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DIA/FDA Oligonucleotide-Based Therapeutics Conference

Convening industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development.


Speakers

  • Xuan  Chi, MD, PhD

    Xuan Chi, MD, PhD

    • Pharmacologist/Toxicology Reviewer, Div. of Cardiovascular & Renal Products, OND
    • FDA, United States

    Dr. Chi is a Pharmacology/Toxicology reviewer in the Division of Cardiovascular and Renal Products at the Office of New Drugs, CDER FDA. Prior to that, she was a clinical analyst and SME in the Federal Healthcare Practice at Deloitte Consulting LLP and her projects were focused on post-marketing drug safety and review modernization effort at OGD. She had also worked as a staff fellow at Office of Blood Review and Research, CBER FDA. She is specialized in analyzing data from toxicological, pharmacological and clinical studies of original NDAs, BLAs, INDs, and 510(k)s. She had Ph.D. in genetics and had postdoctoral training in molecular genetics and pathology.

  • Scott  Henry, PhD

    Scott Henry, PhD

    • Vice President, Nonclinical Development
    • Ionis Pharmaceuticals, Inc., United States

    Dr. Henry received a PhD in Biochemistry form North Dakota State University. He was a post-doc fellow at Parke Davis, Ann Arbor MI, depart. of toxicology. He joined Isis Pharmaceuticals, Inc. as a Sr Scientist in toxicology. He helped characterized and studied mechanisms of various toxicities e.g. the effects of oligonucleotide treatment on clotting time prolongation, alternative complement pathway activation, proinflammatory effects in rodents, platelet alterations and the effects related to the accumulation of oligonucleotide in kidney. As VP of Non-Clinical Development he has participated in the development of ~8 different phosphorothioate oligodeoxynucleotides and 30+ different 2’-MOE modified phosphorothioate oligonucleotides.

  • Aimee L. Jackson, PhD

    Aimee L. Jackson, PhD

    • Senior Director of Research
    • miRagen Therapeutics, United States

    Aimee has been working in the field of RNA interference and microRNAs for >15 years and has authored/co-authored >20 publications. She received her PhD from Univ of Colorado Health Science Cntr and performed post-doc research at Univ of Washington. Aimee joined Rosetta Inpharmatics/Merck, where she established the use of RNAi combined with expression profiling technologies for target identification, target validation, elucidation of drug mechanism-of-action, and patient stratification. She investigated the therapeutic application of siRNAs. Aimee leads the discovery/development of new therapeutic targets in diverse disease indications, pioneering the implementation of translational biomarkers for mechanistic proof-of-concept in patients.

  • Arthur M. Krieg, MD

    Arthur M. Krieg, MD

    • President and CEO
    • Checkmate Pharmaceuticals, United States

    Art Krieg, MD is CSO of Pfizer’s Research Technology Center in Cambridge MA. He was the founding editor of Antisense and Nucleic Acid Drug Development and is a co-founder, of the Oligonucleotide Therapeutic Society. Dr. Krieg has published more than 200 scientific papers.

  • Arthur A. Levin, PhD

    Arthur A. Levin, PhD

    • Executive Vice President, Research and Development
    • Avidity Biosciences , United States

  • Saraswathy V. Nochur

    Saraswathy V. Nochur

    • Senior Vice President, Regulatory Affairs & QA
    • Alnylam Pharmaceuticals, Inc., United States

    In addition to her role in Regulatory Affairs, she also served as Head of Quality Assurance and was the Program Leader for two development programs at Alnylam. Dr. Nochur has 25 years of experience in the areas of Product Development and Regulatory Affairs including early and late stage clinical programs as well as drug and device experience. Prior to Alnylam, she held the position of VPt, Regulatory Affairs at The Medicines Company. She also served in BioTrack, Inc. and DynaGen earlier in her career. Dr. Nochur received her MS in Microbiology from the Univ of Bombay in India, and her PhD in Biochemical Engineering at MIT.

