Project description
New drug for beta-thalassemia major
Patients with beta-thalassemia major require regular transfusions of red blood cells. However, constant transfusions are linked to adverse effects due to the resultant iron overload, including severe impairment of internal organs’ function and heart disease, with cardiac complications causing over two thirds of deaths in patients. The EU-funded Chelator project will address this with an efficient metal chelator shown to decrease iron levels in vitro and in vivo. Specifically, it demonstrates a substantial advantage of safety already confirmed in clinical trials. The drug has already been recognised by the EU and United States’ authorities and has received Orphan Drug Designation for mercury toxicity treatment.
Objective
β-thalassemia major (βTM) is a lethal orphan disease with genetic background, diagnosed in infancy and requiring recurrent blood transfusions to prevent malformations, assure acceptable quality of life and prolong patient’s lifetime. Constant transfusions are related to severe adverse effects due to the resultant iron overload, including severe impairment of internal organs’ function and heart disease, with cardiac complications causing over two thirds of deaths in βTM patients. Life-long, effective iron chelation therapy is the only measure to reduce these risks, but available chelators have low efficacy and tolerability and/or complicated administration route. Together with high costs it hampers compliance, reducing the efficacy of available chelators even more and leaving unmet the need for safe, effective and easy to use iron chelator.
Emeramide is an efficient metal chelator shown to decrease iron levels in vitro and in vivo, with a huge advantage of safety already confirmed in clinical trials up to Phase 2a. The drug has been recognized by the US and the EU authorities and received Orphan Drug Designation for mercury toxicity treatment. Thanks to its great tolerability Emeramide promotes high compliance with chelation therapy, making patients’ lives not only longer, but also as close to normal as possible.
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.
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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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H2020-EU.2.3. - INDUSTRIAL LEADERSHIP - Innovation In SMEs
MAIN PROGRAMME
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H2020-EU.3. - PRIORITY 'Societal challenges
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H2020-EU.2.1. - INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies
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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.
SME-1 - SME instrument phase 1
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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) H2020-EIC-SMEInst-2018-2020
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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.
D06 C8X4 DUBLIN
Ireland
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
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.