Mark EDBROOKE, PhD<\/strong><\/span><\/h4>\nSenior Principal Scientist, Oncology iMed, AstraZeneca, Cambridge, UK<\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]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\u2019s alliances with Ionis Pharmaceuticals, Regulus Therapeutics, and Moderna Therapeutics.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]<\/p>\n
Therapeutic nucleic acids including antisense oligonucleotides \u2013 significant progress towards a viable drug platform for tackling intractable targets in oncology.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]Recent human clinical data has demonstrated potent activity of systemically-administered, unformulated, antisense oligonucleotides (ASOs) when targeted to liver expressed genes. However, robust activity in extra-hepatic tissues including tumours has been limited with initial ASO chemistries. In contrast, high affinity next generation ASOs have been successfully used to target transcription factors and other intracellular proteins in multiple preclinical cancer models, leading to the translation of these drugs into the clinic. Our experience to date with next generation ASO technology suggest this platform is poised to become a key therapeutic modality to modulate a range of tumour targets intractable to small molecules or antibodies.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Jan-GORODKIN-PhD.jpg” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Jan GORODKIN, PhD<\/strong><\/span><\/h4>\nProfessor, University of Copenhagen
Director, Center for Non-Coding RNA in
Technology and Health, Denmark<\/i><\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]Jan Gorodkin holds a MSc in physics from the Niels Bohr Institute and obtained his Ph.D. in Bioinformatics from Center for Biological Sequence analysis at the Technical University, Denmark. He did his post docs at Aarhus University, Denmark and Washington University Medical School, St. Louis. He took up positions at the Royal Veterinary and Agricultural University (now University of Copenhagen), and is now professor and head of the bioinformatics group as well as director of Center for non-coding RNA in Technology and Health in the Department of Veterinary Clinical and Animal Sciences. His research interests span from RNA bioinformatics to animal and human genome analysis and his research group has been involved in developing numerous computational tools as well as applying them on genomic and transcription data.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]Computational analysis of RNA structure and interactions in genomic sequence<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]A key challenge in computational RNA biology is to search for conserved RNA (secondary) structures, as the conservation of base pairs not necessarily implies conservation in sequence. We have addressed this through a RNA structural alignment strategy followed by a CaptureSeq strategy and structure probing on selected candidates. We also address another key RNA bioinformatics challenge for genome\/transcriptome-wide searching of RNA-RNA interactions which is computational expensive. We employ a suffix-array implementation along with a simplified energy model and reach a substantial increase in speed. We, furthermore, use this in an implementation of a siRNA off-target pipeline for which we find inverse relationships between our off-target measure and inhibition efficiencies of siRNAs.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Chuan-HE-PhD.jpg” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Chuan HE, PhD<\/strong><\/span><\/h4>\nProfessor, University of Chicago, USA<\/i><\/em><\/p>\nHoward Hughes Medical Institute<\/i><\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
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\u2019s 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.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]New sequencing technology to map DNA epigenetic modifications<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]<\/p>\n
\n
Cytosine methylation (5mC) is a well-established epigenetic mechanism essential for genomic imprinting, X chromosome inactivation, silencing of retrotransposons, and lineage-specific expression of many developmental regulatory genes. This epigenetic mark is installed and maintained by DNA methyltransfeases (DNMTs), and has been recently shown to be oxidized to 5-hydroxymethylcytosine (5hmC), 5-formylcytosine (5fC) and 5-carboxylcytosine (5caC) by the Ten-eleven- translocation (TET) family of protein dioxygenases. In humans and mice, 5hmC is found in most cell types and tissues, with the abundance ranging from less than 0.1% to 0.4% of all cytosines. By contrast, the abundance of 5fC and 5caC is very low to non-detectable. We have developed several methods that allow selective detection and sequencing of 5hmC, 5fC, and 5caC with limited genomic materials. Our results indicate genome-wide dynamic methylation\/demethylation play critical roles in mammalian gene expression regulation. The presence of 5hmC in promoters, gene bodies, and enhancers of specific loci often associates with gene activation and reflects chromatin state changes. Sensitive detection of 5hmC will therefore offer the best means to interrogate and monitor cell transformation and gene expression changes in future disease diagnosis and prognosis.<\/p>\n<\/div>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Robert-Holt-PhD.png” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Robert HOLT, PhD<\/strong><\/span><\/h4>\nProfessor, University of British Columbia, Canada<\/i><\/em><\/p>\nProfessor, Simon Fraser University, Canada<\/i><\/em><\/p>\nScientist, British Columbia Cancer Agency, Canada<\/i><\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
