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Cavosonstat is an orally bioavailable small molecule inhibitor of S-nitrosoglutathione reductase (GSNOR). It was developed as a modulator for cystic fibrosis, specifically targeting patients homozygous for the F508del-CFTR mutation. By inhibiting GSNOR, cavosonstat increases S-nitrosoglutathione (GSNO) levels, which in turn promotes maturation and plasma membrane stability of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This mechanism is complementary to existing CFTR correctors and potentiators. Cavosonstat reached phase II clinical trials but development for cystic fibrosis has been discontinued[1][3][5].
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