The vast majority of drugs used to treat human disease target proteins. Only a small fraction of proteins known to cause disease have ever been successfully "drugged." Ribometrix is focused on completely changing the landscape of disease treatment using small molecules and is doing so by targeting RNA. Numerous diseases could be cured and lives of many millions of people could be improved by changing the behavior of disease-causing RNA.
Until recently, it was impossible to target RNA broadly and reproducibly because the three-dimensional structures of most RNAs were unknown. Ribometrix uses specialized technologies to discover three-dimensional RNA motifs in therapeutically compelling RNAs. Ribometrix has shown that many RNAs implicated in disease contain clefts and crevices into which small molecule drugs will bind. Ribometrix scientists use state-of-the-art strategies to identify ligands that bind specifically in these crevices and therapeutically modulate these RNAs.
Some RNAs are important because they are messengers that carry the information necessary to synthesize a protein. Targeting these messenger RNAs means that it is possible to "drug" a protein even before it has been made. Many RNAs do not encode for downstream proteins but instead have direct regulatory functions. These RNAs, called non-coding RNAs, comprise up to 70% of the human genome and represent a huge opportunity for novel therapeutics.
Drugging RNA is now uniquely possible due to technologies available to and created by Ribometrix. Welcome to a whole new druggable RNA world!