CORDIS
EU research results

CORDIS

English EN

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.
Leaflet | Map data © OpenStreetMap contributors, Credit: EC-GISCO, © EuroGeographics for the administrative boundaries

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)

Sort alphabetically

Sort by EU Contribution

Expand all

STEINBEIS INNOVATION GGMBH

Germany

EU Contribution

€ 523 953,75

ISTITUTO GIANNINA GASLINI

Italy

EU Contribution

€ 390 002,50

CONSIGLIO NAZIONALE DELLE RICERCHE

Italy

EU Contribution

€ 610 273,75

BIONEER A/S

Denmark

EU Contribution

€ 557 375

BIOKERALTY RESEARCH INSTITUTE AIE

Spain

EU Contribution

€ 591 875

AVIDIN KUTATO, FEJLESZTO ES KERESKEDELMI KFT

Hungary

EU Contribution

€ 732 875

AGENCIA ESTATAL CONSEJO SUPERIOR DEINVESTIGACIONES CIENTIFICAS

Spain

EU Contribution

€ 519 368,75

Project information

Grant agreement ID: 667079

Status

Closed project

  • Start date

    1 January 2016

  • End date

    31 December 2018

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 4 591 287,50

  • EU contribution

    € 4 591 287,50

Coordinated by:

UNIVERSIDAD DE BURGOS

Spain

This project is featured in...