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.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]Towards personalized T cell receptor therapeutics: Interrogating the T cell repertoire<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]The loci encoding adaptive immune receptors (eg. T cell receptors) have highly unique properties. Stochastic recombination events at these loci during lymphocyte development generate diverse and remarkably personalized immune repertoires that serve to protect self from non-self. With next-generation sequencing methods it is now possible to interrogate immune repertoires at clonotype resolution and obtain a comprehensive view of the adaptive response to immune challenge, including the anti-cancer immune response. However, linking T cell receptors to the specific antigens they recognize remains difficult, and new high-throughput methods will be required to define T cell targets and exploit the full potential of engineered T cell therapeutics.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Jin-Soo-KIM-PhD.jpg” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Jin-Soo KIM, PhD<\/strong><\/span><\/h4>\nProfessor, Seoul National University
Director ,Center for Genome Engineering,
Institute for Basic Science and Department of Chemistry, South Korea<\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
– Scientific interests and expertise
\nGenome engineering via programmable nucleases including the CRISPR-Cas system
\n1) Genome editing in plants, animals, and cultured human cells including iPS\/ES cells
\n2) Engineered nuclease-mediated gene and cell therapy
\n3) Functional genomics using genome-scale libraries of programmable nucleases<\/p>\n
– Received degrees (including places and years)
\n1987, BS, Dept. of Chemistry, Seoul National University
\n1989, MS, Dept. of Chemistry, Seoul National University
\n1994, PhD, Dept. of Biochemistry, University of Wisconsin-Madison<\/p>\n
– Current position(s) and role(s) in affiliated organizations
\n1994-1997, Research Associate, Howard Hughes Medical Institute\/MIT
\n1997-1999, Principal Investigator, Samsung Biomedical Research Institute
\n1999-2005, CEO and CSO, ToolGen, Inc.
\n2005-present, Assistant\/Associate\/Full Professor, Seoul National University
\n2014-present, Director, Institute for Basic Science (IBS)
\nHome pages: http:\/\/cge.ibs.re.kr\/html\/cge_en and http:\/\/gel.snu.ac.kr<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]<\/p>\n
CRISPR RNA-guided Genome Editing in Human Stem Cells, Animals, and Plants<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]<\/p>\n
Genome editing that allows targeted mutagenesis in cells and organisms is broadly useful in biology, biotechnology, and medicine. We have developed ZFNs, TALENs, and Cas9\/Cpf1 nucleases to modify chromosomal DNA in a targeted manner. In particular, we used purified Cas9\/Cpf1 proteins rather than plasmids to correct large chromosomal inversions in the factor VIII gene that cause hemophilia A in patient-derived iPSCs or to modify diverse genes in animals and plants. The resulting cells and organisms contained small indels at target sites, which are indistinguishable from naturally-occurring variations, possibly bypassing regulatory requirements associated with use of recombinant DNA.
\nDespite broad interest in RNA-guided genome editing, Cas9 and Cpf1 are limited by off-target mutations. We developed nuclease-digested whole genome sequencing (Digenome-seq) to profile genome-wide specificities of Cas9 and Cpf1 nucleases in an unbiased manner. Digenome-seq captured nuclease cleavage sites at single nucleotide resolution and identified off-target sites at which indels were induced with frequencies below 0.1%. We also showed that these off-target effects could be avoided by using purified Cas9\/Cpf1 ribonucleoproteins (RNPs) and modified guide RNAs. Digenome-seq is a robust, sensitive, unbiased, and cost-effective (< USD 1,500) method for profiling genome-wide off-target effects of programmable nucleases.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Troels-KOCH-\u535a\u58eb-1.png” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Troels KOCH, PhD<\/strong><\/span><\/h4>\nVP and Head of Research, RNA Therapeutics<\/i><\/em><\/p>\nRoche, Denmark \/ Switzerland<\/i><\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
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.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]<\/p>\n
New perspectives in LNA therapeutics<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]A new modelling strategy has been employed to get a better understanding on how the various structural units will impact the properties of LNA. The modelling pointed to the internucleoside phosphorothioate (PS) linkage and demonstrated that stereo defined LNA PS serve as specific determinants for LNA activity. Pre-clinical data will be used to illustrate how stereo defined PS can influence and improve central drug properties of LNA. PS has been a \u201chot topic\u201d and debated over the years and adding to this the use of stereo defined vs. random mixture PS will be discussed in a bigger picture.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Ekkehard-Leberer-Ph.D.-Professor-of-Biochemistry.jpg” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Ekkehard LEBERER, Ph.D., Professor of Biochemistry<\/strong><\/span><\/h4>\nSenior Director of R&D Alliance Management, Sanofi, Germany<\/i><\/em><\/p>\nScientific Managing Director of COMPACT Consortium, Innovative Medicine Initiative, Belgium<\/i><\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.12″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
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).<\/p>\n
