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N30 Pharma Announces Presentation of Preclinical Data at the Basic Science Meeting of the European Cystic Fibrosis Society
Date:4/2/2013

gs that conserve S-nitrosoglutathione (GSNO), an endogenous, signaling molecule that has been shown to modulate the function of key proteins involved in the pathophysiology of CF through S-nitrosation.  N30 Pharma's drugs restore GSNO levels by inhibiting GSNOR, the enzyme in the body that breaks down GSNO.  Levels of S-nitrosothiols, including GSNO, have been reported to be reduced in the lungs of CF patients.  Furthermore, GSNOR activity and expression have also been reported to be increased in airway cells from patients with CF compared with normal cells.

N30 Pharma's GSNOR inhibitors have a number of potential effects in CF.  In experimental models of CF, they rescue or "correct" the abnormal chloride channel function that causes the thick, tenacious secretions of the lung and other organs characteristic of CF.  They also have anti-inflammatory and bronchodilatory effects that are relevant to the pathogenesis of CF lung disease.

About Cystic FibrosisCF is the most common, life-threatening, genetic disease of Caucasians, affecting approximately 30,000 people in the United States and 70,000 people worldwide.  Approximately 48% of the total CF patient population in the United States have two copies of the F508del mutation, and an additional 39% of the total CF patient population have one copy of the F508del mutation.

CF is characterized by a defect in the chloride channel of human cells.  This channel, known as the "cystic fibrosis transmembrane conductance regulator", or CFTR, is critical for adequate water coating, or "hydration" in airways and other organs of the body. As a consequence of a mutation in the gene for CFTR, F508del-CFTR, patients produce a form of CFTR that is broken down and discarded in the cells of the body.  N30 Pharma's new drugs aim to rescue the damaged CFTR, decrease inflammation, and restore proper hydration to critical organs
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SOURCE N30 Pharmaceuticals, Inc.
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