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SMiRNA™ Up Close
RNA structures drive disease that can be targetable by orally available small molecules
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We define druggable RNA structures by using knowledge of genetics and evolution
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We utilize proprietary tools to small molecule libraries, Chem-CLIP, NMR /docking /computational tools “RNA specific medicinal chemistry) efficiently identify drug-like small molecules that bind and modulate these structures.
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We solve three-dimensional structures of small molecule-RNA complexes to enable efficient optimization of lead medicines
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We leverage RNA-specific medicinal chemistry to develop potent and specific compounds.
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We develop target specific biochemical assays and analysis in patient-derived cells
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Lead compounds are optimized in pre-clinical models to deliver orally bioavailable medicines to treat the whole patient