Speaker Biographies

Sheron Branham, Associate Director, ASO Process Engineering and Manufacturing, Biogen

Sheron Branham is the Associate Director of Process Engineering and Manufacturing at Biogen and is responsible for the operation of their new GMP antisense oligonucleotide manufacturing facility.  She serves on the site leadership team for Biogen’s RTP facility and has been at Biogen for five years.  Sheron has twenty years of experience in the pharmaceutical industry with the majority of her career in small molecule drug substance and drug product manufacturing for companies like Merck and Glaxosmithkline.  She transitioned into oligonucleotides in 2016 to lead the startup of Biogen’s first internal ASO manufacturing facility.  She holds a Bachelor’s in Chemical Engineering from Clemson University and an MBA from NC State University.

Philip John (P.J.) Brooks, PhD, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health

Philip John (P.J.) Brooks joined the NCATS Office of Rare Diseases Research as a program director in August 2018. Prior to that time, he was in the NCATS Division of Clinical Innovation, where he was the lead program director for the Clinical and Translational Science Awards (CTSA) Program Collaborative Innovation Awards, designed to fund projects that will result in novel and creative approaches to overcoming roadblocks in translational science (PAR-18-244 and  PAR-18-245. Brooks represents NCATS on the Trans-NIH Microbiome Working Group and Gene Therapy Working Groups. He is also the Working Group Coordinator for the NIH Common Fund program on Somatic Cell Genome Editing. Brooks received his Ph.D. in neurobiology from the University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at the Rockefeller University, Brooks became an investigator in the intramural program of the National Institute on Alcohol Abuse and Alcoholism. He developed an internationally recognized research program focused on two distinct areas: the molecular basis of alcohol-related cancer, and rare neurologic diseases resulting from defective DNA repair, including xeroderma pigmentosum, Cockanye syndrome and Fanconi anemia.

Marvin Caruthers, PhD, Distinguished Professor, University of Colorado

Marvin H. Caruthers is a Distinguished Professor at the University of Colorado.  A Guggenheim Fellow, Dr. Caruthers completed the Ph.D. at Northwestern University and post-doctoral studies at MIT. Professor Caruthers interests include nucleic acids chemistry and biochemistry. Approximately 35 years ago, the methodologies for chemically synthesizing DNA/RNA were developed in his laboratory and incorporated into instruments for synthesizing DNA/RNA as used by biochemists, biologists, and molecular biologists. More recently his laboratory has developed methods for RNA chemical synthesis and for the synthesis of DNA/RNA on chips. 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 several academic and research awards including The National Academy of Sciences Award in the Chemical Sciences, The Prelog Medal, The Economists Award in Biotechnology, and The US National Medal of Science. 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, and a Corresponding Member of the German Academy of Science Gottingen. He is a co-founder of Amgen, Applied Biosystems, Array Bioscience, Barafold, and Miragen.

Hyung J. Chun, MD FAHA, Associate Professor of Medicine and Pathology, Yale School of Medicine

Hyung Chun is an Associate Professor of Medicine and Pathology at Yale School of Medicine.  His group's research focuses on mechanisms and novel therapeutic targets in cardiovascular disease, including pulmonary hypertension, heart failure, atherosclerosis, and diabetes.  

Will Dampier, PhD, Assistant Professor, Department of Microbiology & Immunology, Center for Molecular Virology & Translational Neuroscience, Drexel University College of Medicine

Will Dampier is an Assistant Professor with the Department of Microbiology and Immunology at the Drexel University College of Medicine. He is a computation biologist who studies the effects of HIV genetic variation on patient outcomes.

Paul Enríquez, JD, LLM, PhD Candidate, Structural and Molecular Biochemistry, North Carolina State University

Paul Enríquez is in the Department of Structural and Molecular Biochemistry at North Carolina State University. Outside the laboratory, his scholarly research and writing focuses on the intersection of law, science, and policy. He has published work in various legal, scientific, and popular media publications, and has been invited to speak at national and international conferences.

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.

