As advances in genetics continue to improve our understanding of disease, RNAi will have the opportunity to create entirely new classes of medicines.
Researchers say RNA-based therapeutic technology will have a tremendous impact on pharmaceutical research in the next five to 10 years. RNAi (RNA interference) represents a breakthrough in understanding how genes are turned on and off in cells.
“RNAi is a transformative discovery in modern biology that has already revolutionized biomedical research,” says John Maraganore, Ph.D., CEO of Alnylam Pharmaceuticals. “But the real opportunity with RNAi is to discover entirely new classes of medicines for patients. With RNAi therapeutics, we can harness the RNAi pathway and silence disease-causing genes that were previously…
Current RNA Research Efforts
RNAi At a Glance
Experts on this topic
Jim Jenson, Ph.D. CEO, Dicerna Pharmaceuticals, a private, venture-backed biopharmaceutical company developing novel therapeutic agents using proprietary technology that triggers RNA interference. For more information, visit dicerna.com.
John Maraganore, Ph.D. CEO, Alnylam Pharmaceuticals Inc., a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. For more information, visit alnylam.com.
Stuart W. Peltz, Ph.D. President and CEO, PTC Therapeutics Inc., a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. For more information, visit ptcbio.com.
Louis M. Renzetti, Ph.D. VP and Global Head, RNA Therapeutics, Roche, a research-oriented healthcare group with core businesses in pharmaceuticals and diagnostics. For more information, visit rocheusa.com.
Kleanthis G. Xanthopoulos, Ph.D. President and CEO, Regulus Therapeutics Inc., a biopharmaceutical company aiming to discover, develop, and commercialize microRNA-based therapeutics. For more information, visit regulusrx.com.
Daniel Zurr, Ph.D. CEO and President of Quark Pharmaceuticals Inc., a development-stage pharmaceutical company discovering and developing novel RNAi-based therapeutics. For more information, visit quarkpharma.com.