Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. have announced a co-exclusive license agreement with Stanford University related to the discovery and development of therapeutic products for hepatitis C virus (HCV) infection by inhibiting liver-specific microRNA.
Data published on September 2, 2005 by Jopling et al. in the journal Science demonstrate that the microRNA known as miR-122 is required for HCV replication in mammalian cells.
This study shows link of endogenous expression of a specific microRNA with a major infectious disease, and suggests that antagonism of miR-122 may comprise a therapeutic strategy against HCV.
"The recent discoveries that over 250 human genes encode microRNAs and that these microRNAs may control gene expression for as much as one-third of the genome suggests that this part of the RNAi pathway plays a major role in human health and disease," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam Pharmaceuticals.
"As part of our March 2004 agreement with Isis, we are together actively engaged in consolidating intellectual property in the microRNA field, and researchers at both companies are working to identify novel therapeutic opportunities."
"The identification of miR-122 as a critical host factor for HCV infection is a remarkable finding that has implications for the discovery and development of novel anti-HCV therapeutics,” said C. Frank Bennett, Ph.D., Vice President, Antisense Research of Isis Pharmaceuticals.
“These exciting results further validate that inhibiting or antagonizing microRNAs using antisense oligonucleotides has the potential to lead to exciting new therapeutics for diseases with unmet medical needs."
"Together with Alnylam, we continue to believe that approaches to antagonize or possibly replace microRNA function with traditional antisense oligonucleotides or with siRNA will represent an important new therapeutic strategy for the future."
The study by Jopling et al. from the laboratory of Peter Sarnow, Ph.D. at Stanford University demonstrates that miR-122 interacts directly with a specific 5' noncoding sequence of the HCV genome leading to increased abundance of the viral mRNA.
Antagonism of miR-122 function using an antisense oligonucleotide resulted in a dramatic decrease of viral RNA.