Jennifer Franklin, Director, CMC Regulatory Affairs, Ionis Pharmaceuticals

Jenny has worked for Ionis Pharmaceuticals, Inc. in Carlsbad, CA, for 12 years.  For the past two, she has been responsible for CMC Regulatory Affairs for Ionis’ late phase/commercial compounds, as well as all development compounds.  Prior to that, she was responsible for Quality Assurance oversight of oligonucleotide drug substance manufacturing and testing activities, and associated functions. Before joining Ionis, Jenny worked in Quality Control for Gala Biotech (now Catalent BioPharma) in Middleton, WI.  She earned her B.S in Biology and French from the University of Wisconsin.

Samantha Gao-Sheridan, PhD, Senior Director, Regulatory Affairs CMC, Alnylam Pharmaceuticals

Dr. Gao-Sheridan is the Head and Senior Director of Regulatory Affairs CMC at Alnylam Pharmaceuticals, a company with a core focus in leading development of RNAi therapeutics toward genetically defined targets for the treatment of serious, life-threatening diseases. Dr. Gao-Sheridan is a drug development and regulatory affairs professional with extensive experience providing leadership in developing innovative and compliant regulatory strategy in biologics and drugs for rare and unmet medical need programs. While having broad knowledge and experience in regulatory affairs overall, she specializes in CMC regulatory affairs and has led global regulatory CMC strategy development, regulatory agency interactions, submissions, registration, and/or post-approval life cycle management of a broad range of therapeutics, including monoclonal antibodies, fusion proteins, enzyme replacement therapies, drug-device combination products, peptides, and RNAi therapeutics. Dr. Gao-Sheridan is passionate about the regulatory science and applying sound scientific approach to developing innovative regulatory solutions. She has participated and contributed in several industry organizations and working groups including PhRMA QbD Working Group, BIO Manufacturing, Quality, and Distribution Committee, and DIA conferences. Prior to joining Alnylam, Dr. Gao-Sheridan worked in Shire Pharmaceuticals, Wyeth Pharmaceuticals, and Vical Inc. She is a protein chemist and molecular biologist by training. She earned a B.S. in Medicine from Peking Union Medical College, M.S. in Toxicology from Rutgers University, and Ph.D. in Biochemistry and Molecular Biology from University of California, Irvine, followed by a post-doctoral fellowship in anthrax toxin research at Harvard Medical School.

Richard S. Geary, PhD, Senior Vice President (SVP), Development, Ionis Pharmaceuticals

Dr. Geary is Senior Vice President of Drug Development at Ionis Pharmaceuticals. He is responsible for preclinical and clinical development of antisense drugs, as well as manufacturing. Since joining Ionis in 1995, Dr. Geary has been involved in discovery and development including the regulatory submission of more than thirty investigational new drug applications and multiple successful NDAs to U.S. and other regulatory agencies. Dr. Geary received his Ph.D. in Biopharmaceutics from the University of Texas, College of Pharmacy, Austin, Texas and his B.S. in Biology from Texas A&M University, College Station, Texas.

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.

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.

James Hamilton, MD, Vice President Clinical Development, Arrowhead Pharmaceuticals

James currently serves as Vice President of Clinical Development at Arrowhead Pharmaceuticals, Inc., where he is responsible for clinical strategy, clinical trial design, implementation and execution of all clinical programs globally. Previously, he was medical director and head of Corporate Development at Arrowhead. He led Arrowhead's acquisition of the Novartis RNAi assets and led Arrowhead's $670 million cardiovascular deal with Amgen. He earned an MD and MBA at The Ohio State University and is a licensed physician with board certification in emergency medicine.

Bo Rode Hansen, PhD, President & CSO, GeneVant Sciences

Bo joined Genevant from Roche, where he served as Global Head of RNA Therapeutics and General Manager of the Roche Innovation Center Copenhagen. Prior to Roche, Bo was Executive Vice President and Head of Drug Discovery & Alliance Management at Santaris Pharma A/S through the company’s acquisition by Roche in 2014. Over the course of his career, Bo has led over 50 RNA therapeutic discovery programs which have yielded 10 clinical candidates and over 40 patents and patent applications.

