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The drug, called ensifentrine, blocks the activity of two enzymes involved in inflammation and is aimed at all cystic fibrosis patients independent from the mutation they carry.

In addition, it has been shown to activate rare mutations of the CFTR protein, reducing the amount and thickness of the mucus.

The treatments they currently receive aim to repair the defects caused by specific mutations in CFTR proteins, but their effect can be limited and patients still see their lungs getting inflamed and infected.“CFTR repair does not treat the over 10% of cystic fibrosis patients with nonsense mutations and does not cure cystic fibrosis.While the original version made use of tiny fat droplets to carry the DNA, the new one makes use of a hybrid virus.The researchers have modified a monkey lentivirus — a kind of virus that can insert genetic material in the host cell — to carry the external ‘targeting’ proteins of a virus that naturally infects the lung.“Normally with gene therapy, you are delivering small amounts to the brain or the eye, but for the lung you will need a considerable amount of viral vectors, especially if you’re going to nebulize it because maybe half of the vector will be lost just from the nebulization and, obviously, breathed out.”If the therapy proves able to modify enough lung cells, the partners expect the therapy would only need to be given every 3-5 years, or even just once.It would also remove the need for any other medications for lung function.

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