● Latest advances in non-coding RNA research

● Transforming nucleic acids technologies

● Nucleic acids based therapeutics

● Nucleic acids based diagnostics and biomarkers

Dr. Chang’s honors include the Paul Marks Prize for Cancer Research, Judson Daland Prize of the American Philosophical Society, Howard Hughes Medical Institute Early Career Scientist, the Vilcek Prize for Creative Promise, Alfred Marchionini Research Prize, American Cancer Society Research Scholar Award, Damon Runyon Scholar Award, and elected membership to the American Society for Clinical Investigation. His work was honored by the journal Cell as a Landmark paper over the last 40 years and by Science as “Insight of the decade”.

Dr. Crooke is one of the pioneers, most experienced scientists and knowledgeable experts in the oligonucleotide therapeutics field. He established and for more than 20 years supervised drug discovery and development platform, resulting in growing number (currently nearly 30) of therapeutic programs with diverse indications, including cardiovascular and metabolic diseases, inflammation and cancer, severe and rare disorders. Many of the programs are now been developed by leading biopharmaceutical companies, such as AstraZeneca, Biogen Idec, GlaxoSmithKline, Pfizer, Sanofi, Teva, and advanced to phase 2 and phase 3 clinical trials. In January of 2013, an oligonucleotide inhibitor of apolipoprotein B-100 (Mipomersen, Kynamro), discovered and initially developed by Dr. Crooke’s team, was approved by the United States Food and Drug Administration for treatment of familial hypercholesterolemia. Throughout all the years with Ionis Pharmaceuticals, Dr. Crooke also continued contributing to the basics of oligonucleotide science, authoring more than 500 research articles and patents, and editing more than 20 books.

Dr. Crooke received his BS in Pharmacy from Butler University in 1966, PhD and MD at Baylor College of Medicine in 1971 and 1974, correspondingly. Earlier in his career, Dr. Crooke helped create the anticancer program at Bristol-Myers (now Bristol-Myers Squibb), and then led Research and Development at SmithKline Beckman (now GlaxoSmithKline). In 1989, he co-founded Ionis Pharmaceuticals, where he now serves as Executive Chairman and Chief Executive Officer. Dr. Crooke held professor positions at Baylor College of Medicine, University of Pennsylvania Medical School, University of California San Diego, and is currently a Member of the San Diego State University BioScience Center Scientific Advisory Board.

For his contribution to life sciences, Dr. Crooke received a number of awards and honors, including the Lifetime Achievement Award from Scrip, the Director of the Year Award from the Corporate Directors Forum, the Distinguished Scientist Award from the American Chemical Society, the Helix Award for the most important innovation in biotechnology by the Biotechnology Industry Organization, the Ernst and Young Entrepreneur of the Year Award, as well as Distinguished Alumnus at Baylor College of Medicine and at Butler University. In 2006, Nature Publishing Group listed him as decade’s one of the most remarkable and influential personalities in biotechnology.

Additional information about Dr. Crooke can be found in the following sites:

http://www.ionispharma.com/about/management/

http://ir.ionispharma.com/phoenix.zhtml?c=222170&p=irol-govBio&ID=189311

Chuan He is the John T. Wilson Distinguished Service Professor in the Department of Chemistry, Director of the Institute for Biophysical Dynamics at the University of Chicago, and an Investigator of the Howard Hughes Medical Institute. He is also a Cheung Kong Professor and Director of Synthetic Functional Biomolecules Center (SFBC) at Peking University in China. His recent research concerns reversible RNA and DNA methylation in biological regulation. Chuan He’s laboratory has spearheaded development of enabling technologies to study the biology of 5-hydroxymethylcytosine (5hmC) in mammalian genomes. His laboratory also discovered the reversible methylation of N6-methyladenosine (m6A) in human messenger RNA (mRNA) in 2011.

