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PRO-CF-MED

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

Total Cost €

EC-Contrib. €

Partnership

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Outcomes and
results

PRO-CF-MED project word cloud

Explore the words cloud of the PRO-CF-MED project. It provides you a very rough idea of what is the project "PRO-CF-MED" about.

translation intestines decrease first conductance mucus absence fibrosis f508del qr medication 010 transform mutation palliative shortening pro market treatment life patients leads cf symptoms causes preclinical modifying cftr lungs gene chloride dysfunctional threatening worldwide manageable suffer transmembrane med dramatic disease fast 90 introducing clinical regulator ing exist efflux viscous mutations caused membrane designed efficacy severe vital protein trials organs oligonucleotide innovative cystic progressive plasma authorisation

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 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.	NL (LEIDEN)	coordinator	4˙453˙544.00
2	UNIVERSITE CATHOLIQUE DE LOUVAIN UNIVERSITE CATHOLIQUE DE LOUVAIN Organization address address: PLACE DE L UNIVERSITE 1 city: LOUVAIN LA NEUVE postcode: 1348 website: www.uclouvain.be contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	BE (LOUVAIN LA NEUVE)	participant	394˙612.00
3	INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE Organization address address: RUE DE TOLBIAC 101 city: PARIS postcode: 75654 website: www.inserm.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (PARIS)	participant	359˙927.00
4	PATERGRUS BVBA PATERGRUS BVBA Organization address address: SPARHOEKDREEF 57 city: AALTER postcode: 9880 website: www.biotechsubsidy.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	BE (AALTER)	participant	330˙085.00
5	EUROPEAN CYSTIC FIBROSIS SOCIETY EUROPEAN CYSTIC FIBROSIS SOCIETY Organization address address: KASTANIEPARKEN 7 city: KARUP postcode: 7470 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.	DK (KARUP)	participant	230˙720.00
6	UNIVERSITAIR MEDISCH CENTRUM UTRECHT UNIVERSITAIR MEDISCH CENTRUM UTRECHT Organization address address: HEIDELBERGLAAN 100 city: UTRECHT postcode: 3584 CX website: www.umcutrecht.nl contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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 â€˜friendlyâ€™ 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

List of publications.
year	authors and title	journal	last update
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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