N30 Pharmaceuticals recently released pre clinical data for its GSNORi inhibitors (N6022 and N91115):

– N30s GSNORi improved F508del CFTR expression and improved chloride channel function
in human airway cells from F508del homozygotes.

– N30s GSNORi corrected CFTR channel function in F508del mice, decreased lung inflammation, and relaxed smooth muscle in the airways in isolated rat trachea.

– This demonstrates “that GSNORi have the potential to affect CF lung disease by improving a number of factors including: chloride channel function, inflammation, and lung perfusion.”

– N30 “is the first company to discover and develop small molecules targeting GSNOR. N30 Pharma is conducting a clinical trial of its lead compound, N6022 (an injectable formulation) in patients with two copies of the F508del mutation. In parallel, the company is rapidly advancing an oral dosage form, N91115, through preclinical testing and plans to begin clinical trials of N91115 in the first half of 2014.”

Sources: http://www.n30pharma.com/docs/news/2013-04-02-Malaga.pdf


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