Speakers

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Eli Eisenberg
Professor
Tel Aviv University

Eli Eisenberg is a Professor of Physics and a member of the Sagol school of neuroscience at Tel Aviv University. Since 2002, he has been working on bioinformatic analyses of RNA expression data. In particular, he has developed computational approaches for detection, quantification and analysis of RNA editing, focusing on the most common type of RNA editing, adenosine-to-inosine editing. These methods were utilized to chart the editome of multiple species, study the genomic and transcriptomic characteristics governing the scope of editing in each species, and understanding the ways in which editing may have a functional impact on the cell and the whole organism, in health and disease.

Day Two

Wednesday November 20 2019

1:50 pm | The Alu-Editing-Index: Transcriptome-Wide Quantification of ADAR activity

Maria Montiel Gonzalez
Scientist, RNA editing platform
Beam Therapeutics

Maria Montiel Gonzalez a native from Maracaibo, Venezuela moved to Puerto Rico to pursue her undergraduate studies at the Universidad del Sagrado Corazon. Following this, she obtained her Ph.D. from the Department of Biochemistry of the University of Puerto Rico at the Medical Sciences Campus under the supervision of Dr. Joshua Rosenthal at the Institute of Neurobiology in Old San Juan, Puerto Rico. During her doctorate she published several pioneering studies on site-directed RNA editing using a novel approach to direct the catalytic activity of human ADAR2 to specific adenosines within the RNA. She continued developing this promising therapeutic tool adapting the system for viral delivery at the Marine Biological Laboratory at Woods Hole, Massachusetts. Currently she accepted a position as an R&D Scientist at Beam Therapeutics landing her expertise to the platform of site-directed RNA editing.

Day One

Tuesday November 19 2019

9:50 am | Current Strategies for Site-Directed RNA editing using ADARs

Erez Levanon
Professor
Bar-Ilan University

Erez Levanon is a professor of biology at the faculty of life sciences, Bar-Ilan University; Israel, where he is the head of the genomic research lab, focusing mainly with the study of RNA editing. He received his PhD from Tel-Aviv University after graduating the Adi Lautman Interdisciplinary Program for Outstanding Students. Later he was a postdoctoral research follow at the genetics department of Harvard Medical School. Prior to that, he was a senior scientist in Compugen LTD. He has published over 80 manuscripts and won several awards and fellowships.

Day One

Tuesday November 19 2019

11:20 am | Developing Technology & Algorithms to Uncover A-to-I RNA Editing in Humans & Animal Models

Gene Yeo
Professor
University of California, San Diego

Gene Yeo PhD MBA is a Professor of Cellular and Molecular Medicine at the University of California, San Diego (UCSD). Dr. Yeo has a BSc in Chemical Engineering and a BA in Economics from the University of Illinois, Urbana-Champaign, a Ph.D. in Computational Neuroscience (Machine Learning) from Massachusetts Institute of Technology and an MBA from the UCSD Rady School of Management. Gene’s research interest is in understanding and manipulating RNA processing in development and disease using induced pluripotent stem cell and animal models. Gene’s lab develops and utilizes multidisciplinary technologies ranging from machine learning, biochemistry, molecular biology, genomics, chemistry and materials research. Gene is widely collaborative and emphasizes on reproducibility and robustness in his data and results, and transparency and honesty in communicating with trainees and colleagues. Dr. Yeo has authored more than 160 peer-reviewed publications including invited book chapters and review articles in the areas of neurodegeneration, RNA processing, computational biology and stem cell models; and served as Editor on two books on the biology of RNA binding proteins. Recently, his lab developed enhanced CLIP for the purposes of large-scale mapping of protein-RNA interactions. His lab also demonstrated in vivo RNA targeting with CRISPR/Cas proteins with proof of concept in repeat expansion disorders. Gene is on the Editorial Boards of the journals Cell Reports and Cell Research. Gene joined UCSD as an Assistant Professor in 2008, was promoted with tenure to Associate Professor in 2014 and to Professor in 2016. Gene was the first Crick-Jacobs Fellow at the Salk Institute (2005-2008) and is a recipient of the Alfred P Sloan Fellowship in recognition of his work in computational molecular biology (2011), Singapore National Research Foundation Visiting Investigatorship Award (2017), the inaugural Early Career Award from the International RNA Society (2017), a Blavatnik National Award Finalist (2018) and San Diego Xconomy Award Finalist (2019). Gene’s research has been funded by the National Institute of Health, National Science Foundation, California Institute for Regenerative Medicine, TargetALS, ALS Foundation, Department of Defense, Myotonic Dystrophy Association, Myotonic Dystrophy Foundation and Chan-Zuckerberg Initiative. Gene is a co-founder of biotech companies which includes Locana, Eclipse Bioinnovations, Enzerna and Proteona. Gene serves or had served on the scientific advisory boards of the Allen Institute of Immunology, Locana, Eclipse Bioinnovations, Proteona, Aquinnah, Cell Applications, Nugen and Ribometrix.

