Regulus Therapeutics
# Regulus Therapeutics

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.

## 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, RG-012, is intended as a treatment for alport syndrome in phase 2 clinical trials.

## 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, the multinational 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.

