Speaker Biographies

Oligonucleotide Discovery and Delivery

Oligonucleotide CMC and Regulatory Strategies

Artificial Intelligence for Drug Discovery & Development

Rare Disease Drug Development


Oligonucleotide Discovery and Delivery

Annemieke Aartsma-Rus, PhD, Professor of Translational Genetics, Leiden University Medical Center
Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. She played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy during her PhD research (2000-2004) at the Leiden University Medical Center (the Netherlands). As of December 2007 she became leader of the “DMD exon skip group”. Since 2013 she has a visiting professorship at the Institute of Genetic Medicine of Newcastle University (UK). She is President of the Oligonucleotide Therapeutics Society (2019-2020) and vice-chair of COST Action CA17103 (Delivery of antisense RNA therapies). In 2013 she was elected a member of the junior section of the Dutch Royal Academy of Sciences (DJA), which consists of what are considered the top 50 scientists in the Netherlands under 45. In 2018, she was one of the founding members of Young Academy Leiden (YAL), the local Leiden counterpart of the DJA. She has been selected as most influential scientist in Duchenne muscular dystrophy in the past 10 years by Expertscape based on contributions to the understanding and treatment of Duchenne muscular dystrophy four times in a row (2015-now).

Kerry Benenato, PhD, Senior Director, Discovery Chemistry, Moderna
Kerry received her B.S. in Chemistry from Providence College after which she moved to Boston College, where she obtained her Ph.D., working in the labs of Amir Hoveyda, focusing on Cu-catalyzed enantioselective allylic substitution reactions. Following that she worked in the labs of Matt Shair at Harvard University as a NIH postdoctoral fellow.  After her post-doc she joined Astra Zeneca Pharmaceuticals, where she worked in the Infectious Diseases group, focusing on the identification of new therapies for Gram-negative infections.  After seven years at AZ, she moved to Moderna, where she currently leads the department of Discovery Chemistry, which focuses on the development of mRNA-based therapies.        

David Corey, PhD, Professor, Department of Pharmacology, UT Southwestern
Dr. Corey graduated with a BA from Harvard in 1985 and a PhD from UC Berkeley under the direction of Dr. Peter Schultz in 1990. He has had his own laboratory at UT Southwestern since 1992 and is currently the Rusty Kelley Professor of Biomedical Science in the Department of Pharmacology.

Marvin Caruthers, PhD, Distinguished Professor, University of Colorado
Professor Marvin H. Caruthers research includes nucleic acids chemistry and biochemistry. Approximately 35 years ago, the methodologies that are used today for chemically synthesizing DNA and RNA were developed in his laboratory. His laboratory has also pioneered the synthesis of many new nucleic acid analogs that have found applications in the nucleic acid diagnostic and therapeutic areas. He is the recipient of the National Academy of Sciences Award for Chemistry in Service to Society, the Prelog Medal, the Economists Award in Biotechnology, and the US National Medal of Science. He is also the recipient of The National Academy of Science Award in the Chemical Sciences, The American Chemical Society Award for Creative Invention, The Frantisek Sorm Medal-The Academy of Sciences of the Czech Republic and The Life Time Achievement Award of the Oligonucleotide Therapeutic Society. Dr. Caruthers is an elected member of The US National Academy of Sciences, The American Academy of Arts & Sciences, The National Inventors Hall of Fame, The National Academy of Inventors, and a Corresponding Member of the German Academy of Science Gottingen. One of the co-founders of Amgen and Applied Biosystems, Dr. Caruthers remains active in the Biotechnology arena – most recently as a co-founder of Array BioPharma and miRagen Therapeutics.

Elena Feinstein, MD, PhD, CSO, Quark Pharmaceuticals
Elena Feinstein MD, PhD is a Chief Scientific Officer for Quark Pharmaceuticals, Inc. She received her MD degree (with honors) from the Russian State Medical University (former 2nd Moscow State Medical School) and PhD degree (with honors) in Molecular and Cellular Biology from the Weizmann Institute of Science in Israel. Since joining Quark, she has been holding increasingly senior positions, first leading the high throughput functional gene discovery programs in numerous disease areas and then taking the lead in establishment of Quark’s siRNA therapeutics platform, drug discovery and early development. Prior to joining to Quark, Dr. Feinstein conducted academic research in the fields of apoptosis and cancer in the Weizmann Institute of Science. She is a principle or senior author of dozens of publications in the high impact peer-reviewed journals and an inventor on numerous awarded patents and patent applications.

Chantal Ferguson, Senior PhD Student, RNA Therapeutic Institute, University of Massachusetts Medical School
Chantal Ferguson is a graduate of Wesleyan University with a BA in Neuroscience. At Wesleyan, she perfumed research in Dr. Stephen Devoto’s neuromuscular development lab, studying the genetic regulatory network involved in zebrafish neuromuscular development. After graduating college, Chantal performed research in the Breast Cancer Center at Massachusetts General Hospital, focusing on treatment related complications, outcomes, and patient care. As she embarked on her MD/PhD at UMass Medical School (UMMS), Chantal's passion for studying neurological diseases combined with her interest in RNA interference led her to Dr. Anastasia Khvorova’s lab in the RNA Therapeutics Institute. The Khvorova lab develops and chemically optimizes short interfering RNAs (siRNAs) for CNS delivery. Chantal’s thesis project focuses on developing and using siRNAs to understand the relationship between Apolipoprotein E and neurodegeneration. Chantal’s clinical interests include neurology and interventional radiology.