Day One

Tuesday November 19 2019

1:50 pm | Identification and modulation of RNA binding protein binding sites

Nina Papavasiliou
University Professor and Division Head
German Cancer Research Center, Heidelberg, Germany
Visiting Professor
Rockefeller University, New York

I have a long-standing interest in processes that generate informational diversity within cells. This interest originally directed my lab's work to DNA mutation in the context of the adaptive immune response specifically to mechanisms that direct mutation to antibody genes in the germinal center. This process which depends on AID-mediated DNA deamination followed by error prone repair generates immunoglobulin sequence diversity and underlies affinity maturation of the antibody response. More recently my interest in molecular processes that generate informational diversity within cells has expanded to the field of epitranscriptomics where my lab has been a leader in the field of RNA editing and modification with a focus on the functional relevance of the resulting transcriptomic diversity in healthy tissue (e.g. innate immune cells) and in disease (in the context of cancer).  

Day Two

Wednesday November 20 2019

12:20 pm | Realizing RNA Editing as a Critical Immuno-Oncology Tool

Jonathan Gootenberg
McGovern Fellow
McGovern Institute for Brain Research, MIT

Jonathan Gootenberg draws from fundamental microbiology to engineer new molecular tools, which he applies to the study of aging. These tools, including the popular genome editing system CRISPR, allow for unprecedented manipulation and profiling of cellular states in the body, and have multiple applications in basic science, diagnostics, and therapeutics. Along with McGovern Fellow Omar Abudayyeh, Gootenberg uses gene editing, gene delivery, and cellular profiling methods to understand the changes that occur in the brain and other organs during aging, with the goal of generating new therapies for degenerative disease.

Day One

Tuesday November 19 2019

9:10 am | RNA Editing with CRISPR-based systems

Dr. Joshua Rosenthal
Senior Scientist
Marine Biological Laboratory

Dr. Joshua Rosenthal is a Senior Scientist at the Marine Biological Laboratory in Woods Hole, Massachusetts. He received his B.S. in biology from Haverford College. He completed his doctoral work at Stanford University under the supervision of William Gilly, focusing on the voltage-dependent ion channels expressed in the squid giant axon. As a postdoc at UCLA with Francisco Bezanilla he studied RNA editing in the messages that encode the squid ion channels and how they affected function.  Before moving to the Marine Biological Laboratory, he rose from Assistant Professor to Professor at the Institute of Neurobiology in San Juan, Puerto Rico. Dr. Rosenthal’s lab has long focused on high-level RNA editing in cephalopods. These studies inspired his group to pioneer strategies to use RNA editing by adenosine deamination as a therapeutic. He is an academic founder of Korro Bio and continues to develop new strategies for site-directed RNA editing.

