Objective
Gene therapies (GTs) based on hematopoietic stem and progenitor cells (HSPCs) hold great promise, but their tumorigenicity risk, already observed in patients, is a significant concern. Current safety tests rely on oversimplified in vitro systems, often based on murine cells, and in vivo mouse models, which poorly reflect human biology and therefore lack sufficient predictivity. Starting from the output of the CRACK IT CleanCut Challenge of the NC3Rs, sponsored by leading industries that contributed to shaping the NAM development, STEM-SAFE aims to validate our disruptive MOAB-BM-LN, a dual-organ in vitro assay that recreates essential features of human bone marrow and lymph nodes. By detecting preleukemic events, our NAM enables evaluation of the actual GT products under physiologically relevant conditions. Uniting a world-class multidisciplinary team with access to unique real-world GT facilities, Fondazione Telethon (FTELE) within STEM-SAFE brings a bold vision to transform the preclinical testing service market of advanced therapy medicinal products. Our primary goal is to set a new analytical gold standard that addresses tumorigenic risks by reducing the need for animal testing with a more predictive and human-relevant in vitro assay. This assay has the potential to reduce development costs and accelerate the delivery of safer, life-saving therapies to patients while enhancing accessibility and setting an entirely new benchmark for success in the European GT industry. During phase 1, leveraging the collaboration with leading end users from the FTELE ecosystem and GT industry players committed to integrating the NAM into their GT development pipelines, we will demonstrate the viability of the NAM, advancing from TRL4 to TRL5 while elaborating the roadmap for adoption. In phase 2, following the regulatory plan and roadmap set in phase 1, we will transition the MOAB-BM-LN to a regulatory-qualified prototype suitable for broader adoption (TRL 6) and scale-up.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
This project's classification has been human-validated.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
This project's classification has been human-validated.
- medical and health sciences basic medicine pharmacology and pharmacy drug safety
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences basic medicine pharmacology and pharmacy pharmaceutical drugs
- engineering and technology other engineering and technologies microtechnology lab on a chip
Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.3.1 - The European Innovation Council (EIC)
MAIN PROGRAMME
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HORIZON.3.1.3 - Additional EIC activities
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-EIC - HORIZON EIC Grants
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) HORIZON-EIC-2026-AIC
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
00185 ROMA
Italy
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.