Isis and Alnylam Launch Regulus Therapeutics

Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. launched Regulus Therapeutics LLC, a joint venture focused on the discovery, development, and commercialization of microRNA (miRNA) therapeutics. These therapeutics represent a new approach to target the pathways of human disease. Regulus will combine Isis’ and Alnylam’s technologies and intellectual property with leadership from a management team and Scientific Advisory Board to be chaired by Nobel laureate David Baltimore and include key pioneers in the miRNA field.

Regulus will have exclusive licenses to Isis and Alnylam’s  miRNA therapeutic applications, as well as certain patents in the miRNA field including the “Tuschl III” patent. Alnylam will make an initial investment of $10 million and thereafter Isis and Alnylam will share funding of Regulus. Alnylam and Isis will retain rights to develop and commercialize miRNA therapeutic products that Regulus decides not to develop. Regulus will be operated as an independent company.

“The emerging biology of microRNAs points to a completely new understanding of cellular mechanisms for regulation of gene expression,” said Dr. Baltimore, of California Institute of Technology. “I believe that microRNAs represent previously unexplored disease targets where pharmacological approaches could lead to the emergence of novel therapies for many human disorders. Accordingly, I’m very excited to join in the formation of Regulus and to help build the leading microRNA therapeutics company.”

“The opportunity to antagonize microRNAs could create a new frontier for pharmaceutical research where an entire disease pathway is targeted for intervention, not just a single disease target. We believe microRNA therapeutics could have profound implications for the treatment of a broad range of diseases including cancer, viral infection, and metabolic disorders,” said John Maraganore, Ph.D., president and chief executive officer of Alnylam. “Isis’ and Alnylam’s intellectual property and technologies open the door to these new opportunities and, when combined to form Regulus, create an unmatched effort to establish the leading microRNA therapeutics company.”

“We are excited to embark on this venture, which represents an opportunity to invest in a focused expansion of our ongoing microRNA research efforts through Regulus’ application of our antisense technology platform to create microRNA therapeutics. It is timely to extend our know-how and clinical advances with antisense drugs to the field of microRNAs, an area that stands at the forefront of modern biology,” said Stanley Crooke, M.D., Ph.D., chairman and chief executive officer of Isis. “Regulus will be fully enabled to create a bold and successful new venture.”

Regulus’ new Scientific Advisory Board will be chaired by Dr. Baltimore, who will also serve as director, and initially will comprise the following members: David Baltimore, Ph.D., Professor of Biology at California Institute of Technology and the recipient of the 1975 Nobel Prize in Physiology of Medicine; David Bartel, Ph.D., Professor of Biology at MIT and a member of the Whitehead Institute for Biomedical Research; Scott Hammond, Ph.D., Assistant Professor of Cell and Developmental Biology at the University of North Carolina School of Medicine; Markus Stoffel, M.D., Ph.D., Professor for Metabolic Diseases at the Institute of Molecular Systems Biology, Swiss Federal Institute of Technology (ETH); Thomas Tuschl, Ph.D., Associate Professor at the Rockefeller University; and Phillip D. Zamore, Ph.D., Gretchen Stone Cook Professor of Biomedical Sciences at the University of Massachusetts Medical School.