Regulus Therapeutics has announced 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," Mr. Maraganore added.
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.