Regulus Therapeutics Inc. or Regulus (NASDAQ: RGLS) is a clinical stage biopharmaceutical company focused on the development of first-in-class drugs that target microRNAs to treat a broad range of diseases. Regulus was established in September 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals.
MicroRNAs are small naturally occurring RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead have evolved to regulate gene expression.
Anti-miR therapeutics inhibit specific microRNA targets. Animal models showed that modulating microRNAs through anti-miRs effectively regulates biological processes and provides therapeutic benefit to cardiac dysfunction, cancer and hepatitis C virus infection. Administration of anti-miR oligonucleotides is possible through local or parenteral injection. The company's lead drug candidate, RG-012, is intended as a treatment for Alport Symdrome in Phase 2 clinical trials.
Regulus has active collaborations with leading academic researchers from over 30 academic research laboratories globally.
Regulus has more than 900 patents and patent applications, 600 of which cover the method of use, chemical modification and administration of oligonucleotides to address specific targets.