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Clinical Proof of concept for a RNA-targeting Oligonucleotide for a Cystic fibrosis-F508del MEDication

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.

Field of science

  • /social sciences/economics and business/business and management/commerce
  • /medical and health sciences/basic medicine/pharmacology and pharmacy/pharmaceutical drug
  • /natural sciences/biological sciences/genetics and heredity/mutation

Call for proposal

H2020-PHC-2014-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

PROQR THERAPEUTICS NV
Address
Zernikedreef 9
2333 CK Leiden
Netherlands
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 4 453 544

Participants (5)

EUROPEAN CYSTIC FIBROSIS SOCIETY
Denmark
EU contribution
€ 230 720
Address
Kastanieparken 7
7470 Karup
Activity type
Other
UNIVERSITE CATHOLIQUE DE LOUVAIN
Belgium
EU contribution
€ 394 612,50
Address
Place De L Universite 1
1348 Louvain La Neuve
Activity type
Higher or Secondary Education Establishments
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 359 927,50
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Netherlands
EU contribution
€ 228 250
Address
Heidelberglaan 100
3584 CX Utrecht
Activity type
Higher or Secondary Education Establishments
PATERGRUS BVBA
Belgium
EU contribution
€ 330 085
Address
Sparhoekdreef 57
9880 Aalter
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)