● Latest advances in non-coding RNA research

● Nucleic acids based clinical trial

● Nucleic acids based diagnostics and biomarkers

● Nucleic acids based therapeutics

Uttiya Basu received his Ph.D. degree in Molecular Biology from Albert Einstein College of Medicine in 2004. After finishing postdoctoral training in the laboratory of Dr. Frederick Alt at Harvard Medical School in 2009, he joined the faculty of Columbia University, Department of Microbiology and Immunology. His laboratory utilizes technologies Involving genome engineering, genomics and biochemistry. His research is focused on the various mechanisms by which non-coding RNA transcription controls genome architecture in pluripotent and differentiated mammalian cells. He has been awarded the NIH director’s New Innovators Award, the Leukemia Lymphoma Society Career Development award, The Pershing Square Sohn Cancer Research Alliance prize, Irvington Institute and Cancer research institute post-doctoral fellowship and Lymphoma Research Foundation Award for cancer research.

Torsten Hoffmann joined Silence Therapeutics as Chief Operating Officer in June 2017. He has over 20 years of international R&D management experience. Prior to joining Silence, Torsten served as Chief Scientific Officer and Managing Director at Proteros biostructures from July 2015 and before then as Chief Scientific Officer and Executive Vice President at Zealand Pharma from July 2013. He previously spent 16 years at Roche in roles of increasing responsibility up to Site Head of Medicinal Chemistry at the company head quarter in Switzerland, and Head of the Global Roche Postdoc fellowship program. Torsten is the lead inventor of the anti-emetic medicine Netupitant, discovered at Roche and approved by the FDA as Akynzeo© in 2014. Torsten holds a PhD in Chemistry from the ETH Zürich, Switzerland, which was followed by a role as research associate at the Scripps Research Institute in La Jolla, California. He has authored more than 85 publications, patent applications and published conference reports and has served on a number of advisory boards. His awards include the Feodor Lynen award and a fellowship from the Alexander von Humboldt Foundation, Germany.

http://www.silence-therapeutics.com/

https://www.linkedin.com/in/hoffmanntorsten/

Dr. Kay has published over 250 scientific papers. The focus of the laboratory is to establish the scientific principles required for gene and nucleic acid transfer for the treatment of genetic and acquired diseases. Dr. Kay has worked on the development of many DNA gene transfer vectors and the mechanism by which they transduce tissues in mammals. His group has performed two Phase I/II gene therapy trials for hemophilia B. His laboratory was the first to establish therapeutic RNAi in whole non-embryonic mammals, and RNAi-mediated inhibition of a human viral pathogen (HBV) in animals. His work continues towards defining the molecular limits of delivered and expressed RNAi in vivo as well as the mechanisms involved insi/shRNA-mediated gene silencing and the biological mechanisms involved in miRNA-mediated gene repression. In addition, his laboratory is studying the role that a newly discovered small non-coding RNA plays in mammalian gene regulation.

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.

Lilley has made many studies of the interactions between DNA junctions and proteins, especially the resolving enzymes. This most recently led to the crystal structure of the eukaryotic enzyme GEN I bound to the product of cleavage of the four-way DNA junction, which has provided significant insight into the recognition process.

Lilley has had a long-standing interest in the structure and catalytic mechanisms of ribozymes. He has made extensive mechanistic study of the VS and hairpin ribozymes, and in the last three years he has solved high resolution crystal structures of the twister and TS ribozymes.

In RNA structure Lilley has made a detailed study of the structure, folding, protein binding and role of kink-turns, to the point where it is arguably the best-understood structural motif in RNA. Recently this has even gone into the nanotechnology area.

Two guanidine riboswitch structures have recently been solved by X-ray crystallography in the Lilley-lab, and will be discussed in the conference. He has also made a recent study of the structure effect of the common RNA modification N6-methyladenine, and shown how this disrupts trans sheared G•A basepairs, and hence box C/D snoRNP assembly. This will also be addressed in the conference.

In addition to his research, David Lilley (李大卫) is very active in promotion of links with Chinese science. He is a visiting professor at Xiamen University and at the University of the Chinese Academy of Sciences, a consultant for AB Life in Wuhan and has collaborative links with Shanghai Fudan University. He is also a frequent consultant for the Royal Society on Chinese matters.

Additional information about Dr. Lin can be found in the following site:

Web: http://stemcell.yale.edu/people/haifan_lin.profile

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

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

In 1988, the Lodish laboratory identified and cloned the receptor for erythropoietin (Epo), the hormone that controls the production of red blood cells. This led to a lengthy set of ongoing projects on the activation of, and signal transduction by, the erythropoietin receptor in erythroid progenitor cells and the regulation of transcription, apoptosis, and cell division during erythroid development. His laboratory is currently characterizing many novel genes that are important for several stages of erythropoiesis, including chromatin condensation and enucleation. A major focus is determining the roles of many long non-coding RNAs (lncRNAs) that are essential for the differentiation and function of erythroid and myeloid cells, and others essential for formation of white and brown adipose cells.

Most recently his laboratory developed culture systems for generating mature human red blood cells from hematopoietic stem cells. With Flagship Ventures he cofounded Rubius, a company that uses gene- modified red blood cells as vehicles for the long- term introduction of many novel therapeutics, immunomodulatory agents, and diagnostic imaging probes into the human body.

A Founding Member of Whitehead Institute, Lodish joined the MIT faculty in 1968. He has been a professor of biology since 1976 and professor of biological engineering since 1999. He earned his PhD at Rockefeller University in 1966. He was elected a fellow of the American Association for the Advancement of Science in 1986, a member of the National Academy of Sciences in 1987, and a fellow of the American Academy of Arts and Sciences in 1999.

