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Alnylam, Regulus receive US patent covering microRNA Therapeutics

CambridgeThursday, August 13, 2009, 08:00 Hrs  [IST]

Alnylam Pharmaceuticals, Inc, a leading RNAi therapeutics company, and Regulus Therapeutics Inc, a leading microRNA therapeutics company, announced that the United States Patent and Trademark Office (US PTO) has awarded a notice of allowance for the Manoharan patent (application number 11/200,703), which covers several chemical classes of anti-miRs, antisense oligonucleotide inhibitors of microRNAs. This new patent in combination with Regulus' existing intellectual property estate broadly covers approaches to developing microRNA-based therapeutics. "Regulus Therapeutics is advancing a whole new frontier of pharmaceutical research through the discovery of microRNA therapeutics, which have the potential to block broad disease pathways as opposed to a single target," said Kleanthis G Xanthopoulos, president and chief executive officer of Regulus Therapeutics. "Our access to the pioneering research of our academic collaborators and Alnylam and Isis on oligonucleotide-based therapeutics, as evidenced most recently by the award of the Manoharan patent, has enabled Regulus to build a dominant intellectual property estate for microRNA therapeutics. We believe this is a strong foundation to advance our innovative approach to developing novel medicines to address the needs of patients." microRNAs are small, non-coding RNAs that regulate a large number of human genes; there are more than 700 microRNAs in the human genome that play a role in controlling expression of over one-third of all human genes. Abnormal expression and genetic mutation of microRNAs have been linked to numerous diseases including cancer, cardiovascular disease, metabolic disease, autoimmunity, and viral infection, amongst others. In 2005, Alnylam scientists and collaborators at The Rockefeller University were the first to describe antagomirs, which are part of a larger class of microRNA inhibitors known as anti-miRs (Krutzfeldt et al., (2005) Nature 438, 685-689). Anti-miRs were created to selectively inhibit microRNAs expressed in a broad range of tissues and to have pharmacological properties required for microRNA-based therapeutics. This research has created new drug discovery strategies focused on antagonizing microRNAs. "We are pleased to have this important recognition from the USPTO regarding our discovery of antagomirs, a new class of anti-miRs and a novel invention in the field of microRNA-based therapeutics," said Muthiah Manoharan, vice president for Drug Discovery at Alnylam. "With this initial grant, we have received broad claims covering classes of anti-miRs with certain important chemical features for optimal delivery and in vivo pharmacology. Importantly, the inventive nature of these novel compositions was allowed independent of any specific microRNA sequence, thereby providing a broad set of patent claims that add to the innovations Regulus can employ in designing novel microRNA therapeutics, an entire new class of medicines." microRNAs are a recently discovered class of genetically encoded endogenous RNAs, approximately 20 nucleotides in length, that are believed to regulate the expression of a large number of human genes. Regulus Therapeutics is a biopharmaceutical company aiming to discover, develop, and commercialize microRNA-based therapeutics. RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi.

 
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