Regulus Therapeutics Inc. announced 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 programme 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 programmes 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.
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.
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 and Isis Pharmaceuticals.