Objective Cystic fibrosis (CF) is a progressive life-shortening disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene leading to a dysfunctional CFTR protein. The disease affects over 70,000 patients worldwide and while many mutations are known, the F508del mutation affects 90% of all patients. The absence of CFTR in the plasma membrane leads to a dramatic decrease in chloride efflux, resulting in viscous mucus that causes severe symptoms in vital organs like the lungs and intestines. For CF patients that suffer from the life threatening F508del mutation only palliative treatment exist.PRO-CF-MED addresses the specific challenge of this call by introducing the first disease modifying medication for the treatment of the CF patients with F508del mutation. The PRO-CF-MED project has been designed to assess the potential clinical efficacy of QR-010, an innovative disease modifying oligonucleotide-based treatment for F508del patients. Partners within PRO-CF-MED have generated very promising preclinical evidence for QR-010 which allows for further clinical assessment of QR-010 in clinical trials. PRO-CF-MED will enable the fast translation of QR-010 towards clinical practice and market authorisation. PRO-CF-MED has the potential to transform this life-threatening condition into a manageable one. Fields of science medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsnatural scienceschemical sciencesinorganic chemistryalkali metalsnatural sciencesbiological sciencesgeneticsmutationmedical and health sciencesbasic medicinepharmacology and pharmacypharmacokineticsnatural sciencesbiological sciencesgeneticsRNA Keywords Cystic fibrosis oligonucleotide disease- modifying medication First in Humans clinical trial F508del mutation 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-13-2014 - New therapies for chronic non-communicable diseases Call for proposal H2020-PHC-2014-2015 See other projects for this call Sub call H2020-PHC-2014-two-stage Funding Scheme RIA - Research and Innovation action Coordinator PROQR THERAPEUTICS NV Net EU contribution € 452 907,00 Address Zernikedreef 9 2333 CK Leiden Netherlands See on map Region West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,50 Participants (6) Sort alphabetically Sort by Net EU contribution Expand all Collapse all Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. PROQR THERAPEUTICS III BV Netherlands Net EU contribution € 4 000 637,00 Address Zernikedreef 9 2333 CK Leiden 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 West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 15 240 038,50 EUROPEAN CYSTIC FIBROSIS SOCIETY Denmark Net EU contribution € 230 720,00 Address Kastanieparken 7 7470 Karup See on map Region Danmark Midtjylland Vestjylland Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITE CATHOLIQUE DE LOUVAIN Belgium Net EU contribution € 394 612,50 Address Place de l universite 1 1348 Louvain la neuve See on map Region Région wallonne Prov. Brabant Wallon Arr. Nivelles 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 Other funding € 0,00 INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE France Net EU contribution € 359 927,50 Address Rue de tolbiac 101 75654 Paris See on map Region Ile-de-France Ile-de-France Paris 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 Other funding € 0,00 UNIVERSITAIR MEDISCH CENTRUM UTRECHT Netherlands Net EU contribution € 228 250,00 Address Heidelberglaan 100 3584 CX Utrecht See on map Region West-Nederland Utrecht Utrecht 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 Other funding € 0,00 PATERGRUS BVBA Belgium Net EU contribution € 330 085,00 Address Sparhoekdreef 57 9880 Aalter 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 Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent 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 Other funding € 1,50