Since joining Hoechst Marion Roussel in 1998, Dr. Leberer carried out various managing roles in this company, Sanofi\u2019s 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.<\/p>\n
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.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]<\/p>\n
MicroRNA therapeutics for targeting the pathways of human disease<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.12″ open=”off”]MicroRNAs regulate biochemical pathways and networks of pathways by the mechanism of RNA interference (RNAi). Many microRNAs have been demonstrated to be dysregulated in human diseases. For example, microRNA-21 has been implicated in multiple organs as a microRNA associated with fibrosis and cancer. Therefore, microRNAs open a novel target space for drug development.This presentation will summarize the opportunities and challenges of developing microRNA-based drugs and will illustrate the generation of an anti-fibrotic microRNA-based therapeutic approach by targeting microRNA-21 with an antisense oligonucleotide (anti-miR-21).<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Judy-LIEBERMAN-MD-PhD.jpg” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Judy LIEBERMAN, MD, PhD<\/strong><\/span><\/h4>\nCellular and Molecular Medicine Program, Boston Children\u2019s Hospital and Department of<\/i><\/em><\/p>\nPediatrics, Harvard Medical School, Boston MA USA<\/i><\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
Judy Lieberman holds a Chair in Cellular and Molecular Medicine at Boston Children\u2019s 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.<\/p>\n
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.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]<\/p>\n
The Silent Treatment: Targeted gene knockdown<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]<\/p>\n
Effective therapeutic strategies for in vivo siRNA delivery to knockdown genesin cells outside the liver are needed to harness RNA interference for treating most diseases. Here we describe two flexible platforms for targeted delivery that use RNA aptamers or antibodies for selective cell uptake in vivo and gene knockdown.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Muthiah-Mano-MANOHARAN-\u535a\u58eb-1.png” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Muthiah MANOHARAN, PhD<\/strong><\/span><\/h4>\nSenior Vice President, Drug Discovery, Alnylam Pharmaceuticals, USA
Board Director, Oligonucleotide Therapeutics Society<\/em><\/p>\n\n
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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.<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Presentation Title” _builder_version=”3.11.1″ open=”off”]<\/p>\n
RNAi Therapeutics in Human Disease using GalNAc-siRNA Conjugates: How sweet it is to work with sugars<\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”Summary” _builder_version=”3.11.1″ open=”off”]At Alnylam, siRNA-GalNAc conjugates are emerging as a potential new class of medicine supporting a broad clinical pipeline across multiple therapeutic areas. We have been able to continuously optimize the siRNA chemical modifications and design resulting in the Enhanced Stabilization Chemistry (ESC) platform exhibiting improved efficacy and extended duration lasting for several months. Our progress and applications will be presented.<\/p>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][\/et_pb_row][et_pb_row use_custom_gutter=”on” gutter_width=”0″ _builder_version=”3.25″ custom_margin=”|||” custom_padding=”2px|0|72px|0px|false|false” column_structure=”1_3,2_3″][et_pb_column type=”1_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.cnaf.org.cn\/wp-content\/uploads\/2018\/08\/Craig-C.-MELLO-PhD.png” _builder_version=”3.23″ max_width=”70%” custom_margin=”|||” custom_margin_tablet=”90px|||” custom_margin_last_edited=”off|desktop” always_center_on_mobile=”off” locked=”off”][\/et_pb_image][\/et_pb_column][et_pb_column type=”2_3″ _builder_version=”3.25″ custom_padding=”|||” custom_padding__hover=”|||”][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_text_color=”#7e7f93″ text_font_size=”18px” header_font=”||||||||” header_3_font=”Merriweather|700|||||||” header_3_text_color=”#4646c4″ header_3_font_size=”36px” header_3_line_height=”1.4em” locked=”off” inline_fonts=”Arial”]<\/p>\n
Craig C. MELLO, PhD<\/strong><\/span><\/h4>\nProfessor, University of Massachusetts\u00a0<\/em>
Howard Hughes Medical Institute, USA\u00a0<\/em>
Nobel Prize in Physiology or Medicine (2006)<\/em><\/p>\n\n
[\/et_pb_text][et_pb_text _builder_version=”3.27.4″ text_font=”||||||||” text_font_size=”18px” text_line_height=”1.8em”][\/et_pb_text][et_pb_accordion _builder_version=”3.11.1″ toggle_font=”||||||||”][et_pb_accordion_item open=”on” _builder_version=”3.11.1″ custom_padding=”|||” custom_css_toggle_content=”margin-top:-30px;”]<\/p>\n
Dr. Mello\u2019s lab uses the nematode\u00a0C. elegans<\/em>\u00a0as a model system to study embryogenesis and gene silencing.\u00a0 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\u00a0C. elegans<\/em>\u00a0is 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 \u201cknock out\u201d 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.<\/p>\nDr. 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.<\/p>\n
Besides the Nobel Prize, Dr. Mello\u2019s 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\u2019s 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\u00a0American Philosophical Society.<\/p>\n
Additional information about Dr. Mello can be found in the following sites:<\/p>\n
http:\/\/www.hhmi.org\/scientists\/craig-c-mello<\/a><\/p>\nhttp:\/\/profiles.umassmed.edu\/profiles\/ProfileDetails.aspx?From=SE&Person=1009<\/a><\/p>\n
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