Peter Hagedorn, Senior Principal Scientist and Team Leader of Bioinformatics and RNA Biology, Roche Innovation Center Copenhagen
Peter Hagedorn is a Senior Principal Scientist at Roche Innovation Center Copenhagen. He leads a team of RNA biologists and bioinformaticians exploring oligonucleotide mechanisms of action and design strategies. Research in his team is currently focused on custom development of massively-parallel sequencing methods to evaluate RNA target sequence and oligonucleotide properties. Data from such applications are used to build structure-effect machine learning models for computational prediction of drug properties. Peter holds a master’s degree from 2002 in Biophysics from the Niels Bohr Institute, University of Copenhagen, Denmark. In 2006 he obtained his PhD in Molecular Biology and Bioinformatics from the University of Southern Denmark.

Ekkehard Leberer, PhD, Senior Director, R&D Alliance Management, Sanofi
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). 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.

Dong-Ki Lee, PhD, CEO, 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 80 papers including prestigious journals such as Nature, Cell, PNAS, and Molecular Therapy. In 2010, he founded OliX Pharmaceuticals, a publicly traded RNAi therapeutics company, and serves as Chief Executive Officer.

Patrick Lu, PhD, President & CEO, Sirnaomics
Patrick Y. Lu, Ph.D, Founder, President and CEO of Sirnaomics, Inc., Chairman of Suzhou Sirnaomics and Guangzhou Nanotides. Dr. Lu started his biopharmaceutical industry career in 1993 as a lab head in Novartis and was the co-founder and Executive VP of Intradigm Corporation (2001-2006). Patrick has authored more than 50 scientific papers, review articles and book chapters, and is an inventor for more than 50 issued and pending patents. He has been an invited speaker in many international conferences throughout the world and received a number of Government grants. Under his leadership, Sirnaomics has raised more than US$70 million dollars and has developed a series of “first-in-class” siRNA therapeutic candidates at different phases of clinical studies, in both USA and China. The company’s therapeutic programs are focused on anticancer and antifibrotic indications.   Recent development of the company’s leading drug product in combination with immune checkpoint inhibitor (mAb) for treatment of hepatocellular carcinoma has demonstrated a strong therapeutic benefit with a syngeneic mouse model.

Brett Monia, PhD, CEO, Ionis Pharmaceuticals
Dr. Monia is the chief executive officer and a founding member of Ionis Pharmaceuticals. His contributions at Ionis include research into the medicinal chemistry and mechanisms of action of RNA-targeting modalities to treat human diseases, most notably antisense-based therapeutic strategies. Dr. Monia has extensive experience across a range of therapeutic areas, including oncology, metabolic disease, inflammation, neurological disease and cardiovascular disease, which have resulted in a broad range of successful clinical achievements and in marketing approvals for new medicines. Dr. Monia has published more than 200 primary research manuscripts, reviews and book chapters, and is an inventor on more than 100 issued patents. He serves as a senior editor for the journal Nucleic Acid Therapeutics, is on the board of directors for Dynacure, and has served as president of the Oligonucleotide Therapeutics Society (OTS). Dr. Monia is also an adjunct professor of biology at San Diego State University where he lectures at the graduate level on pharmacology. Dr. Monia received his Ph.D. in Pharmacology from the University of Pennsylvania and B.S. degrees in Molecular Biology and Analytical Chemistry from Stockton University in Pomona, New Jersey.

Dmitry Samarsky, PhD, CTO, Sirnaomics
Dmitry Samarsky, PhD has been at the inception of RNAi technology and drug development - starting in 2001 as Director of Technology Development at Sequitur (acquired by Invitrogen) and (in 2005) as Director of Technology Development at Dharmacon (now part of GE). He then served as VP of Technology Development at RXi Pharmaceuticals, USA (2007-2011), SVP of Technology and International Business Development at RiboBio, China (2011-2016) and, most recently, as Chief Scientific Officer at Silence Therapeutics, Germany/UK (2016-2018). Dr. Samarsky has authored more than 40 scientific papers, articles, book chapters, patents and patent applications. He has been an invited speaker at more than 100 international conferences, and currently serves on the Scientific Advisory Boards for the OTS (Oligonucleotide Therapeutics Society). Dr. Samarsky received his doctorate in biochemistry and molecular biology from University of Massachusetts, Amherst (1998), followed by a postdoctoral position as an H. Arthur Smith Fellow for Cancer Research in Michael Green's lab at University of Massachusetts Medical School (1998-2001).

David Tabatadze, PhD, President, ZATA Pharmaceuticals
In 2009, Dr. David Tabatadze cofounded ZATA in partnership with Dr. Paul Zamecnik (1912-2009) where he has served as the President and CEO. Dr. Tabatadze has led the development of novel nucleic acid platform technology enabling the synthesis of next generation oligonucleotides (ON) with custom-designed properties based on the incorporation of various branched chemical groups on the internucleoside phosphates. ZATA’s ONs (ZONs) are applicable to all currently known oligotherapy-based approaches including antisense and CRISPR. Dr. Tabatadze has significant experiencing in consulting medical device companies in the development and FDA clearance of commercially available light-based medical devices. His thirty-plus years of R&D experience includes developing novel drug candidates and diagnostic probes, business development and management, execution of IP portfolio and regulatory submission. Dr. Tabatadze possesses a MS in organic chemistry, a PhD in bioorganic chemistry, and has received post-doctoral training in nucleic acid chemistry at the Worcester Foundation for BioMedical Research (Shrewsbury, MA). He has also co-authored over three dozen peer-reviewed papers, patent applications, and patents. At ZATA, Dr. Tabatadze has established business and collaborative relationships with several biotech and academic institutions locally and internationally.