Day One

Tuesday November 19 2019

8:40 am | Directed RNA Editing, How it has Evolved and General Applications

Omar Abudayyeh
McGovern Fellow, the McGovern Institute for Brain Research
MIT

Omar is currently an independent MIT McGovern Fellow at the McGovern Institute for Brain Research at MIT. Previously, he was an MD/PhD student at Harvard Medical School/Harvard-MIT Health, Sciences, and Technology program and completed my PhD in Feng Zhang’s lab at the Broad Institute of MIT and Harvard in 2018. He has had multiple fellowship positions, including Friends of the McGovern Institute Fellowship, Paul and Daisy Soros Fellowship, National Science Foundation Graduate Fellowship, National Defense Science and Engineering Graduate Fellowship, and the NIH F30 National Research Service Award. He is currently on leave from Harvard Medical School while pursuing my independent research activities. In 2012, I graduated with a S.B. degree in Mechanical Engineering and Biological Engineering from the Massachusetts Institute of Technology as a Henry Ford II Scholar and Barry Goldwater Scholar.  

Day One

Tuesday November 19 2019

9:10 am | RNA Editing with CRISPR-based systems

Thorsten Stafforst
Professor
University of Tuebingen

Thorsten is professor of Nucleic Acid Biochemistry at the Interfaculty Institute of Biochemistry at the University of Tuebingen. He studied chemistry in Göttingen and expanded to chemical biology and bio-engineering during his postdoc in Zürich. In 2011, he started his independent lab in Tübingen, supported by an ERC Consolidator Grant. Since 2016, he is permanent professor in Tübingen supported by a Heisenberg-Professur (DFG). His lab focusses  mainly on site-directed RNA editing strategies and their exploitation in life sciences and medicine.

Day One

Tuesday November 19 2019

11:50 am | RNA Editing: Engineering editases, guideRNAs, and ASOs for application in Life Sciences and Medicine

Yi-Tao Yu
Principal Investigator and Professor
University of Rochester Medical Centre

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

Day One

Tuesday November 19 2019

4:20 pm | Suppression of nonsense mutations in disease genes by targeted RNA pseudouridylation

Hyo Min Park
CTO & co-founder
GenEdit

Dr. Park co-founded GenEdit based on a vision to revolutionize the delivery of gene therapies. His research experience in the field of molecular biology and metabolic disorders equipped him with the technical expertise and managerial experience that are required to effectively execute and lead multiple projects. He has expanded his research to gene therapy field after he and co-founders started GenEdit.  A few projects at GenEdit that Dr. Park have led showed successful results that were published in Nature BME and Nature Communications. He completed his Ph.D. in Metabolic Biology at UC Berkeley.

Day Two

Wednesday November 20 2019

3:20 pm | Delivering Next Generation Genetic Therapeutics with polymer nanoparticle

Scott Ribich
Vice President, Biology
Accent Therapeutics

Scott Ribich is Vice President of Biology, overseeing all aspects of discovery biology at Accent Therapeutics. Prior to joining Accent, Scott was Director of Biology at Epizyme, where he was responsible for leading teams involved with target identification, drug discovery and clinical candidate support. He started his career in drug discovery at GlaxoSmithKline and Sirtris Pharmaceuticals, where he worked on multiple early stage and clinical stage assets. Scott holds a B.S. in biology from Massachusetts Institute of Technology, a Ph.D. in biochemistry from Harvard University and completed his postdoctoral training at Harvard Medical School and Brigham and Women’s Hospital.

Day Two

Wednesday November 20 2019

2:20 pm | ADAR1 as a Therapeutic Target in Type I Interferon Signaling-High Cancer

U. Thomas Meier
Professor
Albert Einstein College of Medicine

Dr. Meier is Professor in the Department of Anatomy and Structural Biology at the Albert Einstein College of Medicine in New York, NY. Originally from Switzerland, he obtained his MSc from the ETH in Zurich and his PhD from the Biocenter of the University of Basel. He then moved to the Rockefeller University in New York, NY for postdoctoral training with the Nobel Laureate Günter Blobel. The work in his laboratory is mainly focused on the function and biogenesis of small nucleolar ribonucleoproteins (snoRNPs), which site-specifically modify target RNAs. Of particular interest is the impact of inherited diseases on these RNA-protein complexes. Based on this work, Dr. Meier was elected Fellow of the American Association for the Advancement of Science (AAAS). Another branch of his laboratory investigates markers of human endometrial receptivity.

