New Patent Strengthens Regulus’ Leadership in microRNA Therapeutics
CAMBRIDGE, Mass. & CARLSBAD, Calif. — Regulus Therapeutics, a joint venture between Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) and Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) formed to discover, develop, and commercialize microRNA therapeutics, announced today that the United States Patent and Trademark Office (USPTO) recently issued a "Notice of Allowance" for the "Sarnow" patent (U.S. patent application 11/122,328). The allowed claims cover methods for targeting a specific microRNA to inhibit Hepatitis C virus (HCV) replication. The Sarnow patent, a Stanford University patent to which Regulus has exclusive rights, relates to the discovery and development of therapeutic products for HCV infection by inhibiting a liver-specific microRNA known as miR-122. Regulus is developing a microRNA therapeutic targeting miR-122 for the treatment of HCV infection as its most advanced therapeutic program.
"The opportunity to selectively antagonize microRNAs involved in the cause or pathway of human disease represents an exciting new frontier for pharmaceutical research. The recent formation of Regulus unites the scientific leadership and intellectual property estates of both Isis and Alnylam to build what we believe is the leading microRNA therapeutics company, and this new U.S. patent allowance represents an early proof point of our value creation strategy," said John Maraganore, Ph.D., Chairman of Regulus, and President and Chief Executive Officer of Alnylam. "HCV is a serious infectious disease representing a major public health concern affecting 170 million people worldwide. Most current and emerging anti-HCV therapies target viral genes and are therefore prone to the emergence of resistance. By targeting a host factor required for viral replication, a microRNA therapeutic targeting miR-122 is an attractive and differentiated approach for the possible treatment of HCV."
"miR-122, one of over 500 microRNAs expressed in human cells, has been shown to be an important host factor for HCV replication and a promising target for microRNA-based therapeutics," commented C. Frank Bennett, Ph.D., Senior Vice President of Research of Isis. "We are pleased to announce this patent allowance, as we feel that the Sarnow patent will provide Regulus with broad protection for its most advanced therapeutic program, which targets miR-122."
Data published in 2005 by the Sarnow lab in the journal Science [Jopling et al. Science 309, 1577 (2005)] demonstrated that miR-122, which is specifically and abundantly expressed in the human liver, is required for HCV proliferation. In these studies, miR-122 was found to interact directly with a specific part of the HCV genome leading to increased expression of viral components. Sequestration of miR-122 using antisense oligonucleotides resulted in a dramatic inhibition of HCV reproduction in cultured human liver cells. This links the endogenous expression of a specific microRNA with a major infectious disease, and suggests that antagonism of miR-122 may comprise a novel therapeutic strategy against HCV.
Following a Notice of Allowance, the process resulting in final issuance of a patent involves several administrative steps that are typically completed within a year.
About microRNAs
microRNAs are a recently discovered class of genetically encoded small RNAs, approximately 20 nucleotides in length, and are believed to regulate the expression of a large number of human genes. microRNA therapeutics represent a new approach for the treatment of a broad range of human disease. When inappropriately encoded, microRNAs represent potential disease targets whose selective antagonism can result in the correction of an entire disease pathway in a manner unachievable by today’s medicines. In fact, the inappropriate absence or presence of specific microRNA molecules in various cells has been shown to be associated with specific human diseases including cancer, viral infection, and metabolic disorders.
About Regulus
Regulus Therapeutics LLC is a biopharmaceutical company formed to discover, develop and commercialize microRNA therapeutics. The company was created as a joint venture between Alnylam Pharmaceuticals, a leader in RNAi therapeutics, and Isis Pharmaceuticals, a leader in antisense technologies and therapeutics. Isis and Alnylam scientists and collaborators were the first to discover microRNA antagonist strategies that work in vivo in animal studies (Krutzfeldt et al. Nature 438, 685-689 (2005); Esau et al. Cell Metab., 3, 87-98 (2006)). Isis and Alnylam have also created and consolidated key intellectual property believed by the companies to be required for development and commercialization of microRNA therapeutics. The company, founded in 2007, maintains facilities in Carlsbad, California. For more information, visit www.regulusrx.com.
About Alnylam Pharmaceuticals
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