Therapeutic Workshop
RNAi: "Fast-Forwarding a Nobel Idea"
Monday, February 11, 2008
9:30 AM
Duke of Windsor
The discovery of RNA interference (?RNAi?) has been heralded as a major breakthrough in science, opening up the potential for treatment of an exhaustive list of human diseases. Yet development of RNAi therapeutics still remain by and large early-stage endeavors as companies focus their efforts on securing intellectual property and developing proprietary delivery methods. In this session panelists will discuss why IP and delivery are essential to the RNAi development path and tell us where they think the field of RNAi will be in five years.
Moderators
- Michael King; Managing Director, Rodman & Renshaw
Panelists
- John Maraganore, Ph.D.; CEO, Alnylam Pharmaceuticals
- Kleanthis Xanthopolus, Ph.D.; CEO, Regulus Therapeutics
- Roberto Guerciolini, MD; SVP, Pharmaceutical Development, Dicerna Pharmaceuticals
- Tod Woolf, Ph.D.; President and CEO, RXi Pharmaceuticals
- Anton McCaffrey, PhD; Assistant Professor, Dept of Internal Medicine, University of Iowa
Who's Who
Expert: Dr. McCaffrey's research focuses the role of RNA in human disease and the development of nucleic acid based therapeutics. Recently, his team developed small animal models of hepatitis B virus (HBV) and hepatitis C virus (HCV). They have used these models, in conjunction with bioluminescence imaging, to perform in vivo structure/function studies of HCV translational initiation. Using in vivo imaging in mice we were also able to show that RNA interference (RNAi) is functional in adult mice. The team then used the methodologies developed in these pilot studies to design and test RNAi-based inhibitors of HBV in mice. They found that short hairpin RNAs (shRNAs) were potent inhibitors of HBV replication in vivo . They are currently assessing the feasibility of delivering nuclease-stabilized short interfering RNAs as well as HBV shRNAs expressed from viral vectors in mice.