European Commission supports further development of cystic fibrosis drug

The European Commission supports further development of a cystic fibrosis (CF) drug targeting rare variants. This milestone comes from the HIT-CF project, coordinated by UMC Utrecht, where researchers from 16 European countries — including and — worked closely together to find a solution for people with rare variants of cystic fibrosis (CF).

What makes this research unique?

  • More than 500 patients from across Europe participated in the FIS test, an innovative biomarker that predicts ex vivo who will benefit from treatment.
  • The new combination CFTR-modulatorcombination of Dirocaftor–Posenacaftor–Nesolicaftor shows promising clinical effects, even in mutations that have never been studied in trials before.
  • The European Commission recognises the value of biomarker-guided therapy and supports further development.


Prof. Dr. Kors van der Ent: ‘Despite all kinds of obstacles, the entire European CF community has shown perseverance and dedication to realising treatments for all patients with CF. We are enthusiastic about the results.’This project demonstrates how international collaboration, patient involvement and innovative technology can lead to personalised care for people with rare diseases.
 

About Regenerative Medicine Utrecht

Regenerative Medicine Utrecht (RMU) is a world-renowned collaboration of excellent knowledge institutes and start-ups, located at the Utrecht Science Park. We bring together researchers from UMC Utrecht, Utrecht ľ¹Ï¸£ÀûÓ°ÊÓ (UU) and the Hubrecht Institute. Our focus is on restoring health and vitality instead of symptom control. By repairing, replacing, or regenerating cells, tissues, and organs, we aim to cure patients. 

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