Thomas R. Cech, PhD (co-chair)

Professor, University of Colorado, Boulder, USA

Howard Hughes Medical Institute

Member of the US National Academy of Sciences

Nobel Prize Winner in Chemistry (1989)


Dr. Cech discovered catalytic properties of RNA, for which he shared the 1989 Nobel Prize in Chemistry. This discovery provided proof against the current belief that biological reactions can only be catalyzed by proteins, as well as support for the hypothesis proposing that RNA molecules were precursors to the life on Earth. Dr. Cech has also made major contribution to understanding structure and function of telomeres (structures at the chromosome ends) and telomerase (RNP complex responsible for telomere maintenance), and their roles in cancer. Dr. Cech’s most recent interest is in long non-coding RNAs, a novel class of molecules engaged in numerous and diverse cellular processes.


Dr. Cech earned his B.A. in Chemistry from Grinnell College in 1970, Ph.D. in Chemistry from the University of California Berkeley, completed post-doctoral research with Prof. Mary Lou Pardue at the Massachusetts Institute of Technology in 1977, and in 1978 joined the faculty of the University of Colorado Boulder, where he became a Howard Hughes Medical Institute Investigator in 1988 and Distinguished Professor of Chemistry and Biochemistry in 1990. In 2000-09 Dr. Cech served as President of the Howard Hughes Medical Institute, and in 2009 he returned to full-time research and teaching at the University of Colorado Boulder, where he also directs the BioFrontiers Institute.


Besides the Nobel Prize (1989), Dr. Cech received numerous awards and honors, including the Heineken Prize of the Royal Netherlands Academy of Sciences (1988), the Albert Lasker Basic Medical Research Award (1988) and the National Medal of Science (1995). Dr. Cech was elected to the US National Academy of Sciences in 1987 and the American Academy of Arts and Sciences in 1988. He is co-founder of the RNA Society in 1993 and Lifetime Professor at the American Cancer Society since 1987.


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

Craig C. Mello, PhD (co-chair)

Professor, UMass Medical School, USA

Howard Hughes Medical Institute

Member of the US National Academy of Sciences

Nobel Prize in Physiology or Medicine (2006)


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. For the layperson, RNAi is the cell’s search engine; the Google of the cell. Using readily produced short synthetic dsRNAs, researchers can now submit their own RNAi search queries to silence any gene in organisms as diverse as corn and humans. RNAi allows researchers to rapidly “knock out” the expression of specific genes and to thus define the biological functions of those genes. RNAi also provides a potential therapeutic avenue to silence genes that contribute to disease.


Dr. Mello received his B.S. degree in Biochemistry from Brown University in 1982, and Ph.D. 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.


Before the Nobel Prize, Dr. Mello’s work on RNAi was recognized with several awards including the National Academy of Sciences Molecular Biology Award(2003), the Canadian Gairdner International Award (2005), the Paul Ehrlich-and Ludwig Darmstaedter Award (2006), and the Dr. Paul Janssen Award for Biomedical Research (2006). 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:

Howard Y. Chang, MD, PhD

Professor, Stanford University, USA

Howard Hughes Medical Institute


Dr. Chang studies how activities of thousands of genes (gene parties) are coordinated to achieve biological meaning, with particular interest in understanding how long non-coding RNAs (lncRNAs) help cells to know and remember their locations in the body. He and co-workers pioneered methods to predict, dissect and control large-scale gene regulatory programs, which provide insights into human development, cancer and aging.


Dr. Chang received his A.B. degree (summa cum laude) in Biochemical Sciences from Harvard University in 1994, Ph.D. in Biology from Massachusetts Institute of Technology with Prof. David Baltimore in 1998, M.D. from Harvard Medical School in 2000, and completed post-doctoral training with Prof. Patrick O. Brown at Stanford University School of Medicine in 2004. He is currently a Professor of Dermatology at the Stanford School of Medicine and an Early Career Scientist at Howard Hughes Medical Institute. Dr. Chang is also a member of Stanford Bio-X Program, Stanford Cancer Center and Stanford Institute for Stem Cell Biology and Regenerative Medicine.


For his scientific achievements, Dr. Chang received numerous awards and honors, among others the Salvador E. Luria Lecture (Massachusetts Institute of Technology, 2012), the Montagna Lecture (Society for Investigative Dermatology, 2012), the Alfred Marchionini Research Prize (Alfred Marchionini Foundation, 2011), the CHANEL-CERIES Research Award (Chanel Research and Technology, 2010), the Senior Scholar Award in Aging (Ellison Medical Foundation, 2009), the Vilcek Prize for Creative Promise in Biomedical Science (Vilcek Foundation, 2009), and the American Cancer Society Research Scholar Award (2007).


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

Runsheng Chen, PhD

Professor, Institute of Biophysics,

Chinese Academy of Sciences, China

Member of the Chinese Academy of Sciences


Dr. Chen is one of the pioneer scientists in theoretical biology, genomics and bioinformatics in China. His group conducted and participated in several prominent studies, including the whole genome assembly and annotation for Thermoanaerobacter tengcongensis (the first complete bacterial genome developed in China), the 1% Human Genome Project and the Draft Sequence of the Rice Genome Project. As a member, Dr. Chen actively participates in the work of Human Genome Organization (HUGO), and his current interests are in identification and characterization of short, mid-range and long non-coding RNAs.


