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Kurzfassung

• TMEM16A inhibitors represent a causal treatment for cystic fibrosis
• They reduce both excessive mucus formation and airway contraction
• Repurposing possible: TMEM16A inhibitors include FDA-approved and well-tolerated drugs

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Hintergrund

It is estimated that more than 70 000 patients worldwide are suffering from cystic fibrosis (CF), a lethal genetic disorder characterized by a severe pulmonary disease caused by production a highly viscous and adherent mucus. Because CF is caused by more than 2000 different mutations, a therapy from which all patients would benefit does not exist. An ideal causal therapy for CF would reduce the excessive mucus formation as well as the airway contraction.

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Bilder & Videos

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Lösung

Both can be achieved by using compounds that inhibit the calcium-activated chloride channel TMEM16A, which plays a crucial role in both airway smooth muscle contraction and mucus formation. Among such compounds, the FDA-approved and well-tolerated drug niclosamide and related substances have been shown to effectively reduce mucus formation in preclinical animal studies. The drugs may ideally be formulated for inhaled delivery with a spray or nebulizer.

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Anwendungsbereiche

• TMEM16A inhibitors reduce axcessive mucus production as well as airway contraction
• Possible repurposing of FDA-proven and well-tolerated drugs
• Development of a drug formulation for inhalation (in form of a spray or nebulizer)