Rob Holt received his PhD in Pharmacology from the University of Alberta, Canada, in 1998. After a postdoctoral fellowship in molecular evolution at the State University of New York, Dr. Holt joined the company Celera Genomics in Rockville, Maryland where he served on Craig Venter’s team as the Scientific Operations Manager for initial sequencing of the human genome. Since 2003 Dr. Holt has been a Senior Scientist at the British Columbia Cancer Agency (BCCA), where he is Co-director of the BCCA Immunotherapy Program and Co-director of the Genome Canada Science & Technology Innovation Centre. Dr. Holt is recognized for his leadership role in decoding some of the first model organism genomes and pathogen genomes and, more recently, for developing next-generation sequencing methods for interrogating the genetics of the adaptive immune system. He has served as a scientific advisor to the NIH Human Microbiome Project and discoveries by his research group have linked new infectious agents to cancer risk. His current research directions are focused on cancer genomics, T cell engineering, and immune interventions in cancer. He has published over 130 scientific papers that have received >49,000 citations.

– Scientific interests and expertise
Genome engineering via programmable nucleases including the CRISPR-Cas system
1) Genome editing in plants, animals, and cultured human cells including iPS/ES cells
2) Engineered nuclease-mediated gene and cell therapy
3) Functional genomics using genome-scale libraries of programmable nucleases

– Received degrees (including places and years)
1987, BS, Dept. of Chemistry, Seoul National University
1989, MS, Dept. of Chemistry, Seoul National University
1994, PhD, Dept. of Biochemistry, University of Wisconsin-Madison

– Current position(s) and role(s) in affiliated organizations
1994-1997, Research Associate, Howard Hughes Medical Institute/MIT
1997-1999, Principal Investigator, Samsung Biomedical Research Institute
1999-2005, CEO and CSO, ToolGen, Inc.
2005-present, Assistant/Associate/Full Professor, Seoul National University
2014-present, Director, Institute for Basic Science (IBS)
Home pages: http://cge.ibs.re.kr/html/cge_en and http://gel.snu.ac.kr

Dr. Troels Koch (TK) is Ph.D. in bio-organic chemistry and has worked in the area of nucleic acid chemistry and biology for more than 15 years. TK is co-founder of several Biotech companies of which Exiqon A/S and Santaris Pharma A/S are most commonly known. He is presently Vice President of Research & CTO in Santaris Pharma A/S with main responsibilities to further build on the fundamental understanding of the chemical and biological properties of LNA, refining the LNA antisense drug discovery process, and establishing a drug pipeline on RNA antagonists targeting both mRNA and microRNA. Collectively this work and associated IP has been a driver behind the four major partner deals that Santaris has entered over the past years. During his academic and biotech career TK has gained experience in managing R & D activities, intellectual property, quality systems, regulatory affairs, antisense drug substance/product manufacturing, and nucleic acid bio-organic chemistry. TK is the author of about 70 peer reviewed scientific papers and inventor of about 25 base patents. He is a frequently invited speaker at international conferences, and he has accepted more than 70 invitations in the past decade.

Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986). He conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada, and then became a Professor of Biochemistry at the University of Konstanz, Germany (1992). He is currently responsible for R&D Alliance Management at Sanofi, and is the Scientific Managing Director of the Innovative Medicine Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes (www.compact-research.org).

Since joining Hoechst Marion Roussel in 1998, Dr. Leberer carried out various managing roles in this company, Sanofi’s predecessor companies and Sanofi itself, including responsibilities in functional genomics, biological sciences and external innovation for oligonucleotide-based therapeutics. He has also served as Head of Biotechnology Germany and a member of the Scientific Review Committee of Aventis Pharma Germany.

Prior to joining pharmaceutical industry, Dr. Leberer served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. His research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the principal discoverer of the p21 activated protein kinase (PAK) family of cell signalling proteins and of novel virulence-inducing genes in pathogenic fungi. He is co-author of more than 60 publications in prestigious peer-reviewed journals including Nature and Science.