Dr. Lodish is a member of the Board of Trustees of Boston Children’s Hospital, where he is also Chair of the Board of Trustees Research Committee. He was Founding Chair of the Scientific Advisory Board of the Massachusetts Life Sciences Center, the group charged with oversight of the state’s 10- year $1 billion investment in the life sciences. He was a founder and scientific advisory board member of several companies including Genzyme, Inc., Arris Pharmaceuticals, Inc, and Millennium Pharmaceuticals, Inc., and has served on the scientific advisory boards of numerous biopharmaceutical companies.

Dr. Lodish is the lead author of the textbook Molecular Cell Biology. The eighth edition was published in April 2016; the book has been translated into 12 languages. During the 2004 calendar year Dr. Lodish served as President of the American Society for Cell Biology.

Tomas Lindahl’s Mutagenesis Laboratory at Clare Hall discovered and characterized different DNA repair pathways in a long-term project to provide better understanding of the cellular defence mechanisms against damage to the human genome.

Damaged sites in the chromosomal DNA can result in cell death or cancer, but may be corrected by DNA repair enzymes prior to phenotypic expression. The properties of several nuclear enzymes that remove harmful lesions or local aberrant structures from DNA have been investigated. The absence of such DNA repair factors may result in an increased frequency of malignant transformation or in some cases may be detected as immunological deficiencies.

Dr. Lindahl closed his lab at the Clare Hall laboratories in 2009 but remains an Emeritus Group Leader at the Francis Crick Institute.

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

http://en.wikipedia.org/wiki/Tomas_Lindahl

http://vimeo.com/63309327

https://www.britannica.com/biography/Tomas-Lindahl

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:

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

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

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

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

Guillermo Montoya is Professor at the University of Copenhagen (UCPH) and Research Director of the Protein Structure & Function Programme at the Novo Nordisk Foundation Centre for Protein Research (http://www.cpr.ku.dk/). Montoya is also the Chairman of ISBUC, the Integrative Structural Biology Cluster at UCPH (http://isbuc.ku.dk/). In the last years, his main research interests are the structural analysis of macromolecular complexes of the cell cycle such as the mammalian chaperonin CCT complex, where he uses the combination of X-ray crystallography and electron microscopy to gain insight about their working mechanisms. Dr. Montoya is also systematically pursuing the structure-function analysis of endonucleases, which are of great interest because of their potential applications in gene therapy. He is member of international project evaluation panels and also reviews projects for European funding agencies (ERC, EU, SNF, DFG, NOW, MINECO, Welcome Trust) and serves as an associate editor and ad-hoc reviewer for high-profile journals.

Dr. Motamedi is an Assistant Professor in Medicine at Harvard Medical School and Massachusetts General Hospital Cancer Center. His group is interested in understanding the molecular mechanisms by which chromatin and non-coding RNAs initiate, maintain and propagate epigenetic states in eukaryotes. He received his Ph.D. from University of Alberta in Canada, and joined the Danesh Moazed lab at Harvard Medical School for his postdoctoral training initially as an NSERC and later as a CIHR postdoctoral fellow. His work as a postdoctoral trainee led to the proposal of the Nascent Transcript Model in which long noncoding RNAs provide a platform for the assembly of chromatin proteins in cis relative to their site of synthesis. His lab is now interested in uncovering the mechanisms by which eukaryotic cells establish the transcriptional program of quiescent cells. He has received several awards including the V Scholar and V Scholar Plus and served as reviewer for several high-profile journals and international granting bodies.

Dr. Richard J. Roberts is the Chief Scientific Officer at New England Biolabs, Beverly, Massachusetts. He received a Ph.D. in Organic Chemistry in 1968 from Sheffield University and then moved as a postdoctoral fellow to Harvard. From 1972 to 1992, he worked at Cold Spring Harbor Laboratory, eventually becoming Assistant Director for Research under Dr. J.D. Watson. He began work on the newly discovered Type II restriction enzymes in 1972 and these enzymes have been a major research theme. Studies of transcription in Adenovirus-2 led to the discovery of split genes and mRNA splicing in 1977, for which he received the Nobel Prize in Medicine in 1993. During the sequencing of the Adenovirus-2 genome computational tools became essential and his laboratory pioneered the application of computers in this area. DNA methyltransferases, as components of restriction-modification systems are also of active interest and the first crystal structures for the HhaI methyltransferase led to the discovery of base flipping. Bioinformatic studies of microbial genomes to find new restriction systems are a major research focus as is the elucidation of DNA methyltransferase recognition sequences using SMRT sequencing and a new approach to m5C containing recognition sequences.

From 2002-2015, she was Pr. Scientist to Sr. Scientific Director at Genzyme/Sanofi, her responsibilities included clinical assay development/validation and supporting clinical sample analysis for drug development and NDA/BLA filings (protein and small molecule drugs, cell/gene therapies, peptides and an antisense oligonucleotide). She was also Scientific Liaison for Sanofi-CRO strategic alliance. Prior to Genzyme, she studied transplantation and induction of immune tolerance at BioTransplant.

Yuanxin received her Ph.D. in Biochemistry (1990) from Iowa State University in Biochemistry as a CUSBEA scholar (China-US government program), and Bachelor of Medicine (1984) from Beijing Medical University (now Peking University). She is an active contributor to AAPS LBABFG, journal reviewer, and invited speaker.