  • Andrew  Teasdale, PhD

    Andrew Teasdale, PhD

    • Principal Scientist, Chair of Impurities Advisory Group
    • AstraZeneca , United Kingdom

    Andrew Teasdale PhD chairs the AstraZeneca’s Impurity Advisory Group. He has published widely in the field of impurties including areas such as mutagenic impurities, metals and extractables and leachables. He has also led a number of industry expert groups; these include both safety and quality groups within Pharmaceutical Research and Manufacturers of America (PhRMA), European Federation of Pharmaceutical Industries and Associations (EFPIA), Product Quality Research Institute (PQRI) and the Extractables and Leachables safety Information exchange (ELSIE). Specifically in the field of Oligonucleotides he leads the Oligonucleotide Safety Working Group Impurities Sub-group

  • James D. Thompson, PhD

    James D. Thompson, PhD

    • Head, CMC Project Management
    • Moderna Therapeutics, United States

    Dr. Thompson is currently Head of CMC Project Management at Moderna Therapeutics. Prior to joining Moderna, Dr. Thompson was Vice President, Development at Quark Pharmaceuticals, Inc. Prior to Quark, Dr. Thompson was Director of Research & Development at Genta, Inc., and before this was Director, Biology Research at Ribozyme Pharmaceuticals/Sirna Therapeutics.

  • Kim  Tyndall

    Kim Tyndall

    • Director, CMC Regulatory Affairs
    • GlaxoSmithKline, United States

    Kim is a Director of CMC Regulatory Affairs at GlaxoSmithKline (GSK). She has been at GSK since 1981 in a variety of chemistry and CMC Regulatory roles. Her primary responsibilities include CMC Regulatory oversight of the oligonucleotide platforms and alliances, as well as due diligence on oligonucleotide opportunities. Kim has held multiple positions in Analytical Development and CMC Regulatory Affairs including Post Approval and Biopharm Regulatory Affairs. Kim received a BA in Biology from East Carolina University in 1981.

  • Fran  Wincott, PhD

    Fran Wincott, PhD

    • President
    • Wincott & Associates LLC, United States

    Dr. Fran Wincott is President of Wincott & Associates, LLC, a consulting firm focused on providing assistance in the area of oligonucleotide manufacturing and development. Prior to founding Wincott & Associates, Dr. Wincott was Vice President of Oligonucleotide Manufacturing & Development at Eyetech Pharmaceuticals, Inc. (2002-2005). Prior to joining Eyetech Pharmaceuticals, Dr. Wincott served as Senior Director of Manufacturing Operations at Ribozyme Pharmaceuticals, Inc. From 1989-1993 she worked as a scientist at Merck, Inc. and Cortech, Inc. Dr. Wincott received her B.A. in Chemistry at the University of Pennsylvania in 1984 and a Ph.D. in Organic Chemistry in 1989 from Yale University

  • Patrik  Andersson, PhD

    Patrik Andersson, PhD

    • Principal Scientist, Discovery Safety Specialist
    • AstraZeneca R&D, Sweden

    I received my PhD in toxicology from Karolinska Institutet, Stockholm in 2003. Joined AstraZeneca R&D in Gothenburg in 2004 as a toxicologist supporting Cardiovascular and Metabolic drug projects in the Discovery phase. Since 2012 focusing on nucleotide drugs, including oligonucleotides and mRNA therapeutics. Currently leading the preclinical safety activities for oligonucleotides and targeted drug delivery in the AstraZeneca strategic collaboration with Ionis Pharmaceuticals in the Cardiovascular and Metabolic therapeutic area.

  • Gerald  Cox, MD, PhD

    Gerald Cox, MD, PhD

    • Chief Medical Officer
    • Editas Medicine, Inc., United States

    Dr. Gerry Cox is Chief Medical Officer at Editas Medicine, a leading genomic medicines company. He was formerly Vice President of Clinical Development for Rare Diseases at Sanofi Genzyme, where for 16 years he oversaw the clinical development programs for several lysosomal storage disorders, including three drug approvals. Dr. Cox is a board-certified clinical geneticist and pediatrician who sees patients part-time at Boston Children’s Hospital, where he was previously on staff and completed his pediatrics and genetics training. He is an instructor in pediatrics at Harvard Medical School. Dr. Cox received a BA in biology from Harvard College in 1980 and an MD, PhD from UCSD in 1989.

