Regulus Therapeutics

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.[2][3]

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.[4] The company's lead drug candidate, RG-012, is intended as a treatment for alport syndrome in phase 2 clinical trials.

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, the multi-national pharmaceutical giant [[Sanofi] –Aventis awarded Regulus with the largest microRNA partnership to date – targeting fibrosis.[2]

Regulus has active collaborations with leading academic researchers from over 30 academic research laboratories globally.[1]

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.[5]

• Regulus Therapeutics official website