Regulus Therapeutics Announces U.S. Allowance of a Key Tuschl III Patent Covering miR-155

- microRNA-155 is a Lead Therapeutic Target of the Regulus-GlaxoSmithKline Immuno-Inflammatory Alliance –

- Third Allowance from the Tuschl III IP Estate Bolsters Leadership by Regulus in the Field of microRNA Therapeutics -

LA JOLLA, Calif.--(BUSINESS WIRE)-- Regulus Therapeutics Inc. announced today that the United States Patent and Trademark Office (USPTO) has allowed claims in a patent application (US Serial No. 12/550,579) derived from the "Tuschl III" patent series. The allowed claims relate to human microRNA miR-155, a microRNA that is a potential target for immuno-inflammatory indications, and a lead program of the Regulus-GSK Strategic Alliance. The strategic alliance with GSK was formed in 2008 to focus on developing microRNA therapeutics for the treatment of immuno-inflammatory diseases such as rheumatoid arthritis and inflammatory bowel disease.

“Regulus is continuing to execute its strategy of maintaining leadership in microRNA therapeutics by advancing multiple programs towards the clinic, expanding our dominant IP position, and forming significant alliances with a limited number of pharmaceutical partners,” said Garry E. Menzel, Ph.D., Executive Vice President Corporate Development and Finance of Regulus. “The Tuschl III patent estate has already yielded several patents directed to key therapeutic microRNA targets. These include miR-122, partnered with GSK for Hepatitis C Viral infection, and miR-21, the lead fibrosis target in our recent alliance with sanofi-aventis.”

“We are pleased with the decision of the USPTO to allow this new patent from the Tuschl III patent series which originated from the pioneering work performed by Dr. Thomas Tuschl while at Max Planck,” said Dr. Joern Erselius, Managing Director, Max Planck Innovation GmbH. “Regulus continues to demonstrate a leadership position in the field and is well positioned to develop and commercialize many successful microRNA therapeutic products.”

Upon activation, miR-155 is expressed in several types of human immune cells including B cells, T cells, macrophages and dendritic cells. Targeting miR-155 with anti-miRs offers a novel therapeutic approach for inflammatory diseases. The newly allowed claims cover the sequences of miR-155, as well as compounds that are complementary to miR-155.

In the field of microRNA, Regulus controls additional fundamental patent rights related to miR-155, including compositions of matter for various anti-miR compounds targeting miR-155, and uses of these compounds as therapeutic agents for the treatment of autoimmune and other inflammatory disorders. Regulus has access to the Tuschl III patent series for microRNA therapeutics through license agreements amongst Max Planck, Alnylam and Isis.

About microRNAs

The discovery of microRNA in humans is one of the most exciting scientific breakthroughs in the last decade. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length that do not encode proteins but instead regulate gene expression. Nearly 700 microRNAs have been identified in the human genome, and more than one-third of all human genes are believed to be regulated by microRNAs. As a single microRNA can regulate entire networks of genes, these new molecules are considered the master regulators of the genome. microRNAs have been shown to play an integral role in numerous biological processes including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation and human development. Most microRNAs are conserved across multiple species indicating the evolutionary importance of these molecules as modulators of critical biological pathways. Indeed, microRNA expression or function has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Targeting microRNAs with anti-miRs, antisense oligonucleotide inhibitors of microRNAs, or miR-mimics, double-stranded oligonucleotides to replace microRNA function, opens the possibility of a novel class of therapeutics and a unique approach to treating disease by modulating entire biological pathways. To learn more about microRNAs please visit

About Regulus Therapeutics Inc.

Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative new medicines based on microRNAs. Regulus is targeting microRNAs as a new class of therapeutics by working with a broad network of academic collaborators and leveraging oligonucleotide drug discovery and development expertise from its founding companies Alnylam Pharmaceuticals (Nasdaq:ALNY) and Isis Pharmaceuticals (Nasdaq:ISIS). Regulus is advancing microRNA therapeutics towards the clinic in several areas including hepatitis C infection, cardiovascular disease, fibrosis, oncology, immuno-inflammatory diseases, and metabolic diseases. Regulus’ intellectual property estate contains both the fundamental and core patents in the field and includes over 600 patents and more than 300 pending patent applications pertaining primarily to chemical modifications of oligonucleotides targeting microRNAs for therapeutic applications. In April 2008, Regulus entered into a major alliance with GlaxoSmithKline to discover and develop microRNA therapeutics for immuno-inflammatory diseases. In February 2010, Regulus entered into a new collaboration with GlaxoSmithKline to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of Hepatitis C Viral infection. In June 2010, sanofi-aventis and Regulus entered into the largest-to-date strategic alliance on microRNA therapeutics focused initially on fibrosis. For more information, visit

Forward-Looking Statements

This press release includes forward-looking statements regarding the future therapeutic and commercial potential of Regulus’ business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus, including the therapeutic potential of targeting microRNA-155. Any statement describing Regulus’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. Such party’s forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause their results to differ materially from those expressed or implied by such forward-looking statements. Although these forward-looking statements reflect the good faith judgment of the management of Regulus, these statements are based only on facts and factors currently known by Regulus. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Regulus’ programs are described in additional detail in each of Isis’ and Alnylam’s annual report on Form 10-K for the year ended December 31, 2009 and their most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from either Isis or Alnylam.


Regulus Therapeutics
Zachary Zimmerman, Ph.D., 858-202-6300
[email protected]
Russo Partners
Heidi Chokeir, Ph.D. (Media), 619-528-2217

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