  • Samantha  Gao-Sheridan, PhD

    Samantha Gao-Sheridan, PhD

    • Senior Director, Regulatory Affairs CMC
    • Alnylam Pharmaceuticals, United States

    Dr. Gao-Sheridan, Senior Director/Head Regulatory Affairs CMC at Alnylam Pharmaceuticals, specializes in leading regulatory CMC strategy through development, registration, and life cycle management of innovative biological and pharmaceutical therapies for rare diseases and unmet medical needs. She holds a Ph.D. in Biochemistry and Molecular Biology, M.S. in Toxicology, and B.S. in Medicine.

  • Sudip  Parikh, PhD

    Sudip Parikh, PhD

    • Senior Vice President and Managing Director, DIA Americas
    • DIA, United States

  • Mohan K. Sapru, PhD, MS

    Mohan K. Sapru, PhD, MS

    • CMC Lead, Office of Pharmaceutical Quality, CDER
    • FDA, United States

    Mohan Sapru, Ph.D., is a CMC Lead, and Application Technical Lead; and Member, Emerging Technology Team, Office of Pharmaceutical Products; FDA. Dr. Sapru is involved with leading, managing, and integrating CMC team reviews and review teams. As Emerging Technology Team project lead, Dr. Sapru is involved with guiding pharmaceutical innovation. Prior to joining the FDA, he served as faculty at Northwestern University, Chicago where his research focused on targeted drug delivery, oligonucleotide therapeutics, and RNAi-based allele-specific gene therapy. He holds several US patents, and has authored a number of research publications in prestigious peer-reviewed journals.

  • G. Susan Srivatsa, PhD

    G. Susan Srivatsa, PhD

    • President
    • Elixin Pharma, United States

    Susan is Founder and President of ElixinPharma, a scientific consulting firm dedicated to assisting emerging pharmaceutical companies with their chemical development needs and writing/defense of CMC sections of regulatory applications, for oligonucleotide-based therapeutics (antisense, siRNA, aptamers, etc.) in simple (parenteral) and complex (topical, oral, pulmonary, liposomes, nanoparticles, etc.) dosage forms. Since 1999, Susan has provided strategic regulatory leadership in the CMC area for a number of leading companies in various stages of clinical development. Susan received a Ph.D. in Analytical Chemistry from the University of California in 1985 followed by post-doctoral research in NMR spectroscopy.

  • Peter  Wijngaard, PhD

    Peter Wijngaard, PhD

    • Executive Vice President and Chief Development Officer
    • The Medicines Company, United States

    Peter is Executive Vice President and Chief Development Officer at The Medicines Company. Peter has extensive experience in the areas of global project leadership, business development, medical affairs, and pharmaceutical marketing. Previously, Peter led European Medical Affairs and Development at ViroPharma and has held various positions at Hoffmann-La Roche including International Medical Manager and Lifecycle Leader for the transplantation portfolio, as well as Global Alliance Director managing the Genentech alliance.

  • James  Wild, PhD

    James Wild, PhD

    • Pharmacologist, CDER
    • FDA, United States

    James Wild received a MS and PhD in Pharmacology and Tox at the Univ of California, Davis. Areas of study included idiopathic pulmonary fibrosis and characterization of a novel, ryanodine-sensitive receptor in the lung. Subsequently he completed two postdoctoral fellowships specializing in asthma research. In later career positions, James conducted discovery pulmonary disease research at EpiGenesis Pharmaceuticals Schering-Plough Research Institute, and Johnson and Johnson PRDUS. Areas of interest include anti-infective drugs, oligonucleotide therapies, pulmonary research, and drug regulation.

