Cystic Fibrosis - Sophion

Cystic fibrosis

Read more about cystic fibrosis drug discovery in our whitepaper co-authored with Enterprise Therapeutics

Cystic fibrosis (CF) is a devastating disease, graphically described from the patient perspective as drowning from the inside. It is an inherited disorder that causes organ damage throughout the body, with particularly severe damage to the lungs and digestive system, impairing normal growth and development.


In people with CF, a defective gene causes secretions in the lung and digestive tract to become sticky and thick. This leads to ducts, tubes, and passages becoming blocked, causing tissue damage and increasing the risk of infections, especially in the lungs.


Cystic Fibrosis is a channelopathy caused by mutations of the CFTR gene that lead to defects in CFTR chloride ion channel function, resulting in dysregulation of epithelial fluid transport in the lung. This dysregulation leads to mucus becoming thick and sticky. The role of mucus is now compromised; it fails to clear bacteria and other foreign matter, leading to infection and inflammation, and can ‘plug’ the airways, further compromising lung function.


CFTR is not the only chloride ion channel present in the airway epithelium – there is also a calcium-activated chloride conductance channel (CaCC). Although the presence of this channel has been known for decades, its molecular identity was only elucidated as TMEM16A (sometimes referred to as Anoctamin1 (Ano1)), in 2008.


Below see selected publications, posters and reports on relevant ion channel targets performed on QPatch or Qube.

Selected publications


  • View TMEM16A on Qube 384
    Year: 2017 First author: J McGivern


  • View TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis
    Year: 2020 First author: Danahay et Al.
  • View Development of a QPatch-Automated Electrophysiology Assay for Identifying TMEM16A Small-Molecule Inhibitors
    Year: 2020 First author: Henckels et Al.
  • View Development of Automated Patch Clamp Technique to Investigate CFTR Chloride Channel Function
    Year: 2017 First author: Billet, A et Al
  • View Mashiningan improves opioid-induced constipation in rats by activating cystic fibrosis transmembrane conductance regulator chloride channel
    Year: 2017 First author: Harada, Y.
  • View Drug Repurposing: The Anthelmintics Niclosamide and Nitazoxanide are Potent TMEM16A Antagonists that Fully Bronchodilate Airway
    Year: 2018 First author: Miner et Al., 2018
  • View Novel bacterial topoisomerase inhibitors derived from isomannide
    Year: 2020 First author: Okumu et Al.


  • View CFTR channels activated by flouride on QPatch
    Year: 2016 First author: Sørensen
  • View NIH 3T3 CFTR on QPatch
    Year: 2012 First author: Jacobsen et al.
  • View TMEM16A on Qube
    Year: 2017 First author: Sauter, D


  • View Automated Patch Clamp in Cystic Fibrosis Drug Discovery
    Year: 2021 First author: Gosling et al.
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