Regulus is advancing multiple microRNA therapeutic programsto the clinic in the areas of fibrosis, HCV infection, immuno-inflammatorydisease, metabolic disease and oncology. MicroRNAs are small RNA molecules,typically 20 to 25 nucleotides in length, which do not encode proteins, butinstead regulate gene expression. More than 500 microRNAs have been identifiedin the human genome, and more than one-third of all human genes are believed tobe regulated by microRNAs. MicroRNA expression, or function, has been shown tobe significantly altered in many disease states, including oncology, fibrosisand metabolic diseases. Regulus believes that microRNAs are clinically relevanttherapeutic targets and may be ideally suited as biomarkers for these diseasestates and others, like MS. Emerging data has demonstrated that microRNAsignatures in blood can mimic the expression profile observed in diseasetissues. Regulus has a comprehensive intellectual property estate andoligonucleotide technology know-how.
In addition to its collaboration with Biogen Idec, Regulushas formed strategic alliances with AstraZeneca, GlaxoSmithKline and Sanofi.Earlier this year, the company presented data from its proprietary lead programtargeting microRNA-33a/b (miR-33a/b), which is currently in preclinicaldevelopment for the treatment of atherosclerosis.