Cystic fibrosis (CF) is an inherited disorder that causes severe damage to the lungs, digestive system and other organs in the body. People with CF carry a defect in the CFTR gene causing bodily secretions to become sticky and thick, impeding the functionality of the affected organs.
Worldwide, about 70,000 to 100,000 people have CF. A major problem in treating CF is the diversity of the genetic defect. More than 2000 different mutations have been identified in CF patients, and while only 12 mutations are represented in 50% of the CF population, more than 1900 mutations are distributed in the other 50%.
In an assay developed by its founders, HUB has demonstrated that we can determine a patient’s clinical response (or lack of response) based directly on an organoid of that patient. In addition, HUB has generated a CF biobank which consists of more than 400 organoid cultures derived from rectal biopsies of CF patients and which represents more than 100 mutations, both common and rare. Using this CF biobank, the effectiveness of (novel) compounds on the CF population or on specific mutations can be accurately determined in vitro (Forskolin-induced swelling (FIS) assay)