James J. Hickman, PhD, Professor, Nanoscience Technology, Chemistry, Biomolecular Science, and Electrical Engineering, University of Central Florida

James J. Hickman is the Founding Director of the NanoScience Technology Center and a Professor of Nanoscience Technology, Chemistry, Biomolecular Science, Material Science and Electrical Engineering at the University of Central Florida. Previously, he held the position of the Hunter Endowed Chair in the Bioengineering Department at Clemson University. Dr. Hickman has a Ph.D. from the Massachusetts Institute of Technology in Chemistry. For the past twenty-five years, he has been studying the interaction of biological species with modified surfaces, first in industry and in the latter years in academia. While in industry he established one of the first bioelectronics labs in the country that focused on cell-based sensors and their integration with electronic devices and MEMS devices. He is interested in creating hybrid systems for biosensor and biological computation applications and the creation of functional in vitro systems for human body-on-a-chip applications. He has worked at NSF and DARPA in the area of biological computation. He is also the founder and current Chief Scientist of a biotechnology company, Hesperos, that is focusing on cell-based systems for drug discovery and toxicity. He has 131 publications and 20 book chapters, in addition to 21 issued patents out of 44 total patent applications.

Ingmar Hoerr, PhD, Founder and Chairman of the Supervisory Board, CureVac

Ingmar Hoerr, PhD, MBA, Founder / Chairman of the Supervisory Board co-founded the biopharmaceutical company CureVac in the year 2000 in Tuebingen. His entrepreneurship was motivated by a surprising discovery during his doctoral research. Experiments conducted for his research showed that the mRNA molecule class can be injected into tissue directly and is capable of expressing genes into proteins effectively as well as generating a strong specific immune response, contrary to what had previously been believed. From this key discovery, he built up a company in his role as CEO that was the first ever to treat a human subject with an mRNA drug and that is now a leading German “unicorn” in the research and development of mRNA-based drugs. Since its inception, CureVac has received approximately $330 million (€300 million) in equity investments. The company is valuated at $2 billion. Ingmar Hoerr received his PhD from the University of Tübingen and his MBA from Danube University, Krems, Austria. He currently advises the European Commission as member of the High Level Group of Innovators in developing a European Innovation Council and is juror of the founder prize Weconomy.

David Horn Solomon, PhD, Chief Executive Officer (CEO), Silence Therapeutics

David is an experienced public company biotech CEO, board member and biotech investor. He was the CEO of Zealand Pharma A/S (NASDAQ:ZEAL) from 2008 to 2015. Under David’s leadership the company went public on NASDAQ OMX and its lead product, Adlixin®, a GLP-1 receptor agonist for the treatment of type II diabetes, was approved in the US and globally and is now marketed by Sanofi as a monotherapy and in combination with Lantus as Soliqua®. David was also the CEO of Bionor Pharma ASA (OSL:BIONOR) and Akari Therapeutics, Plc (NASDAQ:AKTX), and was the Managing Partner of Sund Capital, ApS, a Nordic healthcare investment fund.

He has earlier served as a faculty member at Columbia University’s College of Physicians and Surgeons in New York City. From 2003 to 2006, David headed healthcare investment at Carrot Capital Healthcare Ventures in New York. David is currently a member of the Board of Directors of TxCell SA (NYSE EURONEXT:TXCL), and was earlier a member of the Boards at Onxeo SA (NYSE EURONEXT:ONXEO) and Promosome, LLC. Dr Solomon studied at Weil Cornell Medicine of Cornell University and its Graduate School of Medical Science where he received his Ph.D.

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.

Meisam Naeimi Kararoudi, DVM, PhD, Postdoctoral Fellow, Cell Therapy and Cancer Immunotherapy Program, Center for Childhood Cancer and Blood Disorders, Nationwide Children’s Hospital

Meisam Naeimi Kararoudi received his veterinary surgery degree from Iran. His Ph.D., jointly obtained through the University of Perugia and the University of Oxford, UK as an Erasmus scholar, focused on understanding the role of immune cells in cardiovascular diseases and also studied the role of miRNAs in AML using CRISPR/Cas9. During his PhD in Oxford and Italy, he received several International scholarships and grants. Being a veterinary surgeon, immunologist and genetic engineer brought him to Sweden where he used CRISPR/Cas9 to decrease rejection after transplantation of tissue engineered organs at Sahlgrenska University Hospital. During this period, he also worked in SciLifeLab as a visiting Post-Doc to use genetic modification in cancer-related projects. Currently, he works as a post-doc fellow in the Cell Therapy and Cancer Immunotherapy Program at Nationwide Children’s Hospital, Ohio. Under the supervision of Dr. Dean Lee, he developed a method to genetically modify the hard-to-transfect human Natural Killer cells using CRISPR and identified novel approaches to enhance their cytotoxicity and to target cancers via chimeric antigen receptors. Dr. Naeimi is also involved in developing new methods to detect expanded NK cells in-vivo using MRI.