Chandra Vargeese, PhD, Senior Vice President, Drug Discovery, Wave Life Sciences
Dr. Vargeese has over 25 years of experience in oligonucleotide research and development, and has authored numerous scientific publications and patents. Before joining Wave, Dr. Vargeese served as Executive Director of Chemistry and Delivery, RNA Therapeutics Division at Novartis. She led siRNA delivery as Senior Director in the RNA Therapeutics division at Merck. She joined Merck through its $1.1 billion acquisition of Sirna Therapeutics, where she was Vice President of Chemistry. Dr. Vargeese served as Associate Director of Chemistry at NeXstar Pharmaceuticals and is the co-inventor of Macugen® (pegaptanib), an approved therapy for wet AMD. She earned a PhD in organic chemistry at the Indian Institute of Science, Bangalore, India, and completed post-doctoral work at University of Rhode Island.

Brad Wan, PhD, Principal Scientist, Medicinal Chemistry, Ionis Pharmaceuticals
Dr. W. Brad Wan is a Principal Scientist in the Department of Medicinal Chemistry at Ionis Pharmaceuticals located in Carlsbad, CA.  Dr. Wan has been with Ionis pharmaceuticals since 2007 focusing on the synthesis of novel nucleoside monomers and oligonucleotide conjugates to improve potency and uptake in various tissues.  His most recent work has been to evaluate the significance of controlling the stereochemistry of the phosphorothioate linkages that comprise the backbone of antisense oligonucleotide therapeutics.

Mark Yen, PhD, Director, Clinical Development, Arrowhead Pharmaceuticals
Mark Yen has been with Arrowhead Pharmaceuticals a Medical Director within the Clinical Development group since February of 2019.  In the past 15+ years, he has worked in gene therapy, rare disease, various ophthalmology indications, as well as having been a Principal Investigator at a Phase 1 unit.  He graduated from Albert Einstein College of Medicine and finished family medicine training at Kaiser Fontana.

Oligonucleotide CMC and Regulatory Strategies

Phil Baran, PhD, Professor, Department of Chemistry, Scripps Research
Phil Baran was born in 1977 in Denville, New Jersey. He received his BS in chemistry in 1997 (NYU), his PhD at The Scripps Research Institute in 2001, and from 2001-2003 he was a postdoctoral fellow at Harvard. Phil has published over 180 scientific articles and has been the recipient of several ACS awards such as the Corey (2015), Pure Chemistry (2010), Fresenius (2006), and Nobel Laureate Signature (2003), and several international distinctions such as the Hirata Gold Medal and Mukaiyama Prize (Japan), the RSC award in Synthesis (UK), and the Sackler Prize (Israel). In 2013 he was named a MacArthur Foundation Fellow, in 2015 he was elected to the American Academy of Arts and Sciences, and in 2016 he was awarded the Blavatnik National Award. He consults for numerous companies such as Bristol-Myers Squibb (since late 2005), Boehringer-Ingelheim, AstraZeneca, DuPont and TEVA, and is a scientific advisory board member for Eisai, Abide, and AsymChem. In 2016 he was appointed as an Associate Editor for the Journal of the American Chemical Society. He co-founded Sirenas Marine Discovery (2012) and Vividion Therapeutics (2016) and in 2013 he co-authored "The Portable Chemist’s Consultant," an interactive book published on the Apple iBooks store.

Vera Brinks, PhD, Director Pharmaceutics, ProQR Therapeutics
Vera Brinks is director pharmaceutics at ProQR Therapeutics, where she is responsible for formulation development and DP manufacturing process development. She has over 15 years’ experience in the (bio) pharmaceutical field, with a focus on formulation-related quality attributes of oligonucleotides and biologicals. Vera received her PhD in the field of biopharmaceutics at the Leiden University (The Netherlands), after which she has been postdoctoral fellow at the Utrecht University (the Netherlands) and at the FDA (CDER, DTP, US), before joining ProQR in 2014.

Jesse Faber, Senior Process Engineer III, ASO Process Engineering and Manufacturing, Biogen
Jesse Faber is a Senior Process Engineer in the Process Engineering and Manufacturing group at Biogen and is responsible for the operation of an ASO clinical and commercial manufacturing facility.  Jesse has twenty years of experience in the pharmaceutical industry in drug substance and drug product manufacturing for companies including Merck and GlaxoSmithKline.  He has been at Biogen for the past four years where he has helped to lead the startup of Biogen’s first internal ASO manufacturing facility and the subsequent process validation of their first ASO molecule.  Jesse holds a Bachelor’s degree in Chemical Engineering from Clemson University.

Vidhya Gopalakrishnan, PhD, Senior Vice President, Pharmaceutical Development, Quark Pharmaceuticals
Vidhya Gopalakrishnan Ph.D. is currently Senior Vice President, Pharmaceutical Development, at Quark Pharmaceuticals. She has over 20 years of drug development experience spanning small and large molecules and multiple delivery platforms in various therapeutic areas. Prior to joining Quark, she held various positions including SVP, R&D Operations, at Neuraltus Pharmaceuticals, and other leadership positions at Jazz Pharmaceuticals, Aerogen, ALZA, and Cygnus. Vidhya received her PhD in the area of oligoribonucleotide synthesis and structural studies from National Chemical Laboratory, Pune India and MSc in Chemistry from IIT, Madras. She has held post-doctoral positions at Penn State University and Stanford University School of Medicine.