Dr. Chen completed his undergraduate studies in the Department of Biophysics at the University of Science and Technology of China (USTC) in 1964 and his graduate studies in the Institute of Theoretical Chemistry of Jilin University in 1980. From 1985 to 1987, he was a Fellow of the Alexander von Humboldt Foundation at the University of Erlangen-Nuremberg in Germany. Dr. Chen is currently a Professor in the Institute of Biophysics of Chinese Academy of Sciences.


Dr. Chen’s contribution to biosciences has been recognized through a number of national and international awards, including the Kotani Prize in 1996. Dr. Chen is an elected Member of the Chinese Academy of Sciences.


Additional professional info about Dr. Chen can be found in the following sites:

Carlo M. Croce, MD

Professor and Chair, Ohio State University, USA

Member of the US National Academy of Sciences

Foreign Member of Italian Academy of Sciences


During his early research, Dr. Croce discovered that genomic translocations associated with Burkitt’s lymphoma result in activation of MYC oncogene. He then analyzed other cancer-specific chromosomal translocations and identified more cancer causing-genes, including BCL2 (follicular lymphoma), TCL1 (T-cell leukemia) and ALL1 (acute leukemia). In the early 2000s, Dr. Croce demonstrated engagement in cancer of miRNAs, a new class of small regulatory RNA molecules. Since this pioneering research, different miRNAs were demonstrated to be involved in large variety of tumors, and are now considered to be among the most promising oncologic biomarkers and therapeutic targets. Dr. Croce’s studies uncovering the early events of various cancers pathogenesis have led to the development of novel and successful approaches to disease prevention, diagnosis, monitoring and treatment.


Dr. Croce received his M.D. degree (summa cum laude) from the Sapienza University of Rome, Italy in 1969, and moved to the United States the next year to initiate his research at the Wistar Institute of Anatomy and Biology. From 1976, Dr. Croce held professor positions at the Wistar Institute, the University of Pennsylvania School of Medicine, the Children’s Hospital of Philadelphia, the Temple University School of Medicine and the Jefferson Medical College of Thomas Jefferson University. Currently, Dr Croce is the Professor and Chair of Molecular Virology, Immunology and Medical Genetics, Director of the Human Cancer Genetics Program and Director of the Institute of Genetics at The Ohio State University Comprehensive Cancer Center. He is also a Professor of Medical Oncology at the University of Ferrara School of Medicine in Italy.


For his scientific contribution and achievements, Dr. Croce received numerous awards and honors, among others the National Institutes of Health Outstanding Investigator Award (1985, 1992), the General Motors Cancer Research Foundation Charles S. Mott Prize (1993), the Gottlieb Award from MD Anderson Cancer Center (2005), and the Henry M. Stratton Medal from the American Society of Hematology (2007). Dr. Croce was elected a Member of the US National Academy of Sciences (1996), a Foreign Member of the Accademia Nazionale delle Scienze in Italy (2003), and a Fellow of the American Academy of Arts and Sciences (2010).


Additional info about Dr. Croce can be found in the following sites:

Stanley T. CROOKE, MD, PhD

Founder, CEO and Chairman of the Board, Ionis Pharmaceuticals, USA


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:


Jennifer A. Doudna, PhD

Professor, University of California Berkeley, USA

Howard Hughes Medical Institute

Member of the US National Academy of Sciences


Dr. Doudna seeks to understand how non-coding RNA molecules control the expression of genetic information. Since her early research career, Dr. Doudna has been interested in the structure and function of various RNAs, particularly in developing the crystal structures of molecules with catalytic activities. She later expanded her research to studying the roles of RNA structures in regulation of protein synthesis initiation, understanding the structure and function of short interfering RNAs (siRNAs) in RNA interference (RNAi), and, most recently, the role of small RNA molecules derived from Clustered Regularly Interspaced Short Palindromic Repeats (CRISPs), or crRNAs, in bacterial immunity. The CRISP-based systems are currently used to modulate efficient site-specific mutagenesis in mammalian cells.


Dr. Doudna received her B.A. in Chemistry from Pomona College in 1985, and her Ph.D. in Biochemistry from Harvard University in 1989, under the mentorship of Prof. Jack Szostak. She completed her postdoctoral studies as Lucille P. Markey Scholar in Biomedical Science with Prof. Thomas Cech at the University of Colorado Boulder in 1994. As a member of the faculty of Yale University from 1994 to 2002, she was promoted to Henry Ford II Professor of Molecular Biophysics and Biochemistry. In 2002, she joined the faculty of the University of California Berkeley, where she is currently a Professor of Biochemistry and Molecular Biology and a Professor of Chemistry. She has been a Howard Hughes Medical Institute investigator since 1997. Dr. Doudna consulted for several pharmaceutical (Gilead and Merck) and biotechnology (Scriptgen and Nymirum) companies. She has ongoing scientific collaborations with Pfizer and Agilent Laboratories aimed at understanding and engineering the control of gene expression in various systems.


