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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.

Call for proposal

H2020-PHC-2014-2015

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Sub call

H2020-PHC-2014-two-stage

Coordinator

PROQR THERAPEUTICS NV
Net EU contribution
€ 452 907,00
Address
Zernikedreef 9
2333 CK Leiden
Netherlands

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Region
West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,50

Participants (6)

Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

PROQR THERAPEUTICS III BV
Netherlands
Net EU contribution
€ 4 000 637,00
Address
Zernikedreef 9
2333 CK Leiden

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 15 240 038,50
EUROPEAN CYSTIC FIBROSIS SOCIETY
Denmark
Net EU contribution
€ 230 720,00
Address
Kastanieparken 7
7470 Karup

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Region
Danmark Midtjylland Vestjylland
Activity type
Other
Non-EU contribution
€ 0,00
UNIVERSITE CATHOLIQUE DE LOUVAIN
Belgium
Net EU contribution
€ 394 612,50
Address
Place De L Universite 1
1348 Louvain La Neuve

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Region
Région wallonne Prov. Brabant Wallon Arr. Nivelles
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
Net EU contribution
€ 359 927,50
Address
Rue De Tolbiac 101
75654 Paris

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Non-EU contribution
€ 0,00
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Netherlands
Net EU contribution
€ 228 250,00
Address
Heidelberglaan 100
3584 CX Utrecht

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Region
West-Nederland Utrecht Utrecht
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
PATERGRUS BVBA
Belgium
Net EU contribution
€ 330 085,00
Address
Sparhoekdreef 57
9880 Aalter

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 1,50