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Novel therapeutic approaches for the treatment of cystic fibrosis based on small...

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

31/12/2018

UBU

5M€

Project Budget: 5M€

Project leader

UNIVERSIDAD DE BURGOS No se ha especificado una descripción o un objeto social para esta compañía.

total researchers 371

PARTICIPATION DEPralty Sin fecha límite de participación.

Ver 9 Participantes

Financing granted El organismo H2020 notifico la concesión del proyecto The day 2018-12-31

PHC-14-2015: New therapies for rare diseases Scope:Specific challenge: A considerable amount of knowledge has been generated by biomedical resear...

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characteristics of the participant

Este proyecto no cuenta con búsquedas de partenariado abiertas en este momento.

Private Information

No hay información privada compartida para este proyecto. Habla con el coordinador.

9 Participantes

UBU

665.56K€ | Leader

NEOFLEX

282.98K€ | participant

AVIDIN KTF

732.88K€ | participant

ISTITUTO GIANNINA GASLINI

390.00K€ | participant

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Project duration: 36 months Date Start: 2015-12-01
End date: 2018-12-31

Project leader

UNIVERSIDAD DE BURGOS No se ha especificado una descripción o un objeto social para esta compañía.

total researchers 371

Project Budget 5M€

participation deadline Sin fecha límite de participación.

Project description 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.

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