Dr. Doudna has received numerous awards and honors for her scientific achievements, including the Beckman Young Investigator Award (1996), the David and Lucile Packard Foundation Fellow Award (1996), the Johnson Foundation Prize for Innovative Research (1996), the National Academy of Sciences Award for Initiatives in Research (1999), the National Science Foundation Alan T. Waterman Award (2000), the Eli Lilly Award in Biological Chemistry from American Chemical Society (2001) and the American Association for the Advancement of Science Fellow Award (2008). She was also elected a Member of the US National Academy of Sciences in 2002, theAmerican Academy of Arts and Sciences in 2003 and the Institute of Medicine of the National Academies in 2010.


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


Senior Principal Scientist, Oncology iMed, AstraZeneca, Cambridge, UK


Mark Edbrooke earned his PhD in molecular biology from the University of London. He ran a transnational Gene Interference functional genomics department at GSK that generated target identification and validation data for all therapeutic areas across the company (e.g. oncology, cardiovascular, respiratory, neurology and infectious diseases). He then founded and led a Discovery Performance Unit (DPU) within GSK focused on the development of therapeutic siRNAs and, latterly, on therapeutic antisense oligonucleotides. He recently joined AstraZeneca (AZ) and is involved in AZ’s alliances with Ionis Pharmaceuticals, Regulus Therapeutics, and Moderna Therapeutics.

Michael Famulok, PhD

Professor, Life and Medical Sciences Institute

University of Bonn, Member of the German

National Academy of Sciences


Dr. Famulok is one of the pioneers of the nucleic acid in vitro evolution or SELEX (Systematic Evolution of Ligands by Exponential enrichment) methodology in the 1990s, and has been using this technology on the junction of organic chemistry, biochemistry, molecular biology and medicine ever since. His particular expertise is in the field of aptamers and ribozymes, which he applies for development of novel biosensors, diagnostic tools and therapeutics. Most recently, Dr. Famulok expanded his interest into the field of nucleic acids nanotechnology.


Dr. Famulok received his B.A. (1986) and Ph.D (1989) in Organic Chemistry under supervision of Prof. Gernot Boche from the Philipps University of Marburg in 1986. He then conducted post-doctoral training in Supra-Molecular Chemistry at Massachusetts Institute of Technology with Prof. Julius Rebek Jr. till 1990 and in Molecular Biology at Harvard with Prof. Jack W. Szostak till 1992. Since 1999 Dr. Famulok is a Professor in Life and Medical Sciences Institute of University of Bonn. In 2013 he was elected a Vice President of German Research Council.


For his scientific achievements, Dr. Famulok has received numerous awards and honors, among others the Karl Ziegler Prize of the German Chemical Society (1998), the Klung Wilhelmy Weberbank Award for Chemistry (1998), the Gottfried Wilhelm Leibniz Prize of the German Research Council (2002) and GlaxoSmithKline Award for Outstanding Achievement in Chemical Biology (2008). Dr. Famulok was elected a Member of the North Rhine-Westphalian Academy of Sciences, Humanities and the Arts in 2002 and a Member of the German Academy of Sciences Leopoldina in 2007.


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

Xiang-Dong Fu, PhD

Professor, University of California San Diego, USA


In the early 1990s, Dr. Fu co-discovered and provided initial characterization of SR proteins from mammals, a family of conserved RNA-binding proteins involved in various aspects of mRNA production and function, including pre-mRNA splicing, mRNA export, nonsense-mediated decay and translation. His lab was then the first to identify a family of kinases specific for SR proteins, and demonstrated that these kinases are critical for transducing external and intracellular signals to regulate alternative pre-mRNA splicing in the nucleus. More recently, the research scope of Dr. Fu’s group has been expanded to understanding the mechanistic coupling between transcription and splicing, the nuclear architectural basis for regulated gene expression, and the genomics of RNA binding proteins.


Dr. Fu received his M.S. degree in Virology from Wuhan University, China in 1982, Ph.D. degree in Biochemistry from Case Western Reserve University in 1988 and post-doctoral training with Prof. Tom Maniatis at Harvard from 1988 to 1992. In 1992, Dr. Fu joined the faculty of University of California San Diego, where he currently is a Professor of Cellular and Molecular Medicine at the Institute of Genomic Medicine.


Dr. Fu’s scientific achievements have been recognized by selection for the Searle Scholar Award in 1994 and the Leukemia and Lymphoma Society Scholar Award in 1997. In 2010, he was elected a Fellow of the American Association for the Advancement of Science (AAAS).


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

Gregory J. HANNON, PhD

Professor, Group Leader, Cancer Research UK, University of Cambridge, UK

Member of the US National Academy of Sciences


Dr. Hannon is one of the pioneers in RNA interference (RNAi), the field that received a Nobel Prize in 2006. His group was the first to isolate and characterize the RISC (the RNA-Induced Silencing Complex), the key component of the RNAi machinery. Dr. Hannon’s research was also fundamental for assigning complex and diverse roles for microRNAs, a large group of universal eukaryotic small RNA molecules. In particular, he was among the first to understand the role of miRNAs in cancer. Dr. Hannon’s most recent research expanded into identification and characterization of novel classes of small RNAs, using the next generation sequencing (NGS) techniques and approaches.