Hong Jiang, Scientist II, Analytical Development, Biogen
Ms. Hong Jiang received her Master’s degree in 1999 from University of Texas at Austin where she conducted research in the mechanism of phage Mu transposition under the direction of Prof. Rasika M. Harshey. She has accumulated 17 years of experience in drug discovery and development while working at Wyeth BioPharma, Novartis Institutes for BioMedical Research, and currently Biogen. At Biogen Hong is engaged in analytical development and CMC of antisense oligonucleotide APIs and Drug Products. She currently leads a group of scientists focusing on ASO and small molecule impurity identification and analytical development to support the ASO synthesis process development. Her experience before joining Biogen includes drug metabolism, analytical method development for drug discovery, and protein drug formulation.

Marc Lemaitre, PhD, Consultant, Sirnaomics
Marc Lemaître holds a degree in Organic Chemistry and a PhD in Biochemistry from the University of Liège, Belgium.  Since 1985 Marc’s professional interests has been the study of the Nucleic Acids and therapeutics as well as diagnostic applications. Based in the USA since 2006 he worked for Glen Research, as CEO of Girindus America, a CMO with a focus on the cGMP manufacture of oligonucleotides for therapeutic applications.  Marc is now working as consultant since 2013, working mostly on CMC with start-up and Pharma companies developing therapeutic oligonucleotides and peptides.

Paul Manley, President & Principal Consultant, Orvieto Consulting
Paul F. Manley, has accumulated 30+ years of business and senior management experience in regulatory affairs, compliance and overall drug development, across a range of therapeutic areas within the pharmaceutical, biologic and consumer products industries. Mr. Manley’s corporate career encompassed senior management and technical leadership positions within the US and Europe in both large corporations (VP, Global Regulatory Affairs – Oncology, J&J) and start-up-like entities (VP, Regulatory Affairs and Compliance, Genta Incorporated). In 2007 Mr. Manley established Orvieto Consulting, LLC, providing strategic advice to numerous companies across a wide range of scientific disciplines and through all phases of development through to post-marketing.

Melissa Marschel, Director, Regulatory Affairs CMC, Alnylam Pharmaceuticals
Melissa Marschel is an experienced professional with over 15 years working in CMC, Regulatory and QA. She has in-depth knowledge related to biologics development, life-cycle management, and drug substance/drug product manufacturing across multiple therapeutic classes and disease areas. She is currently a Director of Regulatory Affairs – CMC at Alnylam Pharmaceuticals. Melissa joined Alnylam in 2017 as the Reg CMC lead for Alnylam's first GalNAc conjugate siRNA candidate, Givlaari, which was recently approved in the US. She led the strategic development and execution of the CMC content for the initial NDA and MAA submissions, and supported interactions with Health Authorities related to Alnylam’s novel starting materials and impurities control strategies. Moving forward, her and her team are responsible for global CMC strategy, including rest-of-world submissions and life cycle management for Givlaari. Prior to joining Alnylam, Melissa worked in Regulatory CMC at Seres Therapeutics, and over the years has held numerous roles across Regulatory CMC, CMC Program Management and Quality at Biogen, Genzyme and Alkermes. She received her B.S in Biology from Dickinson College and her M.Sc. in Drug Regulatory Affairs and Health Policy from Northeastern University.

Mary M Sherman, PhD, Principal Consultant, Preclinical & Regulatory Consulting
Dr. Sherman has over 25 years’ experience in advancing new therapeutics into the clinic.  Dr. Sherman worked in the pharmaceutical industry for over 15 years, following which she has been a drug development consultant for thirteen years.  Dr. Sherman has a reputation as an effective leader and technically proficient scientist with experience in late-stage (lead optimization/selection) through Phase II Development for Fortune 500 pharmaceutical companies, contract research organizations (CROs), and global drug development consultancy.  Dr. Sherman has expertise in lead optimization/selection, ADME/PK/Bioanalytical, Toxicology, Physical Pharmacy, for small molecules (45%), peptides/proteins (10%) and oligonucleotides (45%) , as well as regulatory issues.  As a consultant she is responsible for designing and managing IND-enabling programs, where she oversaw writing and filing of 7 IND/IMPDs and numerous amended INDs with FDA/EMA in 8 years.  Dr. Sherman has worked in the therapeutic areas of neurosciences, anti-infectives, inflammation, hematology, oncology and immuno-oncology.  She earned her Ph.D. in pharmaceutical chemistry at the University of Wisconsin-Madison and received post-doctoral training at the University of Utah in biological chemistry.

Roumen Radinov, PhD, Director Process Chemistry, Alnylam Pharmaceuticals
Roumen Radinov received his Ph.D. in organic chemistry from Sofia University, Bulgaria under the direction of Professor Marietta Haimova. Following postdoctoral studies in the area of enantioselective methodology and asymmetric synthesis with Wolfgang Oppolzer, Ned Porter, and Barry Trost, he joined the Roche Chemical Synthesis Department where he has developed and scaled up the syntheses of multiple drug candidates and led oligonucleotide drug substance and lipid excipients supply for the siRNA program. In 2012 he joined Alnylam Pharmaceuticals where he is currently Director of Process Chemistry.

