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Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company. The Company is focused on discovering and developing drugs targeting microRNAs to treat diseases with significant unmet medical need. The Company is focused on orphan kidney diseases where microRNA genetic drivers are implicated and there are clear unmet medical needs. Its product candidate, RGLS8429, an anti-miR next generation oligonucleotide targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD), is in Phase 1b clinical development. In addition to this program, the Company continues to advance and expand its internal discovery pipeline to identify potential product candidates. microRNAs are naturally occurring ribonucleic acid (RNA) molecules that play a critical role in regulating key biological pathways. ADPKD is among the common human monogenic disorders and a cause of end-stage renal disease.
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