Project description
Innovative cure for thymus deficiency
The thymus plays a critical role in immune system development and its absence at birth leads to severe immunodeficiency, with affected infants typically having a short life expectancy. Thymus tissue transplantation is the only current cure, but it carries a 30 % mortality rate and high costs. The EIC-funded i-THYMUS project proposes a revolutionary solution by using gene therapy to create thymus organoids from the patient’s own cells, which can be implanted through minimally invasive procedures. The approach has yielded promising preclinical data and the aim is to obtain clinical validation through trials by 2028 with a pharmaceutical partner. The project will initially target DiGeorge syndrome but the technology aims to treat other patients including those with thymus damage after chemotherapy.
Objective
Each year, >250 babies are born without a thymus. These babies have a life expectancy of 2-3 years. The only cure is transplantation of thymus tissue, but this has a 30% mortality rate and costs almost >€2.5M. We developed i-Thymus, a revolutionary cure based on the patient’s own cells leveraging innovative gene therapy to generate thymus organoids that can be implanted minimally invasively in the patient (Fig. 1). We have already shown this works in functional proof of concept in vivo. With this EIC Accelerator, we will finalise development to enter the clinic for a Phase I/II trial, to enter the EU market with a pharma partner by 2028. After rapid market entry with ODD for complete DiGeorge syndrome, we will leverage this clinical success and our first revenues to develop i-Thymus for unmet needs of hundreds of thousands of other patients, starting with AML patients who have lost thymus function after high-dose chemotherapy for stem cell transplantation.
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.
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.
- social sciences sociology demography mortality
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences clinical medicine transplantation
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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)
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Topic(s)
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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-ACC-BF - HORIZON EIC Accelerator Blended Finance
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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-2023-ACCELERATOR-01
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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.
2342 DH Leiden
Netherlands
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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.