Francis Ring, Assistant Director, Manufacturing & Operations, Ionis Pharmaceuticals
Francis Ring is currently an Assistant Director at Ionis Pharmaceuticals in Carlsbad and has worked in oligonucleotide manufacturing for 12 years.  Prior to Ionis, he completed a B.S. in Chemical Engineering at Stanford University.  At Ionis, he leads the design and implementation of automated equipment, including oligonucleotide synthesizers, chromatography skids, and the building automation.  He is also the primary architect of all manufacturing trending programs at Ionis, including developing custom validated scripts for data collection purposes.

Kimberly Tyndall, Principle, CMC Tyndall Consultant
Kim recently retired after 38 years at GlaxoSmithKline.  Her career spanned both analytical chemistry development and as a Director in CMC Regulatory Affairs both pre and post approval dealing with small molecules, biopharms and oligonucleotide drug substance and drug products. Kim supported and filed the first ever rapid microbiology comparability protocol, subsequent submission and Patriot inspection.  In addition, she coordinated and conducting an FDA training class for rapid microbiological techniques.  Kim has worked solid dosage forms, sterile drug substance and drug products as well as novel administration dosage forms. After recently retiring from GSK she formed CMC Tyndall Consultant LLC. whose main drive is to support all CMC aspects to progress new medicines and avenues of hope for those with rare diseases and to progress oligonucleotide therapies. 

Artificial Intelligence for Drug Discovery & Development

Shruthi Bharadwaj, PhD, Senior Scientist, Novartis Oncology Precision Medicine
Shruthi Bharadwaj is a Senior Scientist at Novartis within the Informatics and Analytics group.  She received her Ph.D. in Biomedical Engineering from the University of Florida and continued her research as a post-doctoral fellow at the MD Anderson Cancer Center. Shruthi has been interested and involved in utilizing AI and machine learning approaches in Pharma. She has a patent that involves machine-learning approach to predict the onset of colon cancer in patients with Inflammatory Bowel Disease.  She has won several NIH grants that supported her research in leveraging AI approaches in healthcare.  She has published several research articles, book chapters and abstracts that focus on AI approaches in diagnosis and drug development.

Barun Bhhatarai, PhD, Investigator, Novartis Institute for Biomedical Research
Dr. Bhhatarai joined Novartis (NIBR) since Nov 2015 to support computational ADME projects using machine learning, data integration, ADME modeling and MedChem based approaches. Before, Novartis, he worked in the Predictive Safety Assessment Center at The Dow Chemical Company, Michigan, where he contributed to the development, assessment and implementation of novel in-silico approaches aimed at predicting potential mammalian and environmental health effects. His graduate and post-doctoral research focused on cheminformatics, bio-assay/ chemical ontology and computer-aided drug design at Clarkson University, New York and Center for Computational Science at University of Miami, Florida. After obtaining his PhD, he joined the University of Insubria, Italy where he performed Predictive Toxicology research and Risk Assessment of emerging pollutants for EU – FP7 project. He is a member of the American Society for Cellular and Computational Toxicology (ASCCT) and the American Chemical Society (ACS - COMP, CINF). He has authored 25+ peer-reviewed publications and several presentations.

Olivier Elemento, PhD, Director, The Caryl and Israel Englander Institute for Precision Medicine; Associate Director, Institute for Computational Biomedicine, Weill Cornell Medicine
Dr. Elemento directs the Englander Institute for Precision Medicine, an Institute that focuses on using genomics and informatics to make medicine more individualized. We have developed a systematic approach to create tumor organoids across 12 tumors types and use high-throughput screening identify optimal, patient-specific anticancer treatment options. The focus of my research group is on the systems biology of cancer; we focus on prostate cancer and hematological malignancies. In these cancers, we are elucidating the patterns of aberrant pathway activities, rewiring of regulatory networks and cancer mutations that have occurred in cancer cells. We are also trying to understand how tumors evolve at the genomic and epigenomic level. We use high-throughput sequencing (ChIP-seq, RNA-seq, bisulfite conversion followed by sequencing – specifically RRBS-, ATAC-seq, exome capture and sequencing, single cell RNAseq using DropSeq) to decipher epigenetic mechanisms and regulatory networks at play in malignant cells and study how they affect gene expression. My lab has developed several computational approaches for analysis of deep sequencing data, e.g. ChIPseeqer (for integrative analysis of ChIP-seq data) and SNVseeqer/INDELseeqer (full pipeline for mutation detection and characterization from deep sequencing data). My lab has developed several additional computational approaches that include a pathway analysis tool (iPAGE) several tools for regulatory element detection (FIRE and FastCompare) and RRBseeqer for ERRBS analysis (including detection of differentially methylated regions). We use drug repositioning to identify small molecules that can target mutated signaling pathways and classically undruggable proteins such as transcription factors. We model complex signaling pathways to identify drug combinations that can most efficiently shutdown aberrantly active pathways in cancer. In addition, I am Associate Director of the Institute for Computational Biomedicine.

Peter Hagedorn, Senior Principal Scientist and Team Leader of Bioinformatics and RNA Biology, Roche Innovation Center Copenhagen
Peter Hagedorn is a Senior Principal Scientist at Roche Innovation Center Copenhagen. He leads a team of RNA biologists and bioinformaticians exploring oligonucleotide mechanisms of action and design strategies. Research in his team is currently focused on custom development of massively-parallel sequencing methods to evaluate RNA target sequence and oligonucleotide properties. Data from such applications are used to build structure-effect machine learning models for computational prediction of drug properties. Peter holds a master’s degree from 2002 in Biophysics from the Niels Bohr Institute, University of Copenhagen, Denmark. In 2006 he obtained his PhD in Molecular Biology and Bioinformatics from the University of Southern Denmark.