Dr. Hannon earned his B.A. in Biochemistry (1986) and PhD in Molecular Biology (1992) from Case Western Reserve University. In 1992, he received a Damon Runyon-Walter Winchell Cancer Research Fund Fellowship and completed his postdoctoral training in the laboratory of Prof. David Beach at Cold Spring Harbor Laboratory (CSHL) in 1994. Dr. Hannon became an Assistant Professor in 1996 and a Pew Scholar in 1997. He is currently a full-time Professor in the Watson School of Biological Sciences at CSHL (since 2002), a Howard Hughes Medical Institute Investigator (since 2005), and a Chair of the Program in Genetics and Bioinformatics (since 2007).


For his scientific achievements, Dr. Hannon received numerous awards and honors, including the Rita Allen Scholarship (2000), the U.S. Army Breast Cancer Research Program Innovator Award (2002), the Faculty of 1000 Title (2003), the American Association for Cancer Research Award for Outstanding Achievement in Cancer Research (2005), the National Academy of Sciences Award in Molecular Biology (2007), and the Memorial Sloan-Kettering Cancer Center Paul Marks Prize for Cancer Research (2007). Since 2012, Dr. Hannon is a member of the U.S. National Academy of Sciences.


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

Mark A. Kay, M.D., PhD

Dennis Farrey Family Professor, Head, Division of Human Gene Therapy, Departments of Pediatrics and Genetics at Stanford University School of Medicine


Professor  Kay received his  B.S. in physical sciences from Michigan State University, a Ph.D. in Developmental Genetics and M.D. from Case Western Reserve University. Afterwards, Dr. Kay pursued additional medical training and triple board certification in Pediatrics, Medical Genetics, and Inborn Errors of Metabolism at the Baylor College of Medicine. He was appointed to the faculty at the University of Washington in 1993 and moved to Stanford University in 1998. 

Professor Kay’s lab made seminal contributions in the field of gene  and nucleic acid based therapeutics. His early work on developing  recombinant adeno-associated viral vectors led to the first clinical trial where such a  vector was administered systemically into humans, and in which he held the original IND.  His lab has made important contributions in the area of miRNA biogenesis, their mechanisms of action, and RNAi therapeutics.  While his lab continues to work in these areas, his group was one of the first to promote the idea that tRNA derived small RNAs (tsRNAs)  were not mere degradation products but rather molecules that play important roles in gene regulation. His lab has shown that one type of tsRNA functions in regulating ribosome biogenesis and is a potential target for treating hepatocellular carcinoma. Professor Kay has published over 250 papers, is currently the deputy editor of Human Gene Therapy, and serves on the editorial boards of other peer-reviewed publications.  

Dr. Kay served on the Board of Directors of the Oligonucleotide Therapeutics Society for 10 years. He was on the founding board of the  of the American Society of Gene and Cell Therapy, and served as its President in 2005-2006. He was the  recipient of the society’s Outstanding Investigator Award in 2013.  Dr. Kay received the E. Mead Johnson Award for Research in Pediatrics in 2000. He was elected to the American Society for Clinical Investigation in 1997 and the Association for American Physicians in 2010.  He has organized many national and international conferences, including the first Gordon Conference dedicated to gene therapy.  Professor Kay has and continues to serve on various commercial and academic boards.  He is a scientific co-founder of Voyager Therapeutics, and a co-founder of LogicBio Therapeutics.

Ekkehard LEBERER, PhD

Professor, Senior Director, Alliance Management, Sanofi, France/Germany


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 (


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.


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


Professor, Senior Investigator at Immune Disease Institute, Harvard Medical School, USA


Judy Lieberman earned a Ph.D. in physics from Rockefeller University, worked as a theoretical physicist at the Institute for Advanced Study in Princeton and Fermilab, received an M.D. from Harvard and MIT, and trained in hematology-oncology at Tufts Medical Center and as a postdoctoral fellow at the Center for Cancer Research at MIT. She worked as a hematologist-oncologist at Tufts before moving to Harvard Medical School in 1995. At Harvard Medical School she served as Director of the Division of AIDS from 2005 to 2009 and is currently the Chair of the Executive Committee on Immunology. 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 were the first to demonstrate that siRNAs could protect mice from disease. They developed methods to harness RNAi to inhibit herpes and HIV transmission in animal models and are developing strategies for cell-targeted RNAi to treat viral infection, immune disease and cancer. They also study the role of microRNAs in cancer. The Lieberman laboratory also studies how killer lymphocytes protect us against infection and cancer.

David M.J. LILLEY, PhD

Professor, Director CRUK Nucleic Acids Structure Research Group, University of Dundee, UK

Fellow of the UK Royal Society


Professor David Lilley is investigating how cells repair their DNA when it gets damaged. DNA repair is crucial for preventing genetic faults that can lead to cancer. Professor Lilley leads a world-class group of scientists at the University of Dundee, and their research into the processes at the heart of cancer will pave the way for new approaches to treating the disease. Cancer starts when a cell’s DNA – its genetic ‘instruction manual’ – becomes faulty. This can happen when cells are making copies of themselves, or when the DNA is damaged by things around us, such as chemicals in cigarette smoke or UV light from the sun. Luckily, cells are often able to repair this damage.


Professor Lilley’s team is studying the shapes of protein molecules that help to repair damaged DNA. They are using sophisticated lab techniques, such as taking X-ray images of proteins – a technique known as X-ray crystallography – as well as using powerful microscopes.


