Objectif
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.
Champ scientifique
- medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugs
- natural scienceschemical sciencesinorganic chemistryalkali metals
- natural sciencesbiological sciencesgeneticsmutation
- medical and health sciencesbasic medicinepharmacology and pharmacypharmacokinetics
- natural sciencesbiological sciencesgeneticsRNA
Mots‑clés
Programme(s)
Régime de financement
RIA - Research and Innovation actionCoordinateur
2333 CK Leiden
Pays-Bas
L’entreprise s’est définie comme une PME (petite et moyenne entreprise) au moment de la signature de la convention de subvention.