Housheng Hansen HE, PhD
Associate Professor, Department of Medical
Biophysics, University of Toronto, Canada
Senior Scientist, Princess Margaret Cancer Centre,
University Health Network, Canada
Dr. He received his B.S. degree in physics from Beijing Normal University in the year of 2003, and then transitioned to the genomics and noncoding RNA fields for his Ph.D training in the Institute of Biophysics, Chinese Academy of Sciences. He received his postdoctoral training in cancer genomics and epigenomics at Dana-Farber Cancer Institute. After completing his postdoctoral training in 2011, Dr. He worked as an instructor at Harvard Medical School and was recruited as a scientist at the Princess Margaret Cancer Centre in 2013.
The He laboratory applies a variety of genomic, epigenomic experimental and computational approaches to elucidate the functional role of epigenetic regulation in cancer development, progression and drug response/resistance, with a special focus on the interplay between epigenetic regulator and noncoding RNA under stress conditions such as hypoxia. Dr. He’s work in the fields of noncoding RNA and epigenetics has resulted in ~50 peer-reviewed publications in high-impact journals including Nature, Cell, Nature Genetics, Nature Methods, Cancer Cell and Genome Research.
Institute for Genetic Medicine
Hokkaido University, Japan
Chandrasekhar KANDURI, PhD
Professor in RNA Biology
Department of Medical Biochemistry and Cell Biology
University of Gothenburg, Sweden
Dr. Chandra Kanduri received his PhD from Banaras Hindu University, India, in 1997. For postdoctoral training, he moved to Prof Rolf Ohlsson’s lab at Uppsala University, Sweden, where he explored the functional role of chromatin insulator protein CTCF in genomic imprinting using H19 and IGF2 imprinted cluster as a model system. He later continued his work on long noncoding RNA Kcnq1ot1 and genomic imprinting as an independent investigator. His work, as an early career scientist, contributed significantly towards understanding the functional interaction between RNA and chromatin. His further work on RNA-chromatin connection, revealed RNA molecules enriched in the active and inactive chromatin regions, and also possible mechanisms by which chromatin enriched lncRNAs targeted across the genome.
He became full professor of RNA biology at Uppsala university in 2010. In 2012, he moved his research activities to University of Gothenburg. His current research primarily focused on developing novel long noncoding RNAs (lncRNA) based strategies for the treatment of drug resistant and difficult to treat cancers through exploiting cell cycle based functional screens.
Functional and mechanistic roles of long noncoding RNAs (lncRNAs) in cancer development and progression
Despite rapid improvements in unveiling the importance of long noncoding RNAs (lncRNA) in all aspects of cancer biology, there is still a lot of void in how lncRNAs mechanistically influence various biological processes associated with cancer development. Towards this, by using RNA-seq data from cell cycle-based high-throughput functional screens and different neuroblastoma (NB) prognostic sub-groups, we have identified several cancer-relevant, therapeutically applicable lncRNAs. Functional investigations of these lncRNAs revealed their nexus with oncogenic and tumor-suppressor pathways. I will discuss how these cancer relevant lncRNAs can be used as potential therapeutic targets in different cancer types.
Ailong KE, PhD
Professor, Department of Molecular Biology and Genetics, Cornell University, US
Spacer acquisition mechanism in type II-A CRISPR system.
Molecular memory is created when a short foreign DNA-derived prespacer is integrated
into the CRISPR array as a new spacer. Whereas the RNA-guided CRISPR interference
mechanism varies widely among CRISPR-Cas systems, the spacer integration
mechanism is essentially identical. The conserved Cas1 and Cas2 proteins form an
integrase complex consisting of two distal Cas1 dimers bridged by a Cas2 dimer. The
prespacer is bound by Cas1-Cas2 as a dual-forked DNA, and the terminal 3′-OH of each
3′ overhang serves as an attacking nucleophile during integration. The prespacer is
preferentially integrated into the leader-proximal region of the CRISPR array, guided by
the leader sequence and a pair of inverted repeats inside the CRISPR repeat. Spacer
integration in the well-studied Escherichia coli type I-E CRISPR system also relies on the
bacterial integration host factor. In type II-A CRISPR, however, Cas1-Cas2 alone
integrates spacers efficiently in vitro; other Cas proteins (such as Cas9 and Csn2) have
accessory roles in the biogenesis phase of prespacers. I will present our structural and
biochemical efforts in revealing the spacer acquisition mechanism in the Enterococcus
faecalis type II-A CRISPR system.
Ekkehard LEBERER, PhD
Professor, Senior Director, Sanofi, Germany
Founding Director, COMPACT Consortium
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.
Delivering Therapeutic Oligonucleotides across Biobarriers: Opportunities and Challenges in Drug Development
Dong-ki LEE, PhD
Professor of Chemistry, Sungkyunkwan University, Korea
Founder and CEO, OliX Pharmaceuticals, Korea
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 80 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. OliX successfully went public in July 2018, with 370M USD valuation.
