N30 has started a phase 1b/2a trial with CFs who have 2 copies of F508del. They are trialling a compound called N6022, which is both anti-inflammatory and a CFTR corrector:

“N6022 is the first of a novel class of N30 Pharma’s drugs aimed at conservation of S-nitrosoglutathione (GSNO) a key, endogenous, signaling molecule that has been shown to be reduced in cystic fibrosis patients. N30 Pharma’s drugs restore GSNO levels by inhibiting GSNOR, the enzyme in the body that breaks down GSNO.

N30 Pharma’s GSNOR inhibitors have dual effects in cystic fibrosis. First, in experimental models of cystic fibrosis, they rescue or “correct” the abnormal chloride channel function that causes the thick, tenacious secretions of the lung and other organs characteristic of cystic fibrosis. Secondly, they are potently anti- inflammatory. This is potentially important because inflammation, particularly of the lung, is the cause of major morbidity in CF patients.”

Source: http://www.n30pharma.com/docs/news/2013-03-12-6022-CF-First-Patient.pdf

Link to clinical trial (being held in the US): http://www.clinicaltrials.gov/ct2/show/NCT01746784?term=n30&rank=3


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1. Class 2 Mutations & F508del, 3. N30 - N6022 & N91115