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Regulus Therapeutics Inc. or Regulus () 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 Hepatitis C is an infectious disease caused by the hepatitis C virus (HCV) that primarily affects the liver; it is a type of viral hepatitis. During the initial infection people often have mild or no symptoms. Occasionally a fever, dark urine, ...
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 Alport syndrome is a genetic disorder affecting around 1 in 5,000-10,000 children, characterized by glomerulonephritis, end-stage kidney disease, and hearing loss. Alport syndrome can also affect the eyes, though the changes do not usually affect ...
in phase 2 clinical trials.


Strategic alliances

In April 2008, Regulus and
GlaxoSmithKline GSK plc, formerly GlaxoSmithKline plc, is a British Multinational corporation, multinational pharmaceutical and biotechnology company with global headquarters in London, England. Established in 2000 by a Mergers and acquisitions, merger of Gl ...
(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 Sanofi S.A. is a French Multinational corporation, multinational pharmaceutical and healthcare company headquartered in Paris, France. Originally, the corporation was established in 1973 and merged with Synthélabo in 1999 to form Sanofi-Synth� ...
–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.


References


External links


Regulus Therapeutics official website
{{Authority control Companies listed on the Nasdaq Pharmaceutical companies of the United States Health care companies based in California