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 medical and health sciencesbasic medicinemedicinal chemistrymedical and health sciencesbasic medicinephysiologypathophysiologynatural sciencesbiological sciencesgeneticsmutationengineering and technologyelectrical engineering, electronic engineering, information engineeringelectronic engineeringsensorsmedical and health sciencesbasic medicinetoxicology Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) PHC-14-2015 - New therapies for rare diseases Call for proposal H2020-PHC-2014-2015 See other projects for this call Sub call H2020-PHC-2015-two-stage Funding Scheme RIA - Research and Innovation action Coordinator UNIVERSIDAD DE BURGOS Net EU contribution € 665 563,75 Address HOSPITAL DEL REY 09001 Burgos Spain See on map Region Centro (ES) Castilla y León Burgos Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 665 563,75 Participants (7) Sort alphabetically Sort by Net EU contribution Expand all Collapse all STEINBEIS INNOVATION GGMBH Germany Net EU contribution € 523 953,75 Address ADORNOSTRASSE 8 70599 Stuttgart See on map Region Baden-Württemberg Stuttgart Stuttgart, Stadtkreis Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 523 953,75 ISTITUTO GIANNINA GASLINI Italy Net EU contribution € 390 002,50 Address VIA GEROLAMO GASLINI 5 16147 Genova See on map Region Nord-Ovest Liguria Genova Activity type Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 390 002,50 CONSIGLIO NAZIONALE DELLE RICERCHE Italy Net EU contribution € 610 273,75 Address PIAZZALE ALDO MORO 7 00185 Roma See on map Region Centro (IT) Lazio Roma Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 610 273,75 BIONEER A/S Denmark Net EU contribution € 557 375,00 Address KOGLE ALLE 2 2970 Hoersholm See on map Region Danmark Hovedstaden Nordsjælland Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 557 375,00 BIOKERALTY RESEARCH INSTITUTE AIE Spain Net EU contribution € 591 875,00 Address ENTIDAD ARCAUTE 005 ARCAUTE 01192 ARCAUTE ALAVA See on map Region Noreste País Vasco Araba/Álava Activity type Other Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 591 875,00 AVIDIN KUTATO, FEJLESZTO ES KERESKEDELMI KFT Hungary Net EU contribution € 732 875,00 Address ALSO KIKOTO SOR 11 6726 SZEGED See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Alföld és Észak Dél-Alföld Csongrád Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 732 875,00 AGENCIA ESTATAL CONSEJO SUPERIOR DE INVESTIGACIONES CIENTIFICAS Spain Net EU contribution € 519 368,75 Address CALLE SERRANO 117 28006 Madrid See on map Region Comunidad de Madrid Comunidad de Madrid Madrid Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 519 368,75