PRO-CF-MEDProject ID: 633545
Clinical Proof of concept for a RNA-targeting Oligonucleotide for a Cystic fibrosis-F508del MEDication
Total cost:EUR 21 237 179,50
EU contribution:EUR 5 997 139
Call for proposal:H2020-PHC-2014-two-stageSee other projects for this call
Funding scheme:RIA - Research and Innovation action
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.
EU contribution: EUR 4 453 544
2333 CK LEIDEN
EU contribution: EUR 230 720
EU contribution: EUR 394 612,50
PLACE DE L UNIVERSITE 1
1348 LOUVAIN LA NEUVE
EU contribution: EUR 359 927,50
RUE DE TOLBIAC 101
EU contribution: EUR 228 250
3584 CX UTRECHT
EU contribution: EUR 330 085