  • Tal  Zaks, MD, PhD

    Tal Zaks, MD, PhD

    • Chief Medical Officer
    • Moderna Therapeutics, United States

    As chief medical officer, Dr. Tal Zaks oversees preclinical development, clinical development and regulatory affairs across Moderna and its ventures. Prior to joining Moderna, Dr. Zaks was senior vice president and head of Global Oncology at Sanofi, where he was responsible for all aspects of oncology drug discovery, development and commercialization. Dr. Zaks received his M.D. and Ph.D. from the Ben Gurion University in Israel and conducted post-doctoral research at the U.S. National Institutes of Health. He completed his clinical training in internal medicine at Temple University Hospital followed by a fellowship in medical oncology at the University of Pennsylvania.

  • Firoz  Antia, PhD

    Firoz Antia, PhD

    • Director, Technical Development
    • Biogen, United States

    Firoz D. Antia is currently Director, Antisense Oligonucleotide Process Development and Manufacturing, at Biogen, where he has been a key member of the nusinersen global filing team. In addition to recent work on oligonucleotides, Dr. Antia has extensive CMC drug development experience with peptides, small molecules and biologics in previous positions at Palatin Technologies, Merck, J&J and Sandoz. He holds a Ph.D. in Chemical Engineering from Yale University.

  • Elena  Braithwaite, PhD

    Elena Braithwaite, PhD

    • Toxicologist
    • FDA, United States

    Dr. Elena Braithwaite is a toxicologist at the US Food and Drug Administration and a Diplomate of the American Board of Toxicology. She has a broad background in various aspects of basic research including DNA repair, mutagenesis and signal transduction.

  • Daniel  Capaldi, PhD

    Daniel Capaldi, PhD

    • Vice President, Analytical and Process Development
    • Ionis Pharmaceuticals, Inc., United States

    Daniel received a B.Sc in chemistry and biology and a Ph. D in chemistry from King’s College, London. In March 1996, Daniel joined Ionis Pharmaceuticals, Inc. as a process chemist where he spent four years working on process improvements in large-scale oligonucleotide synthesis. In his current position as Vice President, Analytical and Process Development, Daniel is contributing to the development of antisense therapeutics and has responsibility for a variety of process chemistry and CMC functions including supply chain improvements and process optimization, analytical method development and validation, release and stability testing and impurity characterization

  • Bret  Coldren, PhD

    Bret Coldren, PhD

    • Director, Pharmaceutical Development
    • Ionis Pharmaceuticals , United States

    Bret Coldren is Director of Pharmaceutical Development at Ionis Pharmaceuticals since 2010, where he supports oligonucleotide drug product development and manufacturing. From 2005-2010, he worked at Bayer Biologics and J&J Vision Care on biologic-device and drug-device combination products for pulmonary and ophthalmic treatments, respectively. From 2001-2005, Bret was cofounder of an NSF SBIR-funded start-up company developing lipid nanoparticle delivery technologies for oligos and small molecules. He holds BS and PhD degrees in Chemical Engineering from Stanford University and UC Santa Barbara.

  • Jeffery  Engelhardt, DVM, PhD

    Jeffery Engelhardt, DVM, PhD

    • Vice President, Pathology and Nonclinical Drug Safety
    • Ionis Pharmaceuticals, Inc., United States

    Dr. Engelhardt holds DVM and PhD degrees from Purdue University and is a Diplomate of the American College of Veterinary Pathology. He is also a Fellow in the International Academy of Toxicologic Pathology and a Fellow of the Academy of Toxicological Sciences, a member of the STP Vascular Injury Working Group for Biotherapeutics and Antisense Oligonucleotides, the ESTP Expert Panel on Adversity of Lysosomal Accumulation, and a member of the DIA Oligonucleotide Safety Working Group. He has spent his career studying the toxicopathology of pharmaceutical and biotechnology drug products and is presently Vice President of Pathology and Nonclinical Drug Development for Ionis Pharmaceuticals studying antisense oligonucleotide therapeutics.