Judy Lieberman holds a Chair in Cellular and Molecular Medicine at Boston Children’s Hospital, is Professor of Pediatrics at Harvard Medical School and is Chair of the Executive Committee of Immunology at Harvard Medical School. She earned a Ph.D. in physics from Rockefeller University and worked as a theoretical physicist at the Institute for Advanced Study in Princeton and Fermilab, received an M.D. from Harvard and MIT, did a postdoctoral fellowship in immunology in the Eisen laboratory in the Cancer Center at MIT and worked as a hematologist/oncologist at Tufts Medical Center. She has received numerous awards for her research on AIDS vaccines, immunology and cancer. She is a member of the American Academy of Arts and Sciences.

The Lieberman laboratory has been in the forefront of developing RNAi-based therapeutics and using RNAi for genome-wide screening. They have developed strategies for cell-targeted RNAi to treat viral infection, immune disease and cancer. They also investigate the role of microRNAs in regulating cell differentiation and cancer. The Lieberman laboratory also studies cytotoxic T lymphocytes and their role in immune protection from infection and cancer. They study the molecular pathways used by killer lymphocytes to induce programmed cell death of both mammalian cells and microbes, especially those activated by cytotoxic granule proteases, called granzymes, and immune pore-forming proteins.

Dr. Muthiah Manoharan serves as Senior Vice President of Innovation Chemistry at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. Dr. Manoharan joined Alnylam in 2003. He built the chemistry group at Alnylam and pioneered the discovery of GalNAc conjugated siRNAs for RNA interference (RNAi) based human therapeutics. He was the former Executive Director of Medicinal Chemistry at Isis Pharmaceuticals, Inc., a leading biotechnology company focused on antisense oligonucleotide-based therapeutics where he had a12-year tenure. With a distinguished career as a world-leading nucleic acid and bioconjugate chemist, Dr. Manoharan is an author of nearly 200 publications and over 300 abstracts, as well as the inventor of over 200 issued U.S. patents. Prior to Isis Pharmaceuticals, He earned his Ph.D. in chemistry at the University of North Carolina-Chapel Hill and conducted post-doctoral work at Yale University and the University of Maryland. He was the recipient of the M. L. Wolfrom award of the ACS Carbohydrate Chemistry Division in 2007.

Dr. Mello’s lab uses the nematode C. elegans as a model system to study embryogenesis and gene silencing.  His collaborative work with Dr. Andrew Fire led to the discovery of RNA interference (RNAi), for which they shared the 2006 Nobel Prize in Physiology or Medicine. Together they showed that when C. elegans is exposed to double-stranded ribonucleic acid (dsRNA), a molecule that mimics a signature of viral infection, the worm mounts a sequence-specific silencing reaction that interferes with the expression of cognate cellular RNAs. Using readily produced short synthetic dsRNAs, researchers can now silence any gene inorganisms as diverse as rice and humans. RNAi allows researchers to rapidly “knock out” the expression of specific genes and, thus, to define thebiological functions of those genes. RNAi also provides a potential therapeutic avenue to silence genes that cause or contribute to diseases.

Dr. Mello received his BS degree in Biochemistry from Brown University in 1982, and PhD from Harvard University in 1990. From 1990 to 1994, he conducted postdoctoral research at the Fred Hutchinson Cancer Research Center in Seattle, WA. Now Dr. Mello is an Investigator of the Howard Hughes Medical Institute, the Blais University Chair in Molecular Medicine and Co-director of the RNA Therapeutics Institute at the University of Massachusetts Medical School.

Besides the Nobel Prize, Dr. Mello’s work was recognized with numerous awards and honors, including the National Academy of Sciences Molecular Biology Award (2003), the Wiley Prize in Biomedical Sciences from Rockefeller University (2003), Brandeis University’s Lewis S. Rosnstiel Award for Distinguished Work in Medical Research (2005), the Gairdner Foundation International Award (2005), the Massry Prize (2005), the Paul Ehrlich and Ludwig Darmstaedter Award (2006), the Dr. Paul Janssen Award for Biomedical Research (2006), the Hope Funds Award of Excellence in Basic Research (2008). He is a member of the National Academy of Sciences, the American Academy of Arts and Sciences, and the American Philosophical Society.

