Regulus Therapeutics

microRNA Explained

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

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, labeled RG-101, is intended as a treatment for hepatitis C and is in Phase 1 clinical trials. In June 2016 the U.S. Food and Drug Administration placed the product on clinical hold after two patients in the trial developed jaundice, identified as a serious adverse event.

Strategic Alliances

In April 2008, Regulus and GlaxoSmithKline (GSK) entered into a microRNA-focused strategic alliance for the discovery, development and commercialization of novel microRNA-targeted therapeutics to treat inflammatory diseases such as rheumatoid arthritis. In February 2010, Regulus and GSK announced a new collaboration to develop and commercialize microRNA therapeutics targeting microRNA-122 (miR-122) for the treatment of hepatitis C (HCV) infection.
Most recently, multi-national pharmaceutical giant Sanofi –Aventis awarded Regulus with the largest microRNA partnership to date – targeting fibrosis.

Research Collaborations

Regulus has active collaborations with leading academic researchers from over 30 academic research laboratories globally.

Patents

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.