Eric B. Kmiec, PhD, Director, Gene Editing Institute, Christiana Care Health System

Dr. Eric B. Kmiec is the Founder and Director of the Gene Editing Institute at the Helen F. Graham Cancer Center & Research Institute at Christiana Care Health System. His translational research focuses on gene editing, specifically using CRISPR, to catalyze genetic reengineering. Dr. Kmiec holds faculty appointments at the University of Delaware and the Wistar Institute. His laboratory has been continuously supported by the National Institute of Health grants with other funding from the National Science Foundation, the NIST-BIRD Foundation, and the American Cancer Society, among many others. Dr. Kmiec has been the primary thesis advisor for 18 PhD doctoral students as a full, tenured Professor at the University of Delaware and is the founder of two biotechnology companies. He serves on five editorial boards, has authored 160 peer-reviewed publications, as primary or senior author, and edited several books on gene therapy. Dr. Kmiec has 15 issued patents and has received service medals and awards, including the Genome Canada Lectureship, the Proudford Research Award and the PICC Life Science Innovator Award in 2018. Dr. Kmiec was named an Honorary Commander at Dover Air Force Base and an Eminent Scholar at Marshall University.

Troels Koch, PhD, Vice President & Head of Research, RNA Therapeutics, Roche

Troels Koch (TK) is Ph.D. in bio-organic chemistry and has worked in the area of nucleic acid chemistry and biology for 20 years. TK is a founder of several Biotech companies of which Exiqon A/S and Santaris Pharma A/S are the most commonly known. Santaris Pharma A/S has pioneered the proprietary LNA platform and contributed significantly to the field of RNA-therapeutics. Santaris Pharma A/S was in August 2014 acquired by Roche, and TK is presently VP & Head of Research at Roche Innovation Centre Copenhagen. The main responsibilities are to develop the fundamental understanding of the chemical and biological properties of LNA, improve and upgrade the LNA platform, refine the LNA antisense drug discovery process, and establish a RNA therapeutics drug pipeline in Roche.

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.

Michael N. Liebman, PhD, Managing Director, IPQ Analytics, LLC

Michael N. Liebman, Ph.D (theoretical chemistry and protein crystallography) 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.  He is on the Advisory Board of the International Society for Translational Medicine and on the Editorial Board for the Journal of Translational Medicine, for Clinical and Translational Medicine and for Molecular Medicine and Therapeutics, for Clinico-Economics and Outcomes Research and Biomedicine Hub, and the International Park for Translational Biomedicine (Shanghai).  He is a member of the IUPAC Division on Human Health’s Medicinal Chemistry subcommittee.  He has served on the External Advisory Board for the INBRE (NIH) program for the state of Delaware since 2000.  He is an Invited Professor at the Shanghai Center for Bioinformatics Technology and of the Chinese Academy of Sciences.  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. 

Marc Lemaitre, PhD, COO, Sirnaomics and ML_Consult

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.

Arthur Levin, PhD, Executive Vice President, Research and Development, Avidity Biosciences

Arthur A.Levin, Ph.D. is Avidity Biosciences Executive Vice President of Research and Development.  Dr. Levin has nearly 20 years of experience in the research and development of RNA-targeting therapeutics and 30 years of experience in the pharmaceutical industry. He has been involved in the development of more than 20 oligonucleotide therapeutics in clinical trials including the first approved antisense drugs. Prior to joining Avidity, Dr. Levin ran Research and Development at miRagen Therapeutics and was Chief Development Officer where he led the efforts on the first microRNA targeting therapeutic currently in Phase 2 clinical trials.

