Enhancing cell therapies with genomic techniques

The grant opportunity titled "Enhancing cell therapies with genomic techniques" (HORIZON-HLTH-2025-01-TOOL-01) is part of Horizon Europe’s Health Cluster, focusing on advancing therapeutic innovations in cell therapies through genomic engineering. This call allows for applications from various entities, including research institutions, universities, small and medium-sized enterprises (SMEs), large enterprises, and public-private partnerships involved in biomedical research. Eligible applicants need to collaborate in consortia, with at least three independent entities from different EU member states or associated countries.

The funding type is a grant, specifically categorized under Research and Innovation Actions (RIA), with a total budget of €50 million, enabling each project to secure funding between €8 million and €10 million. The projects are required to have a consortium approach, and they must be focused on development and validation stages, targeted at enhancing cell therapies for various health conditions. Applications are expected to address technical challenges related to genomic techniques like gene editing, improving delivery efficiency, patient safety, and therapeutic effectiveness.

The call emphasizes that eligible proposals must engage with regulatory authorities, include clinical studies, and aim to translate research outcomes into clinically viable solutions. The funding is available to entities from EU member states and associated countries, with no specific countries mentioned beyond this broad scope.

Submissions are part of a single-stage open call, with applications due by September 16, 2025. The success rates for Horizon Europe grants typically range from 10% to 39%. Co-funding is not required for this grant. The call seeks to fund several projects that fulfill its objectives, enhancing the efficacy and scalability of cell-based therapies through innovative genomic techniques, aiming for significant contributions to treatment for conditions such as cancer and genetic disorders.

Overall, this opportunity aims to improve cell therapies through genetic manipulation, facilitating the development of personalized medicine solutions and addressing current limitations faced in the healthcare sector regarding cell therapies.

This is a Horizon Europe call, specifically HORIZON-HLTH-2025-01-TOOL-01, titled "Enhancing cell therapies with genomic techniques." It falls under Cluster 1 - Health and is a HORIZON Research and Innovation Action (HORIZON-RIA). The call adopts a single-stage submission process. The opening date for submissions is May 22, 2025, and the deadline is September 16, 2025, at 17:00:00 Brussels time. The funding is budget-based.

The primary aim is to support activities that contribute to the destination of "Developing and using new tools, technologies, and digital solutions for a healthy society." The expected outcomes include:

Biomedical scientists having access to tools for engineering cells with specific therapeutic features.
Improved methods and assays for biopharmaceutical developers.
Clinicians gaining access to innovative therapeutic approaches for conditions lacking sufficient treatments.
Enrichment of cell engineering, paving the way for personalized therapy options.

The scope of the call focuses on addressing bottlenecks in cell therapies, which have shown effectiveness in treating various health conditions but face challenges in large-scale application. Genome and epigenome editing are highlighted as tools to overcome these limitations and advance cell-based therapies. The call encourages projects that design engineered cells to improve delivery efficiency, patient safety, in vivo persistence, therapeutic effect, immune tolerance, and manufacturing workflows.

Key aspects of the call's scope include:

Emphasis on genetic engineering, particularly gene editing tools, in the design of engineered cells.
Therapeutic action should rely on the cells' endogenous capabilities, excluding the use of cells as drug carriers.
Engineered cells must be derived from human cells, using either stem cells or somatic cells of allogeneic origin for "off-the-shelf" cell therapeutics.
Applicants must specify the targeted therapeutic area from a list that includes: Cancer and oncology, Nervous and sensory system, Cardiovascular and circulatory system, Endocrinology and metabolic system, Musculoskeletal system, Digestive system, Infectious diseases, Respiratory system, Dermatology, Immune system and auto-immune diseases, and Other.