For his scientific achievements, Dr. Lilley has received numerous awards and honors, among others the Gold Medal of G. Mendel (Czech Academy of Sciences, 1994), the Prelog Medal in Stereochemistry (Zurich, Switzerland, 1996), RNA and Ribozyme Chemistry Award (Royal Society of Chemistry, 2002), Interdisciplinary Award (Royal Society of Chemistry, 2006). He was also elected aMember of the European Molecular Biology Organization in 1984, Fellow of the Royal Society of Edinburgh in 1988 and Fellow of the Royal Society in 2002.


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

Dong-ki LEE, PhD

Professor, Sungkyunkwan University, South Korea

Chief Executive Officer, OliX Pharmaceuticals


Prof. Dong-ki Lee received B. S. in Chemistry from Korea Advanced Institute of Science and Technology (KAIST) in 1993, and Ph. D. in Biochemistry from Cornell University in 1999. After post-doctoral training in Pohang University of Science and Technology (POSTECH), Toolgen Inc., and KAIST, he joined POSTECH as an assistant professor in 2004. In 2008, He moved to Sungkyunkwan University and is a full professor of Chemistry since 2012. In 2008, Prof. Lee was selected as the principal investigator of the Global Research Laboratory program, funded by Korean government, to develop novel RNAi medicine. He is currently serving as the Asian editor of “Nucleic Acid Therapeutics” and a editorial board member of “Molecular Therapy: Nucleic Acids”. His work on novel siRNA structures, nucleic acid aptamers, and eukaryotic gene regulation has been published as over 70 papers including prestigious journals such as Nature, Cell, PNAS, and Molecular Therapy. In 2010, he founded OliX Pharmaceuticals, a RNAi therapeutics company focusing on topically administrable diseases, and serves as Chief Executive Officer.


Senior Vice President, Drug Discovery

Alnylam Pharmaceuticals, USA

Director, Oligonucleotide Therapeutics Society


Muthiah Manoharan earned his PhD in Chemistry at the University of North Carolina, Chapel Hill. He did post-doctoral research on DNA repair enzymes at Yale University and the University of Maryland with Prof. John Gerlt. He has been an oligonucleotide chemist since 1983 and became involved in oligonucleotide-based therapeutics at Lifecodes (Valhalla, New York), during the early days of antisense research. He moved to Isis Pharmaceuticals in 1990, and contributed to company’s growth through discovery of numerous chemical modifications and development of conjugation chemistry to enable oligonucleotide delivery. Since his move to Alnylam in 2003, Dr. Manoharan worked to make siRNAs therapeutically viable using chemical modifications, siRNA conjugates, cationic lipids and other delivery systems. His research has embraced all major areas of oligonucleotide therapeutics, including antisense, RNAi, antagomirs, immunostimulatory oligonucleotides and pre-mRNA splicing modulation. Dr. Manoharan authored on over 180 publications and 160 issued US patents. In 2007 he was awarded American Chemical Society M. L. Wolfrom Award for contributions to Carbohydrate Chemistry.

Henrik Ørum, PhD

Head of RNA Therapeutics Research, Roche, Denmark


Experience Summary:


PhD in molecular Biology. 60+ peer reviewed publications and books; countless scientific and business presentations at international conferences and symposiums
– Founder of 2 biotech companies; Exiqon A/S – a diagnostic company listed on the Copenhagen Stock Exchange (EXQ), and Santaris Pharma A/S- a privately held biotherapeutic company headquatered in Denmark.
– More than 15 years as a senior biotech executive with extensive experience in every aspect of building, leading and managing succesfull companies.
– Hands-on experience in establishing, managing and motivating innovative R & D organisations in the fields of in-vitro diagnostics (PNA Diagnostics, Boehringer Mannheim & Hoffmann la Roche) and human therapeutics (Cureon & Santaris Pharma).
– Led the development of strong technology and IP platforms and instrumental in leveraging them into broad product pipelines.
– Chaired joint steering committees in charge of major corporate partnerships.
– Hands on experience in establishing and leading high-performance business development departments (Cureon & Santaris Pharma), responsible for opportunity identification and evaluation, negotiating in/out-licensing deals, market & competetive analyses, strategic planning and corporate profiling.
– Led negotiations and closed multiple deals with a compounded value in excess of 2.5B $ with big pharmaceutical and biotech companies.
– Raised more than 100M $ from venture capital and institutional investors in multiple rounds of financing.
– Broad experience in creating shareholder value through spin-out, merger, and trade sale.


Dr. Ørum received his MS in molecular biology from the Technical University of Copenhagen in 1987, and PhD in molecular biology from the Medical University of Copenhagen in 1993. He completed his post doctoral training at the Copenhagen Royal School of Pharmacy in 1993. Dr. Ørum is a co-founder of Cureon A/S, which merged with Pantheco A/S to form Santaris Pharma. He was previously Director R&D and CSO at PNA Diagnostics A/S with responsibilities for the overall scientific and business strategy and intellectual property rights of the company.


Dr. Ørum has extensive operational and strategic experience in all aspects of starting, developing, and managing biotech companies. He is the author and co-author of more than 40 scientific publications and books and has been issued out several patents. He is often invited to speak at scientific and biotech conferences and symposia.


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


Professor, Massachusetts Institute of Technology, Howard Hughes Medical Institute, USA


Dr. Regev is interested in biological networks, gene regulation and evolution. Her work focuses on dissecting complex molecular networks to determine how they function and evolve in the face of genetic and environmental changes, in differentiation, evolution and disease.