Igor Jurisica, PhD, DrSc, Senior Scientist, Krembil Research Institute; Professor, Medical Biophysics, University of Toronto
Igor Jurisica, PhD, DrSc is a Senior Scientist at Krembil Research Institute, Professor at U Toronto and Visiting Scientist at IBM CAS. He is an Adjunct Professor at Pathology and Molecular Medicine at Queen's U, an adjunct scientist at the Institute of Neuroimmunology, Slovak Academy of Sciences, and an Honorary Professor at Shanghai Jiao Tong University. Since 2015, he has also served as Chief Scientist at the Creative Destruction Lab, Rotman School of Management. He has published extensively on data mining, visualization and integrative computational biology, including multiple papers in Science, Nature, Nature Medicine, Nature Methods, J Clinical Oncology, J Clinical Investigations, and has over 13,590 citations since 2014, including 848 highly influential citations (SemanticScholar). He has been included in Thomson Reuters 2014, 2015 & 2016 lists of Highly Cited Researchers (http://highlycited.com), and The World's Most Influential Scientific Minds: 2015 & 2014 Reports. In 2019, he has been included in the Top 100 AI Leaders in Drug Discovery and Advanced Healthcare list (Deep Knowledge Analytics, http://analytics.dkv.global).

Milind Kamkolkar, Chief Data Officer, Cellarity

Milind Kamkolkar is the Chief Digital & Data Officer of Cellarity. At Cellarity, digital is the operating model and data is a core value driver of the company. With this integrated approach, Milind is leading Cellarity’s diverse AI and Digital organization, which implements machine learning science on a digital biology landscape to explore new clinical hypotheses that can alter cell behaviors. Milind joined Cellarity from Sanofi where he was the first enterprise Chief Data Officer in the pharmaceutical industry. Milind’s focus was transforming Sanofi from a data-generation organization into a data-driven organization, to empower the lives of patients and enrich the value Sanofi delivered to customers. The approach was drawn from his tenure at Novartis where he was the Global Head of Data Science and AI in Digital Medicines. Milind is a featured speaker (TEDx), Business Transformation Top 150 Leader and thought leader in digital health, AI and big data. Milind has served as a board advisor and mentor to numerous startups, founders and VC firms focused on entering the life science space through digital, data or AI. He has served as an Honorary Lecturer for Physician Entrepreneurship at BartsX, Faculty at Exponential Medicine and a Former Special Advisor to the UN Global Compact Sustainable Development Goals program. Milind holds a B.Sc. from the University of Sydney and M.S. from Thomas Jefferson University.

Mary Jo Lamberti, PhD, Research Assistant Professor; Associate Director of Sponsored Research, Tufts Center for the Study of Drug Development
Mary Jo Lamberti is a faculty member at Tufts and leads multi-company sponsored and grant funded research studies at the Tufts Center for the Study of Drug Development (CSDD) at Tufts University School of Medicine. She has extensive experience conducting research on pharmaceutical and biotechnology industry practices and trends affecting contract research organizations and investigative sites. She has been a speaker at industry conferences and has published articles in trade and peer-reviewed journals. She holds a B.A. from Wellesley College and a Ph.D. in psychology from Boston University.

Michael Liebman, PhD, Managing Director, IPQ Analytics, LLC
Michael N. Liebman, PhD, is the Managing Director of IPQ Analytics, LLC and Strategic Medicine, Inc after serving as the Executive Director of the Windber Research Institute (now ChanSoon-Shiong Institute for Molecular Medicine) from 2003-2007. He is an Adjunct Professor of Pharmacology and Physiology at Drexel College of Medicine and Adjunct Professor of Drug Discovery, First Hospital of Wenzhou Medical University and also Fudan University. Previously, he was Director, Computational Biology and Biomedical Informatics, University of Pennsylvania Cancer Center 2000-2003. He served as Global Head of Computational Genomics, Roche Pharmaceuticals and Director, Bioinformatics and Pharmacogenomics, Wyeth Pharmaceuticals, Director of Genomics for Vysis, Inc. He is a co-founder of Prosanos, Inc (now United BioSource) (2000). He was Associate Professor of Pharmacology and of Physiology/Biophysics at Mount Sinai School of Medicine. He serves on 14 scientific advisory boards and the Board of Directors of the Nathaniel Adamczyk Foundation in Pediatric ARDS and Innovene Pharmaceuticals. Michael is Chair of the Informatics Program and also Chair of Translational Medicine and Therapeutics for the PhRMA Foundation and a member of their Scientific Advisory Board. His research focuses on computational models of disease progression that stress risk detection, disease processes and clinical pathway modeling, and disease stratification from the clinical perspective. He utilizes systems-based approaches and design thinking to represent and analyze risk/benefit analysis in pharmaceutical development and healthcare.

Debbie Lin, PhD, Executive Director, Venture Fund Digital Health, Boehringer-Ingelheim
Debbie Lin is Executive Director at Boehringer Ingelheim Venture Fund in the US and leads the US efforts in Digital Healthcare.  She is a scientist by training and joined Boehringer Ingelheim in 2008.  Within Boehringer Ingelheim, she worked across various regions and divisions in the US in Medical Affairs, Health Economics and Outcomes with large payers and providers on the West Coast and in Corporate Headquarters in Germany in Corporate Strategy and Development. She led BI’s global venture into stroke rehabilitation setting up the first stroke rehabilitation center pilot in Shanghai, China and in Lisbon, Portugal. She now leads the Venture Fund efforts in digital healthcare for the US.