Day One

Tuesday November 19 2019

3:50 pm | Cellular impact of the RNA modification machinery on pre-mRNA splicing, ribosome biogenesis, and telomere length regulation

Jeffrey Ostrove
CEO & Director
Locana

Jeffrey M. Ostrove, Ph.D. is the CEO of Locana, an RNA targeting gene therapy company developing treatments for a variety of neuromuscular and genetic diseases. Prior to Locana, he was CEO of AbVitro Inc., a therapeutic target discovery company which he sold to Juno Therapeutics.  Prior to AbVitro, Dr. Ostrove was the president and CEO of Ceregene, Inc., a clinical stage company developing treatments for Alzheimer’s and Parkinson’s disease. Ceregene sold to Sangamo. Prior to Ceregene, Dr. Ostrove was COO of NeuroVir, and instrumental in its merger with Medigene. Preceding NeuroVir (Medigene), Dr. Ostrove served as SVP and chief scientist of MAGENTA of Rockville, Maryland, a CDMO he founded while at BioReliance, Inc. where he was VP of scientific development. Dr. Ostrove served on the staff of the NIAID at the NIH. Dr. Ostrove holds a Ph.D. in molecular biology from the University of Florida College of Medicine and completed his post-doc at The Johns Hopkins University School of Medicine. Dr. Ostrove is also a past Board member of the Biotechnology Innovation Organization (BIO).

Day Two

Wednesday November 20 2019

10:10 am | Harnessing RNA-binding proteins to develop a portfolio of RNA-targeting gene therapies

Alan Herbert
Founder & Head of Discovery
InsideOutBio

Alan Herbert identified the Zα domain of ADAR. He was able to show using Mendelian Genetics that  Zα loss of function mutations with diminished binding to the left-handed Z-DNA and Z-RNA double helical conformations map directly to type I interferonopathies, establishing a biological role for these left-handed structures. Dr Herbert also pioneered family-based Genome Wide Association Studies in human populations and identified an immunosuppressive molecule predictive of response to anti-PD-1 cancer therapeutics while at Merck & Co. He founded InsideOutBio in the fall of 2017.

Day Two

Wednesday November 20 2019

2:50 pm | ADAR Editing: From ‘A’ to ‘Z’

Francois Vigneault
Co-Founder, President & CEO
Shape Therapeutics

Day Two

Wednesday November 20 2019

9:10 am | Application of ADAR in Development of Rare Disease Therapeutics

Jin Billy Li
Associate Professor
Stanford University

Dr. Li's laboratory focuses on studying ADAR-mediated RNA editing. His group has made contributions to identifying and mapping RNA editing in many different species, as well as understanding the cis and trans regulation. Currently, the Li Lab is primarily interested in ADAR1’s roles in innate immunity and transcriptome engineering.

Day Two

Wednesday November 20 2019

12:20 pm | ADAR1 RNA editing evades MDA5-mediated dsRNA sensing in innate immunity

Michael Jantsch
Professor
Medical University of Vienna

Studies of Biology (PhD), University of Vienna

  • Understanding heterochromatin evolution
Post Doctoral Research Carnegie Institution for Science, Baltimore
  • Studies on snRNP assembly and Cajal body composition
Assistant and Assoc Prof University of Vienna
  • Deciphering double stranded RNA binding domain
  •  Cloning and molecular dissection of Xenopus ADAR1
  •   Identification of massive RNA editing in repeat regions
  •  Identification of novel coding substrates
  • Distinguishing ADAR1 and ADAR2 function
Full Prof. Medical University of Vienna
  •  Understanding the impact of RNA editing on protein-coding substrates
  • Establishment of mouse models to study the impact of editing on protein function
  •  Study of innate immunity triggers in ADAR1 deficient mice