Additional information about Dr. Mello can be found in the following sites:

http://www.hhmi.org/scientists/craig-c-mello

http://profiles.umassmed.edu/profiles/ProfileDetails.aspx?From=SE&Person=1009

http://www.nobelprize.org/nobel_prizes/medicine/laureates/2006/mello-bio.html

http://en.wikipedia.org/wiki/Craig_Mello#Awards_and_honors

Mikiko C. Siomi earned her Ph.D. in Agricultural Chemistry from Kyoto University, Japan in 1994, and then did post-doctoral studies with Prof. Gideon Dreyfuss, HHMI/University of Pennsylvania School of Medicine. Later, Dr. Siomi earned another Ph.D. in Medical Science from the University of Tokushima, Japan in 2003. Dr. Siomi started a joint laboratory with Prof. Haruhiko Siomi in the University of Tokushima, Japan in 1999 for elucidating the function of FMRP and the mechanism of RNAi using the Drosophila system. The laboratory discovered that Ago2 protein, the key player of RNAi, interacts with FMRP, an RNA-binding protein that is encoded by the fmr1 gene, the responsible gene for causing Fragile X Mental Retardation. Later, Dr. Siomi focused on elucidating how RNAi mechanistically occurs and the molecular mechanisms of piRNA biogenesis in the germlines. Dr. Siomi started her own laboratory at the University of Tokyo in 2012 (http://www-siomilab.biochem.s.u-tokyo.ac.jp/index.html). Dr. Siomi co-authored numerous research articles, reviews and book chapters, and currently serves as the president of the RNA Society of Japan and the vice-president of the Molecular Biology Society of Japan.

Erik Sontheimer is Professor in the RNA Therapeutics Institute (RTI) at the University of Massachusetts Medical School. He received his Ph.D. in 1992 from Yale, where he did his thesis work with Joan Steitz on pre-mRNA splicing mechanisms. He was then a Jane Coffin Childs Fund postdoctoral fellow with Joe Piccirilli at the University of Chicago. In 1999, Sontheimer joined the faculty at Northwestern, where he turned his attention to small RNA-based gene regulation in eukaryotes. In 2008 his laboratory also began working on genetic interference in bacteria. Among other advances, they provided the first demonstration that CRISPR RNAs (crRNAs) can target DNA, as well as the first explicit recognition of crRNA’s potential for gene targeting in eukaryotes, pointing the field down the path of RNA-guided genome engineering. He has received an NSF CAREER Award, a New Investigator Award from the Burroughs Wellcome Fund, and the Nestlé Award from the American Society for Microbiology, and he has also been elected to the American Academy of Microbiology. In 2014 he moved to the RTI, where he is continuing his research on the fundamental roles of RNA molecules, and on their uses in biomedical research and medicine. He is a co-founder and Scientific Advisory Board member of Intellia Therapeutics, which is developing Cas9-based genome editing for clinical applications.

Born in Shanghai, after high school, he was sent to France by Chinese government to study theoretical physics. After M.S. degree, he served as assistant professor in the Department of Physics at Fudan University. Later on, he returned to Paris to study nucleic acids, was awarded Ph.D and Habilitation degrees in biophysics at Pierre & Marie Curie University. He also had an entrepreneurial training at HEC business school.

His research in nucleic acids (100+ peer-reviewed publications, 8 patents) led him to co-invent an original concept with Dr. Marie Dutreix at the Institut Curie – “the signal interfering DNA (siDNA)” which jams the recognition and signaling of double strand break (DSB) by using a short dsDNA mimicking a DSB. Acting agnostically at upstream, it can blind DSB repair signaling, thus inhibit all DSB repair pathways causing cancer cell death due to unrepaired DSB, while preserving normal cells.