Before joining Santaris Pharma, Dr. Levin consulted for leading biotechnology and pharmaceutical companies, conducting research and development in RNA-based therapies such as mRNA, microRNA, and siRNA. Prior to consulting, Dr. Levin was Senior Vice President of Development at Ionis Pharmaceuticals, where he was responsible for the drug development of Ionis’ products across a range of therapeutic areas. His expertise was instrumental in advancing more than a dozen oligonucleotide drugs from basic research to clinical development in areas such as neuromuscular diseases, infectious diseases, metabolic disorders, cardiovascular disease and cancer. He joined Isis from Hoffmann-La Roche Inc. where he was Research Leader and made fundamental discoveries in retinoid research. Dr. Levin holds a Ph.D. in Toxicology from the University of Rochester School of Medicine and Dentistry, New York and a B.S. in Biology from Muhlenberg College. He completed his post-doctoral work at the Chemical Industry Institute of Toxicology in Research Triangle, North Carolina. He is the author of more than 60 papers and book chapters.

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.

Marie Wikstrom Lindholm, PhD, VP, Head of Technology Innovation, Silence Therapeutics Marie joined Silence

Therapeutics in December 2017 as Head of Innovation Technology, leading a skilled team focusing on fine-tuning design of GalNAc-conjugated siRNA and exploring extra-hepatocyte siRNA targeting. Before that she acquired oligonucleotide research expertise at Santaris Pharma , where after that the site transitioned to Roche Innovation Centre Copenhagen Marie was appointed Expert Scientist in Discovery Technology and Head of Targeted Delivery of oligonucleotide conjugates. Marie has authored >50 patents and scientific publications, many in the fields of antisense drug design, safety, and function.

Patrick Lu, President & CSO, Sirnaomics

Patrick Y. Lu, Ph.D., is the Founder and President of Sirnaomics, Inc., USA, and Founder and Chairman of Suzhou Sirnaomics Pharmaceuticals, Ltd. and Guangzhou Nanotides Pharmaceuticals, Co. Ltd.  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 40 issued and pending international and China patents. He has been an invited speaker in many international conferences throughout the world, and awarded a number of grants from NIH and the State of Maryland governments. Under his leadership Sirnaomics has developed novel siRNA therapeutic programs and initiated clinical studies for novel siRNA therapeutic candidate, STP705, for antifibrotic and anticancer indications, in both USA and China.

Thomas Madden, PhD, President & CEO, Acuitas Therapeutics

Dr. Madden obtained his BSc. and Ph.D. in Biochemistry from the University of London, U.K. He has held several senior academic and industry positions including Assistant Professor in Pharmacology at the University of British Columbia and Senior Director, Technology Development and Licensing at Tekmira Pharmaceuticals (formerly INEX Pharmaceuticals). At Tekmira Pharmaceuticals Dr. Madden was responsible for product development of several liposomal anticancer agents including Marqibo™ (liposomal vincristine), Alocrest™ (liposomal vinorelbine) and Brakiva™ (liposomal vinorelbine). All of these products were subsequently licensed to Talon Therapeutics. Dr. Madden founded Acuitas Therapeutics in February 2009 and through his role as President and CEO and has guided the company to a leadership position in the application of lipid nanoparticle (LNP) technology for delivery of nucleic acid therapeutics. This includes the development of the LNP formulation with MC3 which was subsequently used by Alnylam Pharmaceuticals in its Patisiran clinical candidate for the treatment of transthyretin amyloidosis. Patisiran (Onpattro™) successfully completed clinical evaluation and received marketing approval by the FDA and EMA in 2018. Following it development of delivery systems for RNAi therapeutics, Acuitas focused on applying its LNP technology to messenger RNA delivery. This research and development has resulted in the advancement of multiple mRNA therapeutics into clinical studies. Dr. Madden has over 60 publications in peer-reviewed journals relating to LNP technology and the development of pharmaceutical products, including recent publications in Nature.

Paul Manley, President & Principal Consultant, Orvieto Consulting

Paul F. Manley, has accumulated over 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.

Bianca Matthee, VP Manufacturing & Pharmaceutics, ProQR Therapeutics

Bianca Matthee holds a PharmD degree from the University of Utrecht. She started her career as formulation scientist at Organon BioSciences and has developed into a manufacturing leader in biotech/pharmaceutical R&D. She has >12 yrs of experience in Chemistry, Manufacturing & Control (CMC) of adenoviral vaccine products and oligonucleotide products, having worked for Merck (Schering Plough, Organon BioSciences) and Johnson & Johnson (Crucell) in different roles including formulation scientist, production manager and CMC Director. Since 2013 she heads up the CMC department at ProQR Therapeutics where she has built the CMC team and manufacturing capabilities from scratch including vendor management, in house facilities, interacting with regulatory authorities and delivering supplies for clinical programs. All of the clinical products she is involved developing are based on oligonucleotides developed for (ultra)orphan indications mainly in ophthalmology and dermatology and are in preclinical, early stage and late stage clinical trials.