Asymmetric siRNA Targeting Fibrotic and Ocular Disorders
OLX10010, an anti-fibrotic cell-penetrating asymmetric siRNA (cp-asiRNA) targeting connective tissue growth factor (CTGF), effectively reduces target gene expression as well as expression of fibrotic markers in animal model study. Preclinical as well as clinical study update of OLX10010 in anti-skin scar will be presented. In addition to skin scar, OLX10010 has a potential to be developed as therapeutics targeting various fibrotic disorders. We will present preclinical study data of OLX10010 in other fibrotic diseases in lung and eye, such as idiopathic pulmonary fibrosis (IPF) and subretinal fibrosis.
Wei LI, PhD
Professor of Bioinformatics, Dan L. Duncan Cancer Center
Department of Molecular and Cellular Biology
Baylor College of Medicine, USA
Zhen LI, PhD
Zhen Li, PhD
Senior Vice President, Chemistry and Non-Clinical Development
Arrowhead Pharmaceuticals, USA
Dr. Zhen Li is Senior Vice President, Chemistry and Non-Clinical Development at Arrowhead Pharmaceuticals. She leads discovery chemistry, biology and non-clinical development at Arrowhead. She has led the discovery and development of Arrowhead TRiMTM platform for RNAi based therapeutics for hepatic and non-hepatic targets, and she is the key inventor of the platform. Prior to joining Arrowhead in 2014, she held leadership positions at Merck, Schering-Plough and Novartis, and led teams in drug discovery and process development in small molecule pharmaceuticals as well as RNAi therapeutics covering a range of disease areas. She has a demonstrated track record in leading multiple drug development programs from early to late stage. Dr. Zhen Li received her Bachelor of Science degree from Peking University and her Ph.D. from Harvard University.
TRiMTM Platform Based RNAi Therapeutics in Treating HBV and Cardiometabolic Diseases
One of the more important recent advances in biology, RNA interference is a natural cellular process where short oligonucleotides can be used to silence gene expression and regulate the production of proteins. Arrowhead’s RNAi-based therapeutics leverage this natural pathway to shut down specific genes that cause disease.
The presentation will focus on Arrowhead’s clinical candidate ARO-HBV for the treatment of Hepatitis B Virus infection. HBV infection presents a huge unmet medical need, as it affects over 250 million people worldwide and currently there is no cure for this devastating disease. In the presentation, some of our earliest clinical data on ARO-HBV will be presented, and the journey in discovering this powerful drug candidate will be discussed. The presentation will also discuss Arrowhead’s preclinical candidates ARO-ANG3 and ARO-APOC3 for the treatment of cardiometabolic disease such as hypertriglyceridemia. Gene knockdown, reduction of protein levels, and reduction of triglycerides in disease models for Arrowhead’s preclinical candidates ARO-ANG3 and ARO-APOC3 will be presented.
Muthiah MANOHARAN, PhD
Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, USA
Board Director, Oligonucleotide Therapeutics Society
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.
Craig MELLO, PhD
Professor, University of Massachusetts
Howard Hughes Medical Institute, USA
Nobel Prize in Physiology or Medicine (2006)
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.
RNA mediated inheritance
Joseph (Jody) PUGLISI, PhD
Professor, Department of Structural Biology
Stanford University School of Medicine, USA
Member of the US National Academy of Sciences
How mRNA controls the dynamics of translation
Elisabetta Viani PUGLISI, PhD
Senior Research Scientist Department of Structural Biology,
Stanford University School of Medicine, USA
Architecture of the HIV-1 reverse transcriptase initiation complex
Laura SEPP-LORENZINO, PhD
Vice President, Head of Nucleic Acid Therapies,
Vertex Pharmaceuticals, USA
Mikiko C. SIOMI, PhD
Professor, Department of Biological Sciences,
Graduate School of Science, The University of Tokyo, Japan
piRNA biogenesis and functions
Andreas STRASSER, PhD
Division Head, Molecular Genetics of Cancer,
The Walter and Eliza Hall Institute of Medical Research, Australia
Bruce SULLENGER, PhD
Professor, Duke University Medical Center, USA
Dr. Sullenger’s research program focuses upon the discovery and development of therapeutic RNAs. His group has been a leader in the development of nucleic acid aptamers for inhibiting the activities of therapeutically important proteins including coagulation factors and platelet proteins. He is on the Board of Directors of the American Society of Gene and Cell Therapy and the Oligonucleotide Therapeutics Society and is Editor-in-Chief for the Society’s journal Nucleic Acid Therapeutics. Dr. Sullenger founded Regado Biosciences Inc, a company focused upon the development and commercialization of the aptamer-antidote technology his laboratory invented. The lead compound, an aptamer targeting factor IXa and its matched antidote, is in phase 3 clinical studies.
Dr. Sullenger earned his Bachelor of Science Degree from Indiana University (Bloomington, IN) and his Ph.D. from Cornell University Graduate School of Medical Sciences (New York, NY) working at the Memorial Sloan-Kettering Cancer Center. He performed postdoctoral studies at the University of Colorado (Boulder, CO) in the Department of Biochemistry in Dr. Thomas Cech’s laboratory.
Andrew YOO, PhD
Associate Professor, Department of Developmental Biology,
Washington University School of Medicine, USA
MicroRNAs as cell fate controllers and cellular reprogramming factors