Drug intelligence / Profile preview

NMDI14

Development stage
Preclinical
Lead developer
Universidad de Córdoba
Modality
Small Molecules
Administration
Intraperitoneal
01

Overview

NMDI14 is a small molecule inhibitor of the nonsense-mediated mRNA decay (NMD) pathway. It specifically functions by disrupting the protein-protein interaction between SMG7 and UPF1, which are essential factors for the degradation of mRNAs containing premature termination codons (PTCs). By inhibiting NMD, NMDI14 can stabilize PTC-containing transcripts, which has therapeutic implications in diseases where NMD-mediated degradation of mutant transcripts exacerbates the phenotype, or in cancers where NMD supports tumor cell survival and progression. Research has shown that NMDI14 reduces the aggressiveness, proliferation, and migration of hepatocellular carcinoma (HCC) cells, suggesting its potential as a therapeutic agent for HCC and related liver conditions like metabolic dysfunction-associated steatohepatitis (MASH) and metabolic dysfunction-associated steatotic liver disease (MASLD).

02

Targets

SMG7

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