Anton McCaffrey, PhD, Senior Director of Emerging Science and Innovation, TriLink BioTechnologies

Dr. McCaffrey is the Senior Director of Emerging Science and Innovation at TriLink BioTechnologies and an Adjunct Assistant Professor of Internal Medicine at the University of Iowa.  Dr. McCaffrey has over 25 years’ experience designing nucleic acid based therapeutics for antisense, RNA interference, aptamer and genome editing applications.  His research in this area began at an early antisense company called Genta in the late 80s.  Later he studied in vivo ribozyme kinetics at the University of Colorado at Boulder with Dr. Olke Uhlenbeck.  While a postdoctoral fellow at Stanford University in the laboratory of Dr. Mark Kay, Dr. McCaffrey conducted early in vivo proof of concept studies of RNA interference in mammals as well as studies on innate immunity. At UIOWA his research focused on genome editing with zinc-finger nucleases, microRNAs and RNA interference targeting hepatitis viruses.  Most recently, at TriLink BioTechnologies, he established a program manufacturing GMP grade mRNAs for genome editing, gene replacement and vaccines.  TriLink is the leading contract manufacturer of mRNAs and RNA single guide strands for the research and clinical/cGMP market.  His research interests include scalable mRNA manufacturing processes, chemical and sequencing modification of mRNA to reduce innate immune stimulation, the biology of 5’ caps and development of specialized assays for mRNA characterization.

Michael T. Migawa, PhD, Director, Medicinal Chemistry, Ionis Pharmaceuticals

Michael Migawa is currently a director of Medicinal Chemistry at Ionis Pharmaceuticals, in Carlsbad, California.  He has been with Ionis for 20 years.  In 1999, he earned his PhD in Organic Chemistry at the University of Michigan, Ann Arbor, under the direction of Professor Leroy B. Townsend.

Lubo Nechev, PhD, Vice President Process and Analytical Sciences, Alnylam Pharmaceuticals

Dr. Nechev leads the Process and Analytical Sciences Department at Alnylam Pharmaceuticals. He has joined Alnylam in 2004 and has since be responsible for the development, optimization and transfer of the processes used in the manufacture and formulation of siRNAs as well as all the technical CMC aspects of the drug substance and drug product characterization, manufacture and release. Prior to joining Alnylam, Dr. Nechev worked at Ribozyme Pharmaceuticals (RPI) and the Nucleic Acid Synthesis unit of Transgenomic Inc. He received his Ph.D. degree in Organic Chemistry from St. Kl. Ohridski University, Sofia, Bulgaria and completed his post doctoral training at the Department of Chemistry and Center in Molecular Toxicology at Vanderbilt University, Nashville, Tennessee.

Norbert Pardi, PhD, Assistant Professor of Medicine, University of Pennsylvania

Dr. Norbert Pardi is an Assistant Professor at the Perelman School of Medicine of the University of Pennsylvania. His research interest is the development of mRNA-based therapeutics with particular focus on new generation vaccines. He explored the development of a novel protein delivery platform, nucleoside-modified mRNA in lipid nanoparticles (LNPs) and used it to generate highly efficacious vaccines targeting various pathogens.

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.

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital

Khalid Shah is an Associate Professor at Harvard Medical School and the Director of the Center for Stem Cell Therapeutics and Imaging at Brigham and Women’s Hospital (BWH). He is also the Vice Chair of Research for the Department of Neurosurgery at BWH and a Principal Faculty at Harvard Stem Cell Institute in Boston. Since his move to BWH, he has started a joint Center of Excellence in Biomedicine with KACST and is also directing the new Center. Dr. Shah and his team have pioneered major developments in the stem cell therapy field, successfully developing experimental models to understand basic cancer biology and therapeutic stem cells for cancer. These studies have been published in a number of very high impact journals like Nature Neuroscience, PNAS, Nature Reviews Cancer, JNCI, Stem Cells and Lancet Oncology. Previously, Dr. Shah's stem cell work has caught the attention in the public domain and as such it has been highlighted in the media world-wide including features on BBC and CNN. Recently, Dr. Shah’s laboratory has reverse engineered cancer cells using CRISPR/Cas9 technology and utilized them as therapeutics to treat cancer. This work was published in journal Science Translation Medicine and highlighted world-wide including features on Scientific American, New York Times and Fox news. Dr. Shah holds current positions on numerous councils, advisory and editorial boards in the fields of stem cell therapy and oncology. In an effort to translate the exciting therapies develped in his laboratory into clinics, he has recently founded biotech company, AMASA Technologies Inc. whose main objective is the clinical translation of therapeutic stem cells in cancer patients.

