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Small-Molecule Modulation of Read-Through (SMMRT): Discovery of 2-Phenoxyacetanilides as Promoters of PRotein Expression from RNA with Nonsense Codons. Richard G. Wilde et al.

IP.com Disclosure Number: IPCOM000019282D
Publication Date: 2003-Sep-09

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A class of 2-phenoxyacetanilides were discovered by HTS as modulators of mRNA read-through for the treatment of genetic diseases such as DMD. A cell-culture assay (with a luciferase reporter containing a nonsense muation) was used to optimize the SAR of the series. Compound 1 was significantly more potent than gentamicin. Compound 1 was stable in buffer solutions, but showed some degradation in mouse serum. Exposure in mice was much higher if dosed subcutaneously over oral dosing. Compound 1 showed superior efficacy in promotion of dystrophin synthesis in mdx mice compared to gentamicin at one-tenth the delivered concentration.