Objective
This project will develop an innovative therapeutic approach for the treatment of Cystic Fibrosis (CF). This condition originates from the defective function of the CFTR protein, a chloride and bicarbonate permeable transmembrane channel. This project will evaluate small molecules capable of facilitating the transmembrane transport of anions such as chloride and bicarbonate and will thus enable CF treatment by replacing the missing CFTR anion permeation activity. This represents an unexplored path in the treatment of CF and a paradigm shift with respect to current strategies searching for a cure for CF. Instead of focusing on the development of mutation-specific treatments, we plan to develop a therapy applicable to CF patients, regardless of the type of mutation they harbor. Thus, this therapeutic approach overcomes the limitation of current mutation-specific treatments and is applicable to CF patients in general.
To achieve this goal we have set up a comprehensive program to validate a research concept and complete the preclinical development of a new lead compound, making it ready for early clinical development. A rmultidisciplinary team of qualified researchers have been assembled to bring to conclusion a truly translational project from the synthesis of new compounds to validation on animal models.
Cystic Fibrosis affects more people than any other rare disease. Therefore, it could be said, at least in quantitative terms, that CF qualifies as the main target of the topic. This project aims to complete the preclinical development of novel, innovative drugs based on a radically new concept in Cystic Fibrosis therapies. This result fully addresses the expected impact set out in the work programme of advancing the development of new therapeutic options for patients living with rare diseases as well as contributing to reach the IRDiRC objective to deliver 200 new therapies for rare diseases by 2020.
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: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
- medical and health sciences basic medicine medicinal chemistry
- medical and health sciences basic medicine physiology pathophysiology
- natural sciences biological sciences genetics mutation
- engineering and technology electrical engineering, electronic engineering, information engineering electronic engineering sensors
- medical and health sciences basic medicine toxicology
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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.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being
MAIN PROGRAMME
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H2020-EU.3.1.3. - Treating and managing disease
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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.
RIA - Research and Innovation action
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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-PHC-2014-2015
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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.
09001 Burgos
Spain
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.