  • Lois M Freed, PhD

    Lois M Freed, PhD

    • Supervisory Pharmacologist, CDER
    • FDA, United States

  • Yoko  Hirabayashi, MD, PhD

    Yoko Hirabayashi, MD, PhD

    • Div of Cellular Molecular Toxicology Center, Biological Safety & Research
    • National Institute of Health Sciences (NIHS), Japan

    Providing leadership of the Japanese research group related to ICH S6(R1) addressing issue on preclinical safety evaluation of nucleotide therapeutics based on the principle of the ICH S6(R1) guideline which may be applicable to not only biopharmaceuticals but also materials expected to exert their effect in a species-specific manner such as oligonucleotides. Served as the topic leader representing Ministry of Health Labor and Welfare (MHLW), Japan for ICH S6(R1) expert working group from 2008 to 2011. Serving as an external expert in toxicology to evaluate new drug applications of Pharmaceuticals and Medical Devices Agency (PMDA) since 2010.

  • Imran  Khan, PhD

    Imran Khan, PhD

    • Pharmacologist, OMPT, OND, ODEI, DPP, CDER
    • FDA, United States

  • Shwu-Luan  Lee, PhD

    Shwu-Luan Lee, PhD

    • Pharmacologist, Office of New Drug, CDER
    • FDA, United States

  • Ramachandra G Naik, MD

    Ramachandra G Naik, MD

    • Primary Reviewer, Regulatory Project Manager, OVRR, CBER
    • FDA, United States

    Chemist/Regulatory Project Manager (CBER/FDA) - Review INDs, BLAs and related regulatory submission; Serve as scientific and administrative contact and coordinate meetings with pharmaceutical companies and other sponsors of experimental vaccines. Research Biologist (WRAIR) - Initiate and conduct research in major DOD projects (evaluate the usage of human BChE and other protein bioscavengers as a prophylactic or treatment drugs that can detoxify nerve agents). Postdoctoral fellow (Georgetown University) - Determination of detailed structure of P. falciparum GPIs and study their role/involvement in the malaria pathogenesis. Ph.D. in Biochemistry - Characterization of fungal lectins and study their role in host-parasite interactions.

  • Emily J. Place, PhD, MPH

    Emily J. Place, PhD, MPH

    • Pharmacologist, Office of New Drugs, CDER
    • FDA, United States

    Emily is a co-chair of FDA’s Pharmacology/Tox Oligonucleotide Subcommittee. She received a BS in Biology from State Univ of New York in 01’, a PhD in Cell Biology from Univ of Connecticut in 06’. She joined the Research Enhancement Award Program as an Assoc Investigator at San Francisco VA Medical Cntr where her research focused on miRNA dysregulation in prostate cancer. In 08’ she joined the Depart of Hematology at Stanford Univ School of Medicine as a postdoc fellow. In 2012 she completed her MPH in Epidemiology from Univ of California at Berkeley. Emily completed a fellowship at NCI/NIH in the Laboratory of Human Carcinogenesis. Research involved examining the role of extracellular plant small RNA communication in human carcinogenesis.

  • Ramesh  Raghavachari, PhD

    Ramesh Raghavachari, PhD

    • Chief, Branch I, DPMA1, OLDP, OPQ,CDER
    • FDA, United States

  • Sree  Rayavarapu, DVM, PhD

    Sree Rayavarapu, DVM, PhD

    • Toxicologist
    • FDA, United States

  • René  Thürmer, PhD

    René Thürmer, PhD

    • Deputy Head of the Unit Pharmaceutical Biotechnology BfArM
    • Federal Institute for Drugs and Medical Devices, Germany

    Dr. René Thürmer received his diploma in chemistry and his Ph.D. in biochemistry from the University of Tübingen. He joined the BfArM (Federal Institute for Drugs and Medical Devices, Bonn, Germany) in 2000. He currently serves as a CMC reviewer and is Deputy Head of the Unit Pharmaceutical Biotechnology. His experience is in the field of formulation, manufacture and control of medicinal products, in particular in the field of peptides, proteins, liposomes, sustained release polymer drug products, depot formulations, polymer-conjugated drug products, natural and synthetic surfactants, nanomedicine and others. His special focus lies on oligonucleotide preparations.