Arvind Rao, PhD, Associate Professor, Department of Computational Medicine and Bioinformatics, University of Michigan
Arvind Rao is an Associate Professor in the Department of Computational Medicine and Bioinformatics at the University of Michigan. His group uses image analysis and machine learning methods to link image-derived phenotypes with genetic data, across biological scale (i.e., single cell, tissue and radiology data). Such methods have found application in radiogenomics and drug repurposing based on phenotypic screens. Arvind received his PhD in Electrical Engineering and Bioinformatics from the University of Michigan, specializing in transcriptional genomics, and was a Lane Postdoctoral Fellow at Carnegie Mellon University, specializing in bioimage informatics.

 

Rare Disease Drug Development

John Doench, PhD, Director R&D, Genetic Perturbation Platform, Broad Institute of Harvard and MIT
John G. Doench is an Institute Scientist at the Broad Institute of MIT and Harvard. In that capacity, he provides expert guidance on the design, execution, and analysis of genetic screens. He has contributed to numerous publications in fields such as infectious disease, cancer biology, and immunology, highlighting both his commitment to team-based science and ability to mentor and guide scientists from diverse backgrounds on the critical principles of genetic screens. Additionally, as the Director of R&D in the Genetic Perturbation Platform (GPP), John leads a group focused on the development of functional genomic techniques, first with RNAi and more recently with CRISPR technology. Here, his team demonstrated the potential of genetic screens with CRISPR and has since developed leading bioinformatics tools and screening libraries to enable community-wide usage of this powerful technology. Importantly, their efforts emphasize not only staying on the cutting-edge of newest approaches but also focusing on the reduction-to-practice that is critical for enabling collaboration with a broader community of researchers working in diverse and challenging model systems. Prior to joining the Broad in 2009, John did his postdoctoral work at Harvard Medical School, received his PhD from the biology department in Phil Sharp’s lab at MIT, and majored in history at Hamilton College. John lives in Jamaica Plain, MA with his wife and daughter, where he enjoys screaming for the Red Sox and Patriots, playing volleyball, running, and avoiding imminent death while navigating the streets of Boston on a bicycle.

David V. Erbe, PhD, Senior Distinguished Investigator, Alnylam Pharmaceuticals
Dave Erbe joined Alnylam in 2014 with over 20 years’ experience in drug discovery and development at companies including Genentech, Genetics Institute, and Wyeth, translating novel therapeutics from discovery through clinical proof-of-concept across a range of diseases. Dave received a PhD in Biochemistry from Dartmouth following a BS in Chemistry from Carnegie Mellon and currently serves as a Senior Distinguished Investigator in Alnylam Research.

Paloma H. Giangrande, Director, Research, Rare Diseases, Moderna Therapeutics
Paloma Giangrande is an Associate Professor of Internal Medicine at the U of Iowa and a Director of Research at Moderna Therapeutics in Cambridge, MA. She obtained her BS in Biochemistry at Wheaton College in 1994 and her PhD in Pharmacology and Cancer Biology at Duke University in 1999. Dr. Giangrande is an internationally recognized expert in developing new types of RNA therapeutics. She stands at the exciting junction between molecular biology and its clinical applications to solving major health problems. Her contributions to the field have been recognized with awards from the American Association for Cancer Research and the University of Iowa and a prestigious Young Investigator Award from the American Society for Gene and Cell Therapy (ASGCT). Her scientific record, accomplishments and leadership roles make her an exemplary role model for young investigators.

Anthony Hickey, PhD, Director, UNC Catalyst for Rare Diseases, University of North Carolina, Eshelman School of Pharmacy
Dr. Hickey is Distinguished RTI Fellow, at the Research Triangle Institute, Director of the UNC Catalyst for Rare Diseases of the Eshelman School of Pharmacy and Adjunct Professor Biomedical Engineering in the School of Medicine, at the University of North Carolina at Chapel Hill. He obtained Ph.D. (1984) and D.Sc. (2003) degrees in pharmaceutical sciences from Aston University, Birmingham, UK. Following postdoctoral positions, at the University of Kentucky (1984-1988) Dr. Hickey joined the faculty at the University of Illinois at Chicago (1988-1993). In 1990 he received the AAPS Young Investigator Award in Pharmaceutics and Pharmaceutical Technology. He is a Fellow of the Royal Society of Biology (2000), the American Association of Pharmaceutical Scientists (2003), the American Association for the Advancement of Science (2005) and the Royal Society of Biology (2017). He received the Research Achievement Award of the Particulate Presentations and Design Division of the Powder Technology Society of Japan (2012), the Distinguished Scientist Award of the American Association of Indian Pharmaceutical Scientists (2013); the David W Grant Award in Physical Pharmacy of the American Association of Pharmaceutical Scientists (2015); Thomas T Mercer Joint Prize for Excellence in Inhaled Medicines and Pharmaceutical Aerosols of the American Association for Aerosol Research and the International Society for Aerosols in Medicine (2017) and the Ralph Shangraw Memorial Award for Excipient and Excipient Technology of the International Pharmaceutical Excipient Consortium Foundation (2018). He has published numerous papers and chapters (over 250) in the pharmaceutical and biomedical literature, one of which received the AAPS Meritorious Manuscript Award in 2001. He has edited five texts on pharmaceutical inhalation aerosols and co-authored three others on ‘Pharmaceutical Process Engineering’, pharmaceutical particulate science and ‘Pharmaco-complexity’. He holds 25 US patents on a variety of inhaler device technologies, pulmonary and oral drug delivery formulation technologies. He is founder (1997, and formerly President and CEO, 1997-2013) of Cirrus Pharmaceuticals, Inc., which was acquired by Kemwell Pharma in 2013; founder (2001, and formerly CSO, 2002-2007) of Oriel Therapeutics, Inc, which was acquired by Sandoz in 2010 and founder and CEO of Astartein, Inc. (2013-present); member of the Pharmaceutical Dosage Forms Expert Committee of the United States Pharmacopeia (USP, 2010–2015, Chair of the sub-committee on Aerosols) and formerly Chair of the Aerosols Expert Committee of the USP (2005-2010). Dr. Hickey conducts a multidisciplinary research program in the field of pulmonary drug and vaccine delivery for treatment and prevention of a variety of diseases.

