NV914

NV914 is an inhibitor of FTSJ1 (tryptophan tRNA-specific 2'-O-methyltransferase) that blocks the methyltransferase activity of FTSJ1. It induces the readthrough of premature stop codons, thereby allowing the synthesis of full-length proteins from genes carrying nonsense mutations. As a translation readthrough inducing compound (TRID), NV914 demonstrates readthrough activity for nonsense mutations in vitro without affecting natural stop codons, thus restoring CFTR protein expression. In mice, NV914 shows good acute oral tolerance, presenting low health risks and demonstrating favorable safety. NV914 is applicable in research related to cystic fibrosis and Shwachman-Diamond syndrome.