Founded in 2007 with headquarters in Boulder, Colorado.
N30 Pharma is developing a novel class of disease modifying therapies with a focus on cystic fibrosis. The new therapies preserve intracellular GSNO (S-nitrosoglutathione), a key regulator of CFTR (cystic fibrosis transmembrane conductance regulator) trafficking and stability.
Decreased GSNO levels are associated with cystic fibrosis, and with increased activity of GSNOR (GSNO reductase), the primary catabolizing enzyme of GSNO.
N30 Pharma has developed a broad portfolio of proprietary, potent, small molecule inhibitors of GSNOR that modulate F508del-CFTR activity.
- GSNO levels affect trafficking and stability of CFTR, the transmembrane regulator protein that is defective in cystic fibrosis
- GSNO inhibitors have been shown to increase CFTR function using in vitro and in vivo experimental models of cystic fibrosis, and have additive effects when combined with several different CFTR correctors and potentiators
- Anti-inflammatory effects of GSNOR inhibitors relevant to cystic fibrosis include decreased NFκB activation, neutrophilic infiltration, and elastase-mediated lung injuryLead compound, oral N91115, is progressing though Phase 1 testing in healthy subjects with studies in cystic fibrosis patients targeted for the second half of 2014
- Lead compound, oral N91115, is progressing though Phase 1 testing in healthy subjects with studies in cystic fibrosis patients targeted for the second half of 2014