Radhakrishnan P. Iyer, PhD, Co-Founder and CSO, Spring Bank Pharmaceuticals
R. P. Iyer, (Kris) is the co-founder and Chief Scientific Officer of Spring Bank Pharmaceuticals. He has more than 25 years’ experience in drug discovery in diverse therapeutic areas including antivirals, inflammation and immune-oncology. Kris is an innovator in the fields of nucleic acid chemistry, bioorganic chemistry and pharmaceutical sciences with over 100 publications and 150 issued and filed U.S. and international patents. Prior to Spring Bank, Kris was the co-founder and VP of Discovery of Origenix Technologies.

Luke Koblan, Graduate Student, Laboratory of Dr. David Liu, Department of Chemistry and Chemical Biology, Harvard University
Luke Koblan is a graduate student in David Liu’s lab where he has worked on improving current base editor technologies, applying base editors for the correction of disease, and developing new tools for installing different targeted base transformations. Before his time in the Liu Lab, he worked for both Jay Bradner developing cell-based assays for targeted bromodomain inhibition and Todd Golub on a serine protease-profiling project. These experiences led to an interest in using technology development as a platform to illuminate new biology. He is supported by an NSF GRFP fellowship.

Michael Liebman, PhD, Managing Director, IPQ Analytics, LLC
Michael N. Liebman, PhD, is the Managing Director of IPQ Analytics, LLC and Strategic Medicine, Inc after serving as the Executive Director of the Windber Research Institute (now ChanSoon-Shiong Institute for Molecular Medicine) from 2003-2007. He is an Adjunct Professor of Pharmacology and Physiology at Drexel College of Medicine and Adjunct Professor of Drug Discovery, First Hospital of Wenzhou Medical University and also Fudan University. Previously, he was Director, Computational Biology and Biomedical Informatics, University of Pennsylvania Cancer Center 2000-2003. He served as Global Head of Computational Genomics, Roche Pharmaceuticals and Director, Bioinformatics and Pharmacogenomics, Wyeth Pharmaceuticals, Director of Genomics for Vysis, Inc. He is a co-founder of Prosanos, Inc (now United BioSource) (2000). He was Associate Professor of Pharmacology and of Physiology/Biophysics at Mount Sinai School of Medicine. He serves on 14 scientific advisory boards and the Board of Directors of the Nathaniel Adamczyk Foundation in Pediatric ARDS and Innovene Pharmaceuticals. Michael is Chair of the Informatics Program and also Chair of Translational Medicine and Therapeutics for the PhRMA Foundation and a member of their Scientific Advisory Board. His research focuses on computational models of disease progression that stress risk detection, disease processes and clinical pathway modeling, and disease stratification from the clinical perspective. He utilizes systems-based approaches and design thinking to represent and analyze risk/benefit analysis in pharmaceutical development and healthcare.

Ryan Leung, Vice President, Strategy & Corporate Development, Research to the People
Research to the People is a non-proift organization dedicated to advancing research, increasing awareness, and empowering patients with rare diseases through unique patient-partnered research collaborations. By uniting physicians, scientists, and advocates with individual patients and patient communities; we aim to accelerate the pace of discovery, expand our understanding of rare diseases, and develop new care strategies. As Vice President of Strategy & Corporate Development, Ryan leads RttP's strategic planing and expansion efforts across R&D and partnering. Prior, he held various roles in technical development and product management for bioinformatics software, personalized biotherapeutics, and computational medicine.

Jonathan Stokes, PhD Banting Fellow, Laboratory of Dr. James Collins, Broad Institute of MIT and Harvard

Jonathan Stokes is a Banting Fellow in the laboratory of James Collins at the Broad Institute of MIT and Harvard. He received his BHSc in 2011, graduating summa cum laude, and his PhD in antimicrobial chemical biology in 2016, both from McMaster University. His research applies a combination of chemical biology, systems biology, and machine learning approaches to develop novel antibacterial therapies with expanded capabilities over conventional antibiotics. Dr. Stokes is the recipient of numerous awards, including the Canadian Institutes of Health Research Master’s Award, the Colin James Lyne Lock Doctoral Award, and was ranked first of just 23 postdoctoral scholars to be awarded the prestigious Banting Fellowship.

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Oligonucleotide Discovery and Delivery