Clifford Steer, MD, Professor of Medicine and, Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School

Clifford J. Steer is a Professor of Medicine and Genetics, Cell Biology, and Development and a senior investigator at the University of Minnesota, Minneapolis, MN.  He has been active in the field of liver research for more than four decades.  In that capacity, he has been a long-standing member of several National Institutes of Health Study Sections.  He has been co-editor of a major scientific journal in liver diseases and presently serves on the editorial boards of three journals.  He has published his work in top-ranking scientific journals and numerous textbooks.  Steer’s areas of research over the last decade have focused on gene therapy, liver regeneration, neurodegenerative disorders and microRNA regulation of gene function.  He has published over 290 scientific articles; and has organized and chaired many national and international scientific conferences.  He was elected into the American Society for Clinical Investigation in 1991; and in 2014 was made an inaugural Fellow of the American Association for the Study of Liver Diseases.  His work has been written up in newspapers around the world, Time magazine, and in 1998 his work was featured in the Village Voice.

David Tabatadze, PhD, President, ZATA Pharmaceuticals

In 2009, Dr. Tabatadze cofounded ZATA in partnership with Dr. Paul Zamecnik (1912-2009) and has been serving as the President and CEO since then. At ZATA, he led the development of the novel nucleic acid platform technology enabling the synthesis new generation oligonucleotides (ON) with custom-designed properties based on the incorporation various branched chemical groups on the internucleoside phosphates during the automated synthesis. ZATA’s ONs (ZONs) are applicable to all currently known oligotherapy approaches including antisense and CRISPR. In recent past, he has also consulted several medical device companies in the development and clearance of commercially available light based medical devices. His thirty-plus years of R&D experience includes drug candidate and diagnostic probes development, 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 Worcester Foundation for BioMedical Research (Shrewsbury, MA). He has also co-published over three dozen of peer-reviewed papers, patent applications, and patents.
At ZATA he has established business and collaborative relationships with several biotech and academic institutions locally and internationally.   

Yu-Hsuan Tsai, PhD, Lecturer in Chemical Biology, School of Chemistry, Cardiff University

Yu-Hsuan Tsai received the BSc from National Taiwan University in 2006, MSc from Swiss Federal Institute of Technology (ETH) Zurich in 2008, and Dr. rer. nat. from Freie Universität Berlin in 2012. Before joining Cardiff University in 2015, he had worked for Prof Shih-Hsiung Wu (Academia Sinica, Taiwan), Prof Peter H Seeberger (Max Planck Institute of Colloids and Interfaces, Germany) and Prof Jason W Chine (Medical Research Council Laboratory of Molecular Biology, UK). His research focuses on the application of genetic code expansion to control protein function and unravel biological challenges. He was awarded Dow’s Chemistry Award and Chinese Chemical Society Award, as well as fellowships from Novartis, Medical Research Council, British Science Association and EMBO. (https://tsai-lab.org)

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.

Søren Warming, PhD, Senior Scientist, Genentech Inc., a member of the Roche group

Søren obtained his Ph.D. in molecular genetics and molecular biology from the University of Aarhus, Denmark. He then joined the lab of Drs. Neal Copeland and Nancy Jenkins at the National Cancer Institute, Maryland, USA, as a postdoctoral fellow. After completing his postdoc in 2006, Søren joined Genentech where he leads a group that develops and implements cutting-edge tools for genome engineering in rodents as well as for cells in culture. His group is responsible for the design and development of all in-house genetically engineered rodent models at Genentech used to facilitate drug discovery and to help answer important basic scientific questions.