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Novel therapeutic approaches for the treatment of cystic fibrosis based on small molecule transmembrane anion transporters

Objective

This project will develop an innovative therapeutic approach for the treatment of Cystic Fibrosis (CF). This condition originates from the defective function of the CFTR protein, a chloride and bicarbonate permeable transmembrane channel. This project will evaluate small molecules capable of facilitating the transmembrane transport of anions such as chloride and bicarbonate and will thus enable CF treatment by replacing the missing CFTR anion permeation activity. This represents an unexplored path in the treatment of CF and a paradigm shift with respect to current strategies searching for a cure for CF. Instead of focusing on the development of mutation-specific treatments, we plan to develop a therapy applicable to CF patients, regardless of the type of mutation they harbor. Thus, this therapeutic approach overcomes the limitation of current mutation-specific treatments and is applicable to CF patients in general.
To achieve this goal we have set up a comprehensive program to validate a research concept and complete the preclinical development of a new lead compound, making it ready for early clinical development. A rmultidisciplinary team of qualified researchers have been assembled to bring to conclusion a truly translational project from the synthesis of new compounds to validation on animal models.
Cystic Fibrosis affects more people than any other rare disease. Therefore, it could be said, at least in quantitative terms, that CF qualifies as the main target of the topic. This project aims to complete the preclinical development of novel, innovative drugs based on a radically new concept in Cystic Fibrosis therapies. This result fully addresses the expected impact set out in the work programme of advancing the development of new therapeutic options for patients living with rare diseases as well as contributing to reach the IRDiRC objective to deliver 200 new therapies for rare diseases by 2020.

Call for proposal

H2020-PHC-2014-2015

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Sub call

H2020-PHC-2015-two-stage

Coordinator

UNIVERSIDAD DE BURGOS
Net EU contribution
€ 665 563,75
Address
Hospital Del Rey
09001 Burgos
Spain

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Region
Castilla y León Burgos
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00

Participants (7)

STEINBEIS INNOVATION GGMBH
Germany
Net EU contribution
€ 523 953,75
Address
Adornostrasse 8
70599 Stuttgart

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Region
Baden-Württemberg Stuttgart Stuttgart, Stadtkreis
Activity type
Research Organisations
Other funding
€ 0,00
ISTITUTO GIANNINA GASLINI
Italy
Net EU contribution
€ 390 002,50
Address
Via Gerolamo Gaslini 5
16147 Genova

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Region
Nord-Ovest Liguria Genova
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Other funding
€ 0,00
CONSIGLIO NAZIONALE DELLE RICERCHE
Italy
Net EU contribution
€ 610 273,75
Address
Piazzale Aldo Moro 7
00185 Roma

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Region
Centro (IT) Lazio Roma
Activity type
Research Organisations
Other funding
€ 0,00
BIONEER A/S
Denmark
Net EU contribution
€ 557 375,00
Address
Kogle Alle 2
2970 Hoersholm

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Region
Danmark Hovedstaden Nordsjælland
Activity type
Research Organisations
Other funding
€ 0,00
BIOKERALTY RESEARCH INSTITUTE AIE
Spain
Net EU contribution
€ 591 875,00
Address
Entidad Arcaute 005 Arcaute
01192 Arcaute Alava

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Region
Noreste País Vasco Araba/Álava
Activity type
Other
Other funding
€ 0,00
AVIDIN KUTATO, FEJLESZTO ES KERESKEDELMI KFT
Hungary
Net EU contribution
€ 732 875,00
Address
Also Kikoto Sor 11
6726 Szeged

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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
Alföld és Észak Dél-Alföld Csongrád
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
AGENCIA ESTATAL CONSEJO SUPERIOR DE INVESTIGACIONES CIENTIFICAS
Spain
Net EU contribution
€ 519 368,75
Address
Calle Serrano 117
28006 Madrid

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Region
Comunidad de Madrid Comunidad de Madrid Madrid
Activity type
Research Organisations
Other funding
€ 0,00