The challenge

Cystic Fibrosis (CF) is a rare, progressive genetic disorder that affects around 160,000 people worldwide. The disease causes severe damage to the lungs, digestive system and other vital organs.

CF is caused by mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene. These mutations impair the function of the CFTR protein throughout the body. More than 2,000 different mutations of the CFTR gene have been identified to date.

Since 2012 so-called modulator therapies have been developed: medicines that (at least partially) restore the function of the CFTR protein, instead of just combating the symptoms. However, a significant proportion of people with CF are still unable to use these therapies.

Our support

Immunis Foundation, together with the TAAI Foundation, has supplied funding for a phase 2-B study of Fair Therapeutics into an alternative, and cheaper, medicine for patients with CF.