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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.

Field of science

  • /natural sciences/biological sciences/genetics and heredity/mutation
  • /social sciences/social and economic geography/transport

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

UNIVERSIDAD DE BURGOS
Address
Hospital Del Rey
09001 Burgos
Spain
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 665 563,75

Participants (7)

STEINBEIS INNOVATION GGMBH
Germany
EU contribution
€ 523 953,75
Address
Willi Bleicher Strasse 19
70174 Stuttgart
Activity type
Research Organisations
ISTITUTO GIANNINA GASLINI
Italy
EU contribution
€ 390 002,50
Address
Via Gerolamo Gaslini 5
16147 Genova
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
CONSIGLIO NAZIONALE DELLE RICERCHE
Italy
EU contribution
€ 610 273,75
Address
Piazzale Aldo Moro 7
00185 Roma
Activity type
Research Organisations
BIONEER A/S
Denmark
EU contribution
€ 557 375
Address
Kogle Alle 2
2970 Hoersholm
Activity type
Research Organisations
BIOKERALTY RESEARCH INSTITUTE AIE
Spain
EU contribution
€ 591 875
Address
Entidad Arcaute, 005, Arcaute
01192 Arcaute Alava
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
AVIDIN KUTATO, FEJLESZTO ES KERESKEDELMI KFT
Hungary
EU contribution
€ 732 875
Address
Also Kikoto Sor 11
6726 Szeged
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
AGENCIA ESTATAL CONSEJO SUPERIOR DEINVESTIGACIONES CIENTIFICAS
Spain
EU contribution
€ 519 368,75
Address
Calle Serrano 117
28006 Madrid
Activity type
Research Organisations