Small molecule drugs that target functional 3D RNA structures to treat human diseases.
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Ribometrix is discovering novel small molecular medicines that target RNA structure to treat human diseases. The vast majority of drugs are designed to target disease-driving proteins. Instead, Ribometrix is identifying therapeutic drugs that target disease-causing RNAs that downstream could be used to make proteins or the RNAs can have a regulatory role in disease progression. Drugging RNA is now possible due to technologies developed by Ribometrix that enable them to discover the 3D structure of RNA. By analyzing these structures, Ribometrix has uncovered that RNAs contain many clefts and crevices which can be bound by small molecule drugs, modulating the function of the RNA. Ribometrix is collaborating with Genentech to commercialize therapeutic candidates.
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Durham, NC|100+ days ago