Dr. Regev received her M.S. from Tel Aviv University, studying biology, computer science, and mathematics in the Interdisciplinary Program for the Fostering of Excellence. In this program, she did research in both theoretical biology (on the evolution of development) and experimental biology (on genomic instability). Dr. Regev completed her Ph.D. at Tel Aviv University, under the supervision of Eva Jablonka and Ehud Shapiro. She currently is an associate professor in the Department of Biology at MIT and director of the Klarman Cell Observatory at the Broad. In 2009, Dr. Regev was named an Early Career Scientist of the Howard Hughes Medical Institute.


For her scientific achievements, Dr. Regev has received Overton Prize (International Society for Computational Biology, 2008), Earl and Thressa Stadtman Scholar Award, the NIH Director’s Pioneer Award, and a Sloan fellowship from the Sloan Foundation.


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

Nikolaus RAJEWSKY, PhD

Professor, Max Delbrück Center for Molecular Medicine, Germany


Nikolaus Rajewsky earned his Ph.D. in Theoretical Physics at the University of Cologne, Germany (1997). After a postdoctoral period at Rutgers University and at Rockefeller University, he was an “Assistant Professor for Biology and Mathematics” in the Department of Biology at New York University (2003-2006). In 2006 he became a full Professor at the Charite and the Max Delbruck Center for Molecular Medicine in the Helmholtz Association in Berlin, Germany. He conceived and directs the “Berlin Institute for Medical Systems Biology” (BIMSB). BIMSB is a new branch of the MDC and will grow to a size of ~25 PIs in downtown Berlin.


Nikolaus Rajewsky uses both computational and experimental molecular biology methods to study gene regulation in animals. A focus of his work is on the function and mechanisms of small non-coding RNAs and RNA binding proteins in development and stem cells. Nikolaus’ research has been cited over 13,000 times and has been featured in numerous high-ranking scientific journals and in the press. His latest awards include: IUBMB medal, FEBS award, “Berliner Wissenschaftspreis”, EMBO membership, and in 2012 the highest award for research in Germany, the “Leibniz Preis” of the DFG. Since 2008 he is also a “Global Distinguished Professor of Biology at New York University” and in 2014 has received on honorary PhD in Human Biology and Medical Genetics by Sapienza University of Rome, Italy.

Cristina M. RONDINONE, PhD

Vice President, Head of Innovative Medicines

Center of Excellence

AstraZeneca/MedImmune, USA


Dr. Rondinone studied in Universidad de Buenos Aires from 1983 to 1989, and during this period received her B.S., M.S. and Ph.D. in Biochemistry and Molecular Biology.


Working experiences:


VP and Head of Cardiovascular and Metabolic Diseases Innovative Medicines
March 2013 – Present (9 months)


Vice President R&D, Head CV/Metabolic Diseases
March 2011 – Present (2 years 9 months)


Senior Director- Head Dept Metabolic Diseases
Roche Pharmaceuticals
April 2010 – March 2011 (1 year)


Dept Head Metabolic Diseases
Hoffmann-La Roche Inc
2009 – March 2011 (2 years)


Research Director
Roche Pharmaceuticals
April 2005 – April 2010 (5 years 1 month)


Group Leader Metabolic Diseases
1999 – 2005 (6 years)


Associate Professor, Docent in Molecular Medicine, Dept of Internal Medicine
Göteborg University
1995 – 1998 (3 years)


Visiting Fellow
April 1992 – June 1995 (3 years 3 months)


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


Vice President, Alnylam Pharmaceuticals, USA


Dr. Sepp-Lorenzino received degrees in Pharmacy and Biochemistry from the University of Buenos Aires in 1985, and Ph.D. from New York University in 1990. Then She completed her post doctoral training at Memorial Sloan-Kettering.


Vice President, Entrepreneur in Residence
Alnylam Pharmaceuticals
April 2014 – Present (8 months)


Executive Director / Senior Director, RNA Therapeutics Discovery Biology, Department Head
August 2011 – March 2014 (2 years 8 months) | West Point, PA
Responsible for biology strategy and activities towards identification/optimization of siRNAs and delivery vehicles, advancement of siRNA candidates, and utilization of siRNA tools for in vitro and in vivo target/biomarker validation and de-risking across functions and disease areas.
Consolidated operations from Sirna San Francisco into single integrated Biology department at PA site (site closure, asset, personnel and technology transfer).
Member of RNA Therapeutics Core Leadership Team, West Point Site Core Leadership Team, and RNA Review and Licensing Committee.


Senior Director, RNA Therapeutics Delivery Biology, Department Head
January 2007 – August 2011 (4 years 8 months) | West Point, PA
As member of the RNA Therapeutics Leadership team, designed, enabled and implemented area strategy, working effectively across organizational and geographic boundaries.
Led department responsible for identification and development of safe and efficacious oligonucleotide delivery formulations.
Oversaw project teams for three oligonucleotide delivery modalities: lipid nanoparticles (LNP), polymer conjugates and siRNA conjugates, as well as exploratory targeted delivery efforts.
Led an innovative team of biochemists and pharmacologists employing cutting-edge biochemical, cell and in vivo technologies to advance mechanistic understanding of oligonucleotide delivery at the organismal, cellular and subcellular levels. Carried out investigative toxicology studies on delivery formulations and established predictive assays and protocols that enabled >100-fold widening of safety margins.
Identified Lipid Nanoparticle formulations with potency and safety profile compatible with clinical development. Advanced siRNA therapeutic programs through internal R+D stage gates in collaboration with disease areas and development functions. Contributed to regulatory documents.
Evaluated over 500 licensing opportunities; established numerous collaborations and partnerships to advance internal capabilities.