The activities within the projects should include:

Engineering synthetic genetic circuits as switches to modulate cell function, integrated using new genomic techniques.
Utilizing synthetic biology to introduce transgenes or artificial genes for improved therapeutic properties.
Employing gene control systems, including transcriptional, translational, and/or post-translational control, or "sense-and-respond" mechanisms.
Using state-of-the-art tools, including digital tools like Computer-Aided Design (CAD), for efficient design-build-test cycles.
Testing and demonstrating function and performance in suitable in-vitro and ex-vivo systems.
Ensuring added value, safety, and efficacy in appropriate pre-clinical models for a specific therapeutic area.
Demonstrating the safety and therapeutic effect of engineered cells in-vivo.
Engaging with regulatory authorities for qualification of the cell-based therapy and clinical study planning.
Demonstrating feasibility in first-in-human studies is considered an asset.
Addressing sex differences in parent cells and therapeutic applications.
Collaboration with European research infrastructures and EU-supported projects.
Encouraging participation of small and medium-sized enterprises (SMEs).
Considering involvement of the European Commission's Joint Research Centre (JRC) for regulatory science interface and validation of test methods.
Providing details of clinical studies in a dedicated annex.

Admissibility conditions are detailed in Annex A and Annex E of the Horizon Europe Work Programme General Annexes. Proposal page limits and layout are described in Part B of the Application Form. Eligible countries are listed in Annex B of the Work Programme General Annexes. Legal entities from the United States of America are eligible for Union funding. The Joint Research Centre (JRC) can participate as part of the consortium. Projects using satellite data must utilize Copernicus and/or Galileo/EGNOS. Financial and operational capacity and exclusion criteria are in Annex C of the Work Programme General Annexes.

To ensure a balanced project portfolio, grants will be awarded based on ranking and the highest-ranked proposals in different therapeutic areas. Evaluation and award criteria, scoring, and thresholds are in Annex D of the Work Programme General Annexes. The thresholds for Excellence, Impact, and Implementation are each 4, with a cumulative threshold of 12. Submission and evaluation processes are in Annex F, and the indicative timeline for evaluation and grant agreement is also in Annex F of the Work Programme General Annexes. Legal and financial setups are in Annex G.

Application forms, evaluation templates, guidance documents, and model grant agreements are available in the Submission System and through provided links.

The total budget for the HORIZON-HLTH-2025-01 call is allocated across multiple topics, with specific contributions and indicative numbers of grants for each. The HORIZON-HLTH-2025-01-TOOL-01 topic has a budget of 50,000,000 EUR, with individual contributions ranging from 8,000,000 to 10,000,000 EUR, and an indicative number of 5 grants.

There are partner search announcements available, and the Funding & Tenders Portal provides various support resources, including an Online Manual, FAQ, Research Enquiry Service, National Contact Points, Enterprise Europe Network, IT Helpdesk, European IPR Helpdesk, CEN-CENELEC Research Helpdesk, ETSI Research Helpdesk, and information on the European Charter for Researchers.

In summary, this Horizon Europe call aims to advance cell-based therapies by leveraging genomic techniques to engineer cells with improved therapeutic properties. It encourages projects that address current limitations in cell therapies, utilize genetic engineering tools, and demonstrate the safety and efficacy of engineered cells in pre-clinical and clinical models. The call is open to a wide range of therapeutic areas and encourages collaboration with research infrastructures, SMEs, and regulatory authorities. The total budget for the HORIZON-HLTH-2025-01 call is significant, and the call provides extensive support resources for applicants.

The HORIZON-HLTH-2025-01-TOOL-01 call is a funding opportunity within the Horizon Europe program, focusing on enhancing cell therapies through the application of genomic techniques. It's like a contest where researchers and companies can propose innovative ways to engineer cells to make them better at treating diseases. The goal is to overcome current limitations in cell therapies, such as making them safer, more effective, and easier to manufacture.

Imagine you have tiny programmable robots (cells) that can be sent into the body to fight diseases like cancer or diabetes. However, these robots aren't perfect yet. They might not reach the right location, they might not last long enough, or they might trigger unwanted side effects. This call encourages scientists to use tools like gene editing to reprogram these cells, giving them new instructions to perform their tasks more efficiently and safely.

