Regulus Therapeutics Inc. 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.[1]
On June 25, 2025, two months after reporting that it had reached an agreement, Novartis announced the completion of its purchase of the company.[2]
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.[3][4]
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.[5] The company's lead drug candidate, farabursen (RGLS8429), is in development for autosomal dominant polycystic kidney disease.
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,[when?] the multinational pharmaceutical giant Sanofi –Aventis awarded Regulus with the largest microRNA partnership to date – targeting fibrosis.[3]
Research collaborations
Regulus has active collaborations with leading academic researchers from over 30 academic research laboratories globally.[1]
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.[6]
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