Director, Vaccines and Biologics Research
January 2006 – January 2007 (1 year 1 month) | West Point, PA
Pioneered the use of siRNA technology for in vivo studies and therapeutic applications.
Played a significant role in establishing a strategic partnership with Alnylam Therapeutics and in the acquisition of Sirna Therapeutics.
Member of Vaccines and Biologics Leadership Team, and Technology Review and Licensing Committee.


Director / Senior Research Fellow, Cancer Research, Department Head
November 2003 – December 2005 (2 years 2 months) | West Point, PA
Responsible for cell cycle, checkpoint and cell survival Oncology target pipeline.
Advanced three small molecule programs, from target validation through clinical development (Phase I/II).
Member of Site Leadership Team, Franchise Operating Committee and Oncology Review and Licensing Committee.
Participated in establishing and executing on licensing strategy; performed multiple licensing evaluations and due-diligence; in-licensed Vertex VX-680 (member of Joint Steering Committee) and Pierre Fabre IGF-I receptor monoclonal antibody.
Evaluated and implemented enabling technologies: established high-throughput, high-content imaging facility, and performed tumor responder and drug combination in vitro assays for established and pipeline drugs.
Partnered with Biologics Research to identify tumor antigens for targeted delivery.


Research Fellow and Project Team Lead, Cancer Research
December 1999 – October 2003 (3 years 11 months) | West Point, PA
Led interdisciplinary team for small molecule tyrosine kinase inhibitor of KDR and Flt-3. Successfully advanced program through all drug discovery and development stages, from target validation, lead optimization, biomarker development, in vivo pharmacology. Authored biology section of IND; supported phase I trials by developing and performing bone marrow target engagement biomarker assays.
Designed new tools and protocols for anti-angiogenesis drug discovery, including development, validation and qualification of patient enrichment and response biomarkers. First Oncology program at Merck to incorporate patient expression profiling – early adopter of new technology (Rosetta Inpharmatics) – and dynamic contrast enhanced MRI.


Assistant Lab Member, Assistant Attending Molecular Biologist
Memorial Sloan-Kettering Cancer Center
January 1995 – December 1999 (5 years)|New York, NY
Translational cancer programs and research collaborations with several pharmaceutical companies. Investigated mechanism of action of inhibitors of Hsp90, farnesyl transferases, receptor and cytoplasmic tyrosine kinase, and of several fully synthetic natural products in collaboration with Professors Neal Rosen and Sam Danishefsky. Contributed to advancing 17-allyl-amino-geldanamycin Hsp90 inhibitor program to the clinic and contributed to the identification and characterization of next generation Hsp90 inhibitors. Explored targeted delivery of Hsp90 inhibitors to prostate cancer using small molecule and monoclonal antibody conjugates.


Additional professional info about Dr. Sepp-Lorenzino can be found in the following sites:

Robert H. SINGER, PhD

Professor and Co-Chair

Albert Einstein College of Medicine, USA

Member of the US National Academy of Sciences


Rob Singer’s lab seeks to understand the expression and movement of mRNA. His lab developed methods to label RNA endogenously in cell lines and mice. He develops microscopy to observe and quantify single mRNAs in neuronal processes, and can follow the mRNA from transcription through nuclear export, translation and degradation.


Dr. Singer received his B.S. in physical chemistry from Oberlin College, andPh.D. in developmental biology from Brandeis University. He received his postdoctoral training at the Massachusetts Institute of Technology and at the Weizmann Institute of Science in Rehovot, Israel. Currently Dr. Singer is a senior fellow at the Janelia Farm Research Campus. In addition, he also holds severalpositions at the Albert Einstein College of Medicine: Professor and Cochair, Department of Anatomy and Structural Biology, Professor in the Dominick P. Purpura Department of Neuroscience and in Department of Cell Biology, and Codirector of the Gruss-Lipper Biophotonics Center.


For his scientific contributions and achievements, Dr Singer has received numerous awards and honors, including the Massachusetts Center for Excellence Award (Governor Dukakis), the First Annual Faculty Research Achievement Award (Albert Einstein College of Medicine), and the Marshall S. Horwitz Faculty Prize for Research Excellence (Albert Einstein College of Medicine). Dr. Singer is a Member of the National Academy of Sciences and the American Academy of Arts and Sciences.


Additional professional info about Dr. Singer can be found in the following sites:


Chief Scientific Officer

Silence Therapeutics, Germany/UK


Dmitry Samarsky earned his Ph.D. in biochemistry and molecular biology from the University of Massachusetts Amherst in 1998, and completed his post-doctoral training as the H. Arthur Smith Fellow for Cancer Research at Dr. Michael Green’s laboratory, University of Massachusetts Medical School. From 2001 to 2007 Dr. Samarsky was developing oligonucleotide-based technologies at Sequitur / Invitrogen and Dharmacon / ThermoFisher. He then carried responsibilities for technology and business development at RXi Pharma (US), RiboBio (China) and OliX Pharma (Korea), biopharmaceutical companies developing oligonucleotide technologies and therapeutics. In 2016 he has been appointed a Chief Scientific Officer at Silence Therapeutics (Germany/UK). Dr. Samarsky authored dozens of peer-reviewed papers and US patents, and during the past ten years served as advisor, session chair and presenter at more than hundred international scientific conferences.