  • Ronald L. Wange, PhD

    Ronald L. Wange, PhD

    • Pharmacology & Toxicology Reviewer, Div. of Metabolism & Endocrinology Products
    • FDA, United States

    Dr. Wange is a Supervisory Pharmacology/Toxicology Reviewer within the Office of New Drugs in CDER at the FDA, and has over 12 years of experience reviewing small molecule drugs, biotherapeutic proteins and oligonucleotide-based therapeutics. He is a founding member of OND’s Pharmacology/Toxicology Oligonucleotide Subcommittee, which considers issues specifically related to the safety review of oligonucleotide-based therapeutics. Dr. Wange has a bachelor’s degree in Biochemistry from the University of California, and a Ph.D. in Pharmacology from Vanderbilt University. Prior to joining the FDA, Dr. Wange headed the T-Lymphocyte Signaling Unit at the National Institute on Aging at the NIH.

  • Timothy J.N. Watson, PhD

    Timothy J.N. Watson, PhD

    • Senior Director, CMC Advisory Office
    • Pfizer Inc, United States

    Tim was one of the PhRMA expert working group (EWG) members on the ICHQ11 regulatory guidance document for drug substance, and the current Rapporteur for the ICHQ11 Starting Material IWG. He is also served on the ICHQ7 IWG Q&A team, ICHQ3C EWG, and supporting many other ICH efforts (such as Q12). Tim’s primary responsibility at Pfizer is to collaborate with Regulatory CMC team leaders, Co-Development teams (technical teams), and Pfizer Global Supply (PGS) teams from Small Molecule (API and DP), Biotherapeutics, Vaccines, etc. on a number of regulatory and technical issues.

  • Barbara  Wilcox, PhD

    Barbara Wilcox, PhD

    • Pharmacologist, OMPT, ODEI, DNP, OND, CDER
    • FDA, United States

    Dr. Wilcox currently serves as a pharmacologist in the Division of Neurology Products, CDER, FDA. She received her Ph.D from the Univ. of Minnesota. She served as CMC reviewer for biological therapeutics from 1993-2003 and since as a pharmacologist for review of biologics for neurology indications.

  • Iwen  Wu, PhD

    Iwen Wu, PhD

    • Branch Chief of Pharmacology and Toxicology 2, OTAT, CBER
    • FDA, United States

    Iwen Wu is currently Branch Chief of Pharmacology/Toxicology Branch 2 in the Office of Tissues and Advanced Therapies at CBER where she was previously a Pharmacology/Toxicology Team Lead and Reviewer. Prior to joining CBER, she was a lead reviewer in the Renal Devices Branch at the Center for Devices and Radiological Health. She joined the FDA as a Commissioner’s Fellow after completing her Ph.D. in Biomedical Engineering at Johns Hopkins University.

  • Timothy  Yu

    Timothy Yu

    • Assistant Professor, Harvard Medical School
    • Division of Genetics and Genomics, Boston Children’s Hospital, United States

  • Sanjay  Bhanot, MD, PhD

    Sanjay Bhanot, MD, PhD

    • Vice President, Metabolic Diseases, Research & Development
    • Ionis Pharmaceuticals, Inc., United States

  • Ashley  Boam, MS

    Ashley Boam, MS

    • Director, Office of Policy for Pharmaceutical Quality, OPQ, CDER
    • FDA, United States

    Ashley serves as Director of the Office of Policy for Pharmaceutical Quality in the Center for Drug Evaluation and Research at FDA. OPPQ is responsible for developing and clearly communicating science- and risk-based policies & standards related to drug product quality, including application assessment & inspection. Prior to joining CDER, Ashley spent nearly 20 years in the Center for Devices and Radiological Health. She holds bachelor's and master's degrees in biomedical engineering.