In 2006, he co-founded DNA Therapeutics, served as Chairman & CEO, managed from scratch to clinical stage this virtually integrated biopharmaceutical company and executed from concept to clinic a 1^st-in-class drug development with the help of the experts aggregating skills in early stage drug development, CMC, regulatory affairs and business development. After demonstrating good safety and significant antitumor activity of the 1^st– in-class siDNA drug candidate in a phase I/IIa trial in patients with cutaneous metastatic melanoma (presented at the ASCO 2015), the Company was acquired in early 2016 by Onxeo – a public company specializing in the development of innovative oncology therapeutics. After closing, he is back to MNHN, focusing his research on the interplay between DNA damage, cancer and aging.

Prof. Sun received the physical chemistry 1991 award by French Chemical Society, the joint Grand Prize of Life Science 2006 by French Senate, INSERM-Transfert and ESSEC, the Award of Best Innovative Entrepreneur in Health 2008 by French Business Angels investing in health, the Next Gem award of best biotech at Biovision Investor Conference 2013.

Elizabeth Tran earned her PhD in biochemistry at North Carolina State University where she developed an in vitro assembly and methylation system for trans acting box C/D snoRNAs. She then pursued postdoctoral training in the laboratory of Dr. Susan Wente at Vanderbilt University, where she identified the role of the RNA helicase Dbp5 in nuclear mRNA export. She joined the faculty at Purdue University in 2009, where she explores the biochemical mechanism and biological function of DEAD-box RNA helicases, a class of enzymes that are required for all aspects of RNA metabolism but whose in vivo roles are yet to be identified. Her laboratory is most well known for studies of the DEAD-box RNA helicase Dbp2 in S. cerevisiae and insights into the roles of long non-coding RNAs (lncRNAs) in gene expression. These insights span the fields of RNA biology, epigenetics, and metabolism. Moreover, the scientific community has highlighted her work for pivotal, paradigm shifting advances in lncRNA biology (Best of JBC 2012, Nature highlight, Science Signaling highlight). Her long-term goal is to understand the connection between RNA structure, gene regulation, and cellular adaptation in relationship to organismal survival and human pathology. In addition to research, Dr. Tran is a strong supporter of the international scientific community and mentoring the next generation of scientists. She is currently serving a two year term as a Director on the Board of the RNA Society, an international organization with ~1000 members worldwide.

Ada Yonath is focusing on protein biosynthesis, on antibiotics hampering it, on pathogenic parasites and on the origin of life. She graduated from the Hebrew University, Jerusalem, and postdocted at Carnegie-Mellon and MIT (USA). In the seventies she joined the Weizmann Institute and established the first structural-biology laboratory in Israel. During 1986-2004 she also headed Max-Planck-Research-Unit for Ribosome Structure in Hamburg. Among others, she is a member of US-National-Academy-of-Sciences; Israel Academy; German Science Academy (Leopoldina); the Pontificia Accademia-delle-Scienze (Vatican). She holds honorary doctorates from the universities of Oslo, NYU, Mount-Sinai, Oxford, Cambridge, Hamburg, Berlin-Technical, Patras, De-La-Salle, Xiamen, Lodz, Strasbiurg. Her awards include the Israel Prize; Louisa-Gross-Horwitz Prize; Linus-Pauling Gold Medal; Wolf-Prize; UNESCO/L’Oreal Award; Albert-Einstein-World-Award for Excellence; Nobel Prize for Chemistry.

Yi-Tao Yu received his PhD degree in Molecular Biology from Case Western Reserve University in 1994. He was awarded a post-doctoral fellowship from the Damon Runyon Cancer Research Foundation, and did his post-doctoral work (RNA biology) with Joan Steitz at Yale University (HHMI) from 1995 to 1999. He then joined the faculty of the Department of Biochemistry and Biophysics at the University of Rochester in late 1999. He is currently also a member of the Center for RNA Biology and Chair of the RNA Structure and Function Cluster at the University of Rochester. Dr. Yu’s research interests are in the areas of RNA modification, snRNP biogenesis and pre-mRNA splicing. Over the years, he has generated numerous publications and made significant contributions in these areas.