Harold P.Swerdlow, PhD

Vice President of Sequencing, the New York Genome Center, USA


Dr. Swerdlow is a biotechnology expert with extensive experience in the field. Prior to joining NYGC, he served as Head of Research & Development at the Wellcome Trust Sanger Institute, where he was responsible for next- generation DNA sequencing in addition to managing the Institute’s Research and Development. Prior to that, Dr. Swerdlow was the Chief Technology Officer at Dolomite Ltd. and the Senior Director of Research at Solexa Ltd.


Dr. Swerdlow received his PhD at the University of Utah, developing capillary electrophoresis for DNA sequencing. He went on to join the Faculty in the Human Genetics department at the University, managing various molecular biotechnology projects related to genomics.


At the Karolinska Institute in Sweden, he was the Unit Coordinator of the Genomics Technologies Unit and Director of the Microarray Core Facility in the newly formed Center for Genomics Research. His first role in the UK was as Senior Director of Research at Solexa Ltd. (now Illumina, Inc.), responsible for developing the most widely used technology for next-generation DNA sequencing. Before joining the Sanger Institute, Harold was the Chief Technology Officer (CTO) at Dolomite Ltd., a microfabrication and microfluidics consultancy.


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

René Thürmer, PhD

Deputy Head Unit Pharmaceutical Biotechnology, BfArM – Federal Institute for Drug & Medical Devices, Germany


Dr. René Thürmer received his diploma in chemistry and his Ph.D. in biochemistry from the University of Tübingen. He joined the BfArM (Federal Institute for Drugs and Medical Devices, Bonn, Germany) in 2000. He currently serves as a CMC reviewer and is Deputy Head of the Unit Pharmaceutical Biotechnology.


His experience is in the field of formulation, manufacture and control of medicinal products, in particular in the field of peptides, proteins, liposomes, sustained release polymer drug products, depot formulations, polymer-conjugated drug products, natural and synthetic surfactants, nanomedicine and others. His special focus lies on oligonucleotide preparations.


Chairman of the Board and Chief Scientific Officer, Asuragen, USA


Dr. Winkler received his B.S. in Genetics from Genetics University of California at Berkeley in 1974, and Ph.D. in Zoology from University of California, Berkeley in 1979. Currently he is Chairman and Chief Scientific Office at Asuragen. Before that, he founded Ambion in 1989, and worked as its CEO until 2006. He was alsoan Adjunct Associate Professor at the Department of Zoology in University of Texas for more than 19 years, since 1991.


Dr. Winkler founded Ambion in 1988, grew it to M in revenue and sold the research products division in 2005 to Applied Biosystems. He is the founder of Asuragen. Prior to founding Ambion he was an Associate Professor of Zoology at the University of Texas at Austin.


Dr. Winkler received the Ernst and Young Entrepreneur of the Year Austin Winner and National Finalist (2002). He is a member of Scientific and Prevention Advisory Council for the Cancer Prevention & Research Institute of Texas.


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


Professor, Peking University School of

Pharmaceutical Sciences, China

Member of the Chinese Academy of Sciences


Dr. Zhang graduated from the Department of Pharmacy of Beijing Medical College in 1958, and from the Graduate School of Beijing Medical College in 1967, majored in Medicinal Chemistry. Currently, Dr. Zhang is the president of Academic Committee of State Key Laboratory of Natural & Biomimetic Drugs, Peking University and he is also a professor of Medicinal Chemistry, Peking University.


For his scientific achievements, Dr. Zhang received numerous awards and honors, among others the Nagai-Hisamitsu Outstanding Science Award (Asia Pharmaceutical Association,2009), the Natural Science Award, Second Prize (Ministry of Science and Technology, China, 2004), the Science and Technology Award, Second Prize (Ministry of Education, China, 2002), the Millennium Pharmaceutical Scientist Award (FIP, San Francisco, USA, 2000). Dr. Zhang used to be Titular Member of IUPAC, Division III, Biomolecular Chemistry Committee and Fellow of Royal Society of Chemistry.

Nucleic acids science plays a key role in precision medicine, genetic therapy and new solutions for human health. Continuous discovery of new RNA functions and constant emergence of innovative nucleic acids technologies are driving forces for the field and hold enormous promise for the future scientific and technological progress. The Canton Nucleic Acids Forum (CNAF) led by Nobel Prize winners is an international forum at the forefront of these developments, aims to push forward communications and collaborations in and abroad, and has been successfully held seven times since 2013. The CNAF has attracted high-profile speakers, including Nobel Prize winners, to highlight recent advances in nucleic-acid based medicine, scientific discoveries, diagnostics and industrial trends. It has been widely recognized as the premier forum in Asia for advancing nucleic acids research and drug development. The 2021 CNAF will feature 20+ globally prominent experts discussing the latest advances in nucleic acids research and development.

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