  • Philip  Brooks, PhD

    Philip Brooks, PhD

    • Program Director, Office of Rare Diseases Research & Div of Clinical Innovation
    • NIH, National Center for Advancing Translational Sciences (NCATS), United States

    https://ncats.nih.gov/staff/pjbrooks

  • Wouter  Driessen, PhD, MS

    Wouter Driessen, PhD, MS

    • DMPK Project Leader
    • F. Hoffmann-La Roche AG, Switzerland

  • Wildon  Farwell, MD

    Wildon Farwell, MD

    • Senior Director, Clinical Development
    • Biogen , United States

  • Kendall  Frazier, DVM, PhD

    Kendall Frazier, DVM, PhD

    • Director,Cellular and Molecular Pathology
    • GlaxoSmithKline, United States

    Dr. Ken Frazier has been with GSK for~15 years. He has a BS in biology from Wichita State, a DVM from Kansas State College of Vet Medicine, a PhD in molecular biology from the Univ of Miami and is board certified in both Pathology/Toxicology and is a fellow of the IATP He completed a residency in comparative pathology. He has extensive experience in the toxicology and pathology of antisense oligonucleotides. He lectures widely for the Soc of Toxicologic Pathology, American College of Veterinary Pathologists and other organizations and served on editorial boards for multiple journals.

  • Vidhya  Gopalakrishnan, PhD

    Vidhya Gopalakrishnan, PhD

    • Senior Vice President, Pharmaceutical Development
    • Quark Pharmaceuticals, Inc, United States

  • Joseph  E. Italiano, PhD

    Joseph E. Italiano, PhD

    • Director Cellular and Molecular Pathology, Division of Hematology
    • Brigham and Women’s Hospital, United States

    Dr. Joseph E. Italiano Jr. is Associate Professor of Medicine at Brigham and Women's Hospital and Harvard Medical School, Boston, USA. He is also an Assistant Professor of Medicine in the Department of Surgery at Boston Children's Hospital. Dr. Italiano is a Co-founder of Platelet BioGenesis, a biotechnology company that aims to make in vitro platelets. Dr Italiano's scientific interests have long focused on megakaryocyte and platelet biology. His early research focused on using live cell microscopy to identify the basic principles of platelet production. More recently his research efforts have focused on mechanisms of drug-induced thrombocytopenia and understanding how platelets function beyond hemostasis.

  • Matthias  Kretschmer, PhD

    Matthias Kretschmer, PhD

    • Senior Director, Analytical Development
    • Alnylam Pharmaceuticals, United States

  • Kazushige  Maki, DVM

    Kazushige Maki, DVM

    • Senior Scientist, Toxicology
    • Pharmaceuticals and Medical Devices Agency (PMDA), Japan

    Dr. Kazushige Maki is a Senior Scientist for Toxicology of the Pharmaceuticals and Medical Devices Agency (PMDA), Japan. He graduated from Faculty of Veterinary Medicine, Hokkaido University, and received his Ph.D in medical science from University of Tokyo. He worked at Tokyo Metropolitan Institute, as a researcher; at Tokyo Medical and Dental University, as an assistant; at Massachusetts General Hospital, as a postdoctoral fellow; Kyoto University, as a lecturer. In 2008, he started his review for new drugs in PMDA. Currently he is responsible for the toxicological review and consultation in the new drug review office in PMDA.

  • Huw M. Nash, PhD

    Huw M. Nash, PhD

    • Chief Executive Officer
    • Stoke Therapeutics Inc., United States

  • Quynh Nhu  Nguyen, MS

    Quynh Nhu Nguyen, MS

    • Associate Director for Human Factors, DMEPA, CDER
    • FDA, United States

  • Amy  Rhoden Smith, PhD

    Amy Rhoden Smith, PhD

    • Principal Scientist
    • Intellia, United States

    Amy Rhoden Smith is a Principal Scientist at Intellia Therapeutics leading a platform group focused on RNA for CRISPR/Cas9 applications, ranging from development and optimization of Cas9 mRNA to synthetic guide RNA. Prior to her joining Intellia in June 2015, Amy was at Moderna Therapeutics where she developed chemical and biochemical hybrid approaches to modify mRNA. Amy’s research has focused on using chemistry to understand and solve biological problems, and she continues to do so in her role at Intellia. She received her B.S. in Chemistry from College of Charleston, and completed her Ph.D. in Organic Chemistry at the University of Texas at Austin.

