) as well as improving existing therapies to make them more efficient and safer. The latter could include naive fully functional T-cells.

Applying cell therapy to treat cancer patients in a personalised manner

Advanced technological solutions that would enable to apply cell-based therapies to treat patients in a personalised/precision manner. Single cell-based approaches (analysing DNA, RNA, epigenetic marks, proteins, metabolites used in combination with single cell sequencing, single cell imaging and spatial profiling) in particular to allow to map the presence of individual cells in the tumour environment.

New technological solutions including lab-grown cancer organoids or organs-on-a-chip which would allow to test the patient’s response to various cell therapies and drugs, alone or in combination, prior to the initiation of the treatment, are sought.

Improving the effectiveness and lowering the risks of gene delivery systems (vectors)

Novel gene therapy approaches using the power of CRISPR-Cas or other molecular machineries leading to more effective and robust gene delivery systems (vectors) and/or more precise and reliable correction of genetic mutations.

Technological approaches that can tackle long lasting challenges in gene therapy (e.g. the transient instead of stable expression of the transfected gene).

New technological solutions to reduce toxicity, as a result, of administering repetitive doses of viral vector(s) to patients in clinical trials.

 New or improved gene delivery vehicles using next generation AAV or other recombinant vectors with the ability to target specific tissue types and persist in non-dividing cells for long periods of time.

Improving gene therapy manufacturing processes and production

With the first gene therapies on the market and dozens more in trials, the race is on to improve the production and manufacturing processes to deploy gene therapies at scale. Technological solutions are sought to effectively control challenges in the production of viral vectors at the large scales needed to reach the clinical trial step. Speed is critical, yet excessive speed can put product quality, safety, and efficiency at risk.

Specific conditions for this challenge

In order to apply, your proposal must focus on emerging technologies or breakthrough new concept-based technological solution that go far beyond the current state-of-the-art, aimed to overcome cell and gene therapy challenges and obstacles companies are currently being faced with, at the preclinical or clinical level or bio-manufacturing level. Proposers can submit disease or non-disease specific proposals.

Your project must aim to deliver, by its end, at least one of the specific outcomes defined for this challenge. The gender dimension in research content should be taken into account, where relevant.

For more details, see the EIC Work Programme 2021 and the relevant Challenge Guide.

How to apply

In order to apply, your proposal must meet the general as well as possible specific eligibility requirements for a specific Challenge. Please check for particular elements (e.g. specific application focus or technology) in the respective challenge chapter below. 

The Pathfinder Challenges support collaborative research and innovation from consortia or applications from single legal entities (unless stated otherwise in the specific challenge chapter). In case of a consortium your proposal must be submitted by the coordinator on behalf of the consortium that includes at least two independent legal entities. The legal entities may for example be universities, research organisations, SMEs, start-ups, natural persons. In the case of mono-beneficiary projects, mid-caps and larger companies will not be permitted. 

The call deadline for submitting your proposal is 27 October 2021 at 17h00 Brussels local time. You must submit your proposal via the European Funding & Tender Opportunities Portal.

Sections 1 to 3 of the part B of your proposal, corresponding respectively to the evaluation criteria Excellence, Impact, and Quality and Efficiency of the Implementation, must consist of a maximum of 25 A4 pages. 

You will be informed about the outcome of the evaluation 5 months after call deadline (indicative), and your grant agreement will be signed by 8 months after call deadline (indicative).

Documents

Challenge Guide Emerging Technologies in Cell and Gene therapy - Part I
English
(578.58 KB - PDF)
Download
Challenge Guide - Part II
English
(1.15 MB - PDF)
Download