Oligonucleotide Discovery and Delivery
Oligonucleotide CMC and Regulatory Strategies
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
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.