To participate, applicants need to form a team, propose a project that uses genetic engineering to improve cell therapies, and specify which disease they are targeting. The project should include experiments to test the engineered cells in the lab and in animal models, and it should also consider how to manufacture these cells on a large scale.

The European Commission is providing a significant amount of funding for these projects, and they are particularly interested in proposals that involve small businesses, collaborate with existing research facilities, and consider the involvement of regulatory agencies to ensure the safety and effectiveness of the new cell therapies.

Ultimately, this call aims to accelerate the development of next-generation cell therapies that can provide new and improved treatment options for a wide range of diseases.

Eligible Applicant Types: The eligible applicant types include any legal entity established in the United States of America, small and medium-sized enterprises (SMEs), and the Joint Research Centre (JRC) which may participate as a member of the consortium. Other eligible countries are described in Annex B of the Work Programme General Annexes and a number of non-EU/non-Associated Countries that are not automatically eligible for funding have made specific provisions for making funding available for their participants in Horizon Europe projects.

Funding Type: The funding type is a grant, specifically a HORIZON Research and Innovation Action (HORIZON-RIA) and HORIZON Innovation Actions (HORIZON-IA) and HORIZON Coordination and Support Actions (HORIZON-CSA) under the Horizon Europe Programme. The type of Model Grant Agreement is HORIZON Action Grant Budget-Based [HORIZON-AG].

Consortium Requirement: The opportunity appears to require a consortium, as the Joint Research Centre (JRC) may participate as a member of the consortium selected for funding and partner searches are encouraged.

Beneficiary Scope (Geographic Eligibility): Eligible countries are described in Annex B of the Work Programme General Annexes. A number of non-EU/non-Associated Countries that are not automatically eligible for funding have made specific provisions for making funding available for their participants in Horizon Europe projects. Any legal entity established in the United States of America is eligible to receive Union funding.

Target Sector: The target sector is health, specifically focusing on cell and gene therapies, genome editing, synthetic biology, cancer, nervous system disorders, cardiovascular diseases, endocrinology, metabolic disorders, musculoskeletal system, digestive system disorders, infectious diseases, respiratory system disorders, dermatology, and immune system and auto-immune diseases.

Mentioned Countries: United States of America.

Project Stage: The project stage appears to range from research and development to clinical studies, with an emphasis on translation of research into clinically viable solutions and demonstration of feasibility in first in-human studies.

Funding Amount: The funding amounts vary depending on the specific topic within the call, ranging from €2,000,000 to €80,000,000 in total budget, with individual contributions ranging from around €2,000,000 to between €15,000,000 and €20,000,000.

Application Type: The application type is an open call with a single-stage submission process.

Nature of Support: Beneficiaries will receive money in the form of a grant.

Application Stages: The application process is a single-stage process.

Success Rates: The success rates are not explicitly stated, but the indicative number of grants for each topic is provided, allowing for an estimation of the potential success rate based on the number of anticipated applications.

Co-funding Requirement: The information provided does not explicitly state whether co-funding is required.

Summary: This Horizon Europe call, Cluster 1 Health, focuses on "Enhancing cell therapies with genomic techniques" and aims to improve cell-based therapies through genetic and synthetic biology approaches. The goal is to engineer cells with improved therapeutic features, addressing limitations such as delivery efficiency, patient safety, and immune tolerance. The call encourages collaboration with European research infrastructures and SMEs, and it is open to a wide range of applicants, including those from the United States. Projects should include clinical studies and engage with regulatory authorities. The call encompasses various therapeutic areas, including cancer, neurological disorders, cardiovascular diseases, and infectious diseases. Funding is provided through HORIZON-RIA, HORIZON-IA and HORIZON-CSA grants, with a single-stage application process and varying budget allocations for different topics within the call. The call aims to translate research into clinically viable solutions, ultimately providing clinicians with innovative therapeutic approaches and paving the way for personalized therapy options. The call is structured around several topics, each with its own budget, scope, and expected outcomes. The topics range from research and innovation actions (RIA) to coordination and support actions (CSA), reflecting a comprehensive approach to advancing cell and gene therapies.