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PRO-CF-MED

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

Total Cost €

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EC-Contrib. €

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Partnership

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Project "PRO-CF-MED" data sheet

The following table provides information about the project.

Coordinator
PROQR THERAPEUTICS NV 

Organization address
address: ZERNIKEDREEF 9
city: LEIDEN
postcode: 2333 CK
website: n.a.

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Netherlands [NL]
 Project website http://pro-cf-med.eu/
 Total cost 21˙237˙179 €
 EC max contribution 5˙997˙139 € (28%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-PHC-2014-two-stage
 Funding Scheme RIA
 Starting year 2015
 Duration (year-month-day) from 2015-05-01   to  2017-12-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    PROQR THERAPEUTICS NV NL (LEIDEN) coordinator 4˙453˙544.00
2    UNIVERSITE CATHOLIQUE DE LOUVAIN BE (LOUVAIN LA NEUVE) participant 394˙612.00
3    INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE FR (PARIS) participant 359˙927.00
4    PATERGRUS BVBA BE (AALTER) participant 330˙085.00
5    EUROPEAN CYSTIC FIBROSIS SOCIETY DK (KARUP) participant 230˙720.00
6    UNIVERSITAIR MEDISCH CENTRUM UTRECHT NL (UTRECHT) participant 228˙250.00

Map

 Project 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.

 Deliverables

List of deliverables.
PRO-CF-MED project website developed including project updates and patient ‘friendly’ section Websites, patent fillings, videos etc. 2019-03-22 17:18:45

Take a look to the deliverables list in detail:  detailed list of PRO-CF-MED deliverables.

 Publications

year authors and title journal last update
List of publications.
2016 I Sermet-Gaudelus, D Nichols, JA Nick, C De Boeck, JP Clancy, GM Solomon, SM Rowe, JS Elborn, MA MAll, JA Bolognese, F Bouisset, W den Hollander, N Lamontagne, N Tomkinson, N Henig
QR-010 significantly improves CFTR function in nasal potential difference proof of concept study in subjects with CF homozygous for the F508del-CFTR mutation
published pages: , ISSN: , DOI:
2019-05-24
2016 Sabrina Noel, Bela Z Schmidt, Jeremy Haaf, Teresinha Leal
Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
published pages: 127-140, ISSN: 1179-1438, DOI: 10.2147/CPAA.S100759
Clinical Pharmacology: Advances and Applications Volume 8 2019-05-24

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The information about "PRO-CF-MED" are provided by the European Opendata Portal: CORDIS opendata.

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