Day Two

Wednesday November 20 2019

11:50 am | Local & Global Mechanisms Controlling RNA-Editing

Oliver Rausch
Chief Scientific Officer
STORM Therapeutics

Oliver Rausch is Chief Scientific Officer at Storm Therapeutics Ltd. Storm is a spin-out from Cambridge University targeting RNA modification pathways for the development of a new class of anti-cancer treatments. As CSO, Oliver has overall responsibility for developing Storm’s RNA modification analysis platform, and for delivering small molecule drugs from its emerging drug discovery pipeline. Prior to Storm, Oliver was Programme Director at the National Institute for Health Research (NIHR) where he led major industry-focussed collaboration programmes in experimental medicine and early drug development. Oliver has over twenty years’ of drug discovery experience in Inflammation, Neurology and Oncology from organisations such as GlaxoSmithKline, UCB Celltech and Cellzome. He holds a degree in Biochemistry from the Free University in Berlin and a PhD in Biochemistry and Cell Biology from the Institute of Cancer Research in London

Day One

Tuesday November 19 2019

12:20 pm | Targeting RNA modifying enzymes for the treatment of cancer

David Rodman
Executive VP R&D
ProQR

David Rodman, MD is our Executive Vice President of Research & Development. David joined ProQR in 2017 having previously served in leadership roles with Novartis Institutes for Biomedical Research (NIBR), Vertex Pharmaceuticals, miRagen Therapeutics and Nivalis Therapeutics. Prior to moving to industry in 2005, David had a distinguished academic career, leading the Center for Genetic Lung Diseases at the University of Colorado and directing the Cystic Fibrosis Care, Teaching and Research efforts at the National Jewish Medical and Research Center in Denver, Colorado. During 12 years in industry, David has had global responsibility for driving innovation in the translation of cutting-edge science into transformational new therapies for rare diseases including CF, pulmonary fibrosis, pulmonary artery hypertension and severe immunologic and inflammatory diseases. At Vertex Pharmaceuticals he directed early- and late-stage CF clinical development programs including Kalydeco®, Orkambi® and VX-661. David received a BA in Economics from Haverford College in 1976, an MD from the University of Pennsylvania in 1980 and completed training in Internal Medicine, Pulmonary and Critical Care Medicine at the University of Colorado. He has served as an advisor to the National Institutes of Health, was elected to the American Society for Clinical Investigation and is a Fellow of the American Heart Association.

Day Two

Wednesday November 20 2019

9:40 am | Translating RNA Editing to Our Clinical Development Pipelines

Andrew Fraley
Founder & SVP Technology
Korro Bio

Andrew Fraley, a founder of Korro Bio, serves as SVP of Technology. Andrew brings over 20 years of experience in the field of nucleic acid technologies and therapeutics. A scientist and entrepreneur at heart, he has founded biotech companies including Triplet Therapeutics, Korro Bio and Trucode Gene Repair, and additionally functions as an Entrepreneur for Atlas Venture. Prior to Korro, Andrew was a commercial founder and served as SVP of Translational Sciences for a Trucode Gene Repair, an in vivo gene editing company, leading the company's scientific strategy and operations. Dr. Fraley has authored and reviewed scientific publications and is listed as an inventor on patents in areas including nucleic acid therapeutics, oligonucleotide delivery, and macrocyclic drug discovery. He holds a B.A. from DePauw University, earned his Ph.D. in Bioorganic Chemistry at Boston College, and was a Chateaubriand Postdoctoral Research Fellow at the Université de Louis Pasteur.

Day One

Tuesday November 19 2019

4:50 pm | Chairs Closing Remarks

8:30 am | Chair’s Opening Remarks

Prashant Mali
Assistant Professor, Bioengineering
University of San Diego

Day One

Tuesday November 19 2019

2:20 pm | Recruitment of Endogenous and Exogenous ADARs for RNA Editing New Approaches and New Challenges