Regulus Therapeutics, Inc. Initiates an Orphan and Rare Disease microRNA Therapeutic Effort in Brain Cancer
"GBM is a devastating disease with limited treatment options," said Neil W. Gibson, Ph.D. Chief Scientific Officer of Regulus Therapeutics. "At Regulus, we have successfully targeted microRNAs in multiple disease settings and believe that targeting dysregulated microRNAs using chemically modified oligonucleotide anti-miRs provides a potential therapeutic approach for GBM."
"A major hurdle in the discovery and development of novel therapeutics for GBM has been the lack of relevant animal models," said Do Hyun Nam, Ph.D. Professor and Director of Institute for Refractory Cancer Research, Samsung Medical Center. "In our collaboration with Regulus, we will utilize a recently developed model in which tumors derived from human patients are transplanted into mice resulting in a model directly relevant to human disease."
"Regulus has an impressive track record of executing on innovation in the field of microRNA therapeutics," said Max Wallace, Chief Executive Officer, ABC2. "The combination of Regulus' expertise in microRNA therapeutics with Samsung's preclinical GBM models compelled us to support this promising collaborative approach."
Glioblastoma multiforme, also known as glioblastoma or grade IV astrocytoma, is a fast growing brain tumor that forms from glial (supportive) tissue of the brain. GBM is the most prevalent form of primary brain tumor and peak incidence is in adults between 45 and 70 years. Treatment options are limited and survival is little over one year. GBM is considered a rare, or orphan, disease with an incidence in the U.S. of approximately 12,000 per year.
Regulus is advancing multiple microRNA therapeutic programs to the clinic in the areas of fibrosis, HCV infection, immuno-inflammatory disease, metabolic disease, and oncology.
About microRNAs
The discovery of microRNA in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 700 microRNAs have been identified in the human genome, and over one-third of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human 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 potential for a novel class of therapeutics and offers a unique approach to treating disease by modulating entire biological pathways.
About Regulus Therapeutics, Inc.
Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus is using a mature therapeutic platform based on technology that has been developed over 20 years and tested in more than 5,000 humans. The company works with a broad network of academic collaborators and leverages the oligonucleotide drug discovery and development expertise of its founding companies,