RNAi, microRNA, siRNA - Fad or Future

Wednesday, April 23
3:30 pm

The industry for this new generation of drugs has grown to over $14 billion in market cap and M&A deals in less than two years.  A whole new support industry is being spawned around this new class of drugs, providing a novel approaches to IP, delivery systems and manufacturing techniques.  History has shown that you can make money selling picks and shovels even if you don't find gold.  This panel will debate both the short-term and long-term opportunities for RNA-based therapeutics.

Moderator:

• Dr. Reid J. Leonard, PhD, Executive Director, Licensing and External Research, Merck Research Laboratories

Panelists:

• Judy Lieberman, PhD, MD, Director, Division of AIDS, Harvard Medical School and Senior Investigator, Immune Disease Institute
• Bruce Booth, Principal, Atlas Venture and Chairman, Miragen Therapeutics
• Roberto Guerciolini, MD, Sr Vice President, Pharmaceutical Development, Dicerna Pharmaceuticals
• Chiang Li, MD, FACP, Chairman and CEO, Boston Biomedical, Inc. and Director, Skip Ackerman Center for Molecular Therapeutics, Division of Gastroenterology, Beth Isreal Deaconess Medical Center, Harvard Medical School

• Dr. Reid J. Leonard, PhD, Executive Director, Licensing and External Research, Merck Research Laboratories

Dr. Reid Leonard is Executive Director, External Research and Licensing for the Merck Research Laboratories. Reid's home base is Merck's Boston research facility at 33 Avenue Louis Pasteur, in the Longwood Medical Area.

Reid's role is to identify partnering opportunities that fit with Merck's strategic research and development goals across all therapeutic and technology areas. This is an outreach function to complement Merck's existing world-wide licensing and collaborative research activities.  As such, the focus is on early-stage opportunities: university technologies, incubator and start-up companies, and programs within established companies that may be a "good fit" for Merck, but which are not (yet) actively seeking a partner.  His goal to build relationships that will foster lasting, productive partnerships between Merck and other members of the biomedical research community. Reid's geographic area of responsibility includes the Eastern United States and Canada, with a heavy concentration on the Boston/Cambridge bio-pharma cluster.

Dr. Leonard graduated from Brandeis University with a BA in biology and psychology. He earned a Ph.D. in biology (neuroscience) from Purdue. Following a postdoctoral fellowship in molecular pharmacology at Caltech with Profs Norman Davidson and Henry Lester, Reid joined the Merck Research Labs in Rahway, NJ. After ten years of basic and discovery research in ion channel biology, Reid moved into External Scientific Affairs, where he was responsible for the scientific evaluation of partnering opportunities in several therapeutic areas, most recently endocrine/metabolic and cardiovascular diseases. In November 2005, Reid transitioned to the "licensing ambassador/scout" role in Boston.

• Judy Lieberman, PhD, MD, Harvard Medical School

Judy Lieberman, MD, Ph.D., is the Director of the Division of AIDS at Harvard Medical School and a Senior Investigator at the Immune Disease Institute. The Lieberman laboratory was the first to demonstrate in an animal model that RNA interference (RNAi) could be used to protect animals from disease. Her laboratory is actively working to harness RNAi for therapeutic use for HIV and other indications and has developed novel strategies for cell-specific targeting of small interfering RNAs that are effective in vivo. The lab is also investigating the role of the endogenous microRNA pathway in hematopoietic cell differentiation, cellular transformation and viral infection. Her laboratory is working on siRNA-based therapy and methods for delivering siRNAs in vivo.

• Bruce Booth, Principal, Atlas Venture and Chairman, Miragen Therapeutics

Bruce Booth joined Atlas Venture in 2005 as a Principal in the life sciences group.  He focuses on new and emerging companies developing novel biotech or pharmaceutical products, therapeutic platforms, and innovative biomedical technologies.   

Previously, Bruce was a Principal at Caxton Health Holdings L.L.C., a healthcare-focused investment firm, where he focused on the firm’s private equity activities, ranging from early stage venture capital through late stage buyouts. Prior to Caxton, he was an Associate Principal at McKinsey & Company, a global strategic management consulting firm, where he advised clients on R&D productivity, corporate strategy, and business development issues across the biopharmaceutical sector.  

Bruce currently serves on the board of Atlas portfolio companies Miragen, Stromedix, and Zafgen.  He also works closely with ARCA, Archemix, Avila, Dynogen, NxStage Medical (NASDAQ: NXTM), Prestwick, and Proprius.

As a British Marshall Scholar, Bruce earned a Doctorate in Molecular Immunology from the Oxford University's Nuffield Department of Medicine. Prior to graduate studies, he worked on President Clinton’s Domestic Policy Council in the National Office of AIDS Policy. He received his Bachelor of Science degree summa cum laude in Biochemistry from the Pennsylvania State University.  

He currently serves as a Trustee of the New York Academy of Medicine, and is a term member of the Council on Foreign Relations. Bruce has also authored numerous scientific papers and biotech venture capital review articles, including several in Nature Biotechnology and Nature Reviews Drug Discovery.

• Roberto Guerciolini, MD, Sr Vice President, Pharmaceutical Development, Dicerna Pharmaceuticals

• Chiang Li, MD, FACP, Chairman and CEO, Boston Biomedical, Inc. and Director, Skip Ackerman Center for Molecular Therapeutics, Division of Gastroenterology, Beth Isreal Deaconess Medical Center, Harvard Medical School

Chiang Li, MD, FACP, serves as Chairman and CEO of Boston Biomedical Inc., Director of Skip Ackerman Center for Molecular Therapeutics, Division of Gastroenterology, Beth Israel Deaconess Medical Center of Harvard Medical School, MA. Dr. Li is an accomplished physician-scientist and biotech entrepreneur. Dr. Li’s laboratory at Harvard invented the transkingdom RNAi technology, a platform that enables simultaneous manufacture and delivery of RNAi therapeutics (Nature Biotechnology, 2006). Among other accomplishments, Dr. Li pioneered the concept of activated checkpoint therapy, namely treating cancer by activating DNA damage checkpoint/response pathways, which is in phase 2 global clinical trials for treating cancer. Dr. Li and his team invented ARQ 197, the first-in-class specific c-Met inhibitor, which has showed exceptional signs of efficacy and safety in Phase I trials, and is currently in global Phase 2 trials. Dr. Li discovered a major mechanism of T cell death in HIV infection (Science, 1995), which supports Tat protein as a target for AIDS vaccine and therapy. He has over 50 publications in leading biomedical journals and about 40 inventions.  In addition, Dr. Li is a seasoned biotech entrepreneur and executive. He served as Chief Scientific Officer & Executive Vice President of ArQule, Inc. (Nasdaq: ARQL), founder or co-Founder of Cyclis Pharmaceuticals (acquired), ArQule’s R&D unit (current ArQule, Nasdaq:arql), Cequent Pharmaceuticals (a leading therapeutic RNAi company), and Boston Biomedical, Inc. He has led or participated in financing efforts for private and public companies, and was instrumental in forming oncology partnerships of $399 million. He has served as Board of Directors or the Chairman of the Scientific Advisory Board of biotech companies.