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CCM-848, CCM-914, and CCM-930 are a series of small molecule Translational Readthrough Inducing Drugs (TRIDs) developed by Chakrabarti Advanced Technology and its commercial arm, CCM Bio. These compounds are designed to treat genetically inherited diseases caused by nonsense mutations, which result in premature stop codons and truncated, non-functional proteins. The mechanism of action involves promoting the translational readthrough of these premature stop codons, thereby restoring full-length protein expression. Licensed from the University of Palermo and the Italian Cystic Fibrosis Foundation, these leads are currently in IND-enabling development for orphan diseases including Cystic Fibrosis and Duchenne Muscular Dystrophy. Preclinical data in mouse and zebrafish models suggest these compounds are non-toxic and exhibit significantly lower cytotoxicity than ataluren, a previously rejected readthrough drug.
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