  • Christoph  Rosenbohm, PhD, MBA

    Christoph Rosenbohm, PhD, MBA

    • Vice President, Head of Discovery Operations
    • Roche Innovation Center Copenhagen, Denmark

    As VP and Head of Discovery Operations, RTR Christoph is responsible for leading the Oligonucleotides synthesis, Pharmacology, and Screening Operations teams and as a member of the RTR-LT he participates in defining and setting the strategy for RTR.

  • John A. Vest

    John A. Vest

    • Senior Director, Clinical Development
    • Alnylam Pharmaceuticals, Inc., United States

  • John L. Berk, MD

    John L. Berk, MD

    • Associate Professor of Medicine, School of Medicine
    • Boston University, United States

  • Andreas  Dieckmann, PhD

    Andreas Dieckmann, PhD

    • Senior Principal Scientist
    • F. Hoffmann-La Roche , Switzerland

    Dr. Andreas Dieckmann is a Senior Principal Scientist at Hoffmann-La Roche (Basel, Switzerland) focusing on safety and efficacy assessments of antisense oligonucleotides.. He joined Roche in 2013 from Takeda Pharmaceuticals International GmbH (Zurich, Switzerland) as a Fellow Translational Biomarker Expert. Prior to this position, Andreas worked as a Senior Biomarker Specialist at Nycomed (Konstanz, Germany). His previous experience before joining Nycomed included a position as Head of Preclinical Development at Novosom AG (Halle/Saale, Germany) and Group Leader at InDex Pharmaceuticals AB (Stockholm, Sweden). Andreas obtained his PhD in Molecular Biology from the University of Bochum (Germany).

  • Corrie L Gallant-Behm, PhD

    Corrie L Gallant-Behm, PhD

    • Research Scientist III
    • Miragen Therapeutics, Inc., United States

  • Takahiro  Nakazawa

    Takahiro Nakazawa

    • CSO
    • AnGes, Inc., Japan

    Dr Nakazawa was a topic leader representing JPMA at Expert Working Group on ICH S6(R1) guideline. He is also a member of Japanese Working Group on biopharmaceuticals and oligonucleotide therapeutics. He, as Chief Scientific Officer, oversees R&D of gene therapy, oligonucleotide therapeutics and DNA vaccines at AnGes Inc.

  • Lubomir  Nechev, PhD

    Lubomir Nechev, PhD

    • Vice President, Process Sciences
    • Alnylam Pharmaceuticals, United States

  •   Panel Discussion

    Panel Discussion

    • All Session Speakers, United States

  • Joshua  Rosenthal, PhD

    Joshua Rosenthal, PhD

    • Senior Scientist
    • Marine Biological Laboratory, United States

    I am a Senior Scientist at the Marine Biological Laboratory in Woods Hole, MA. My research examines RNA editing through adenosine deamination, a process that occurs in all multicellular animals. Work in my lab focuses on two related areas. The first is RNA editing in cephalopods, because they use this process to rewrite their neural transcriptome at unprecedented levels. The second is how we can redirect RNA editing enzymes to mRNA targets of our choosing. By studying diverse organisms, we gain insight into how to repurpose RNA editing for therapeutic advantage.

  • Louis  St.L. O'Dea

    Louis St.L. O'Dea

    • Executive Vice President, Chief Medical Officer and Head, Regulatory Affairs
    • AKCEA Therapeutics, United States

  • Sarah Beach Voytek

    Sarah Beach Voytek

    • Principal Scientist
    • Novartis Institutes For Biomedical Research, Inc., United States

    Sarah Voytek is a Senior Investigator in Discovery and Investigative Safety at Novartis Institutes for BioMedical Research in Cambridge, MA. Sarah heads a laboratory employing molecular and cell biology techniques to study mechanisms of toxicologic drug effects. She has led the preclinical safety strategy for several non-conventional therapeutic approaches, including oligonucleotides. Prior to joining Novartis in 2009, Sarah was a Scientist at Intelligent Bio-Systems, a next-generation sequencing company. She obtained her Ph.D. from The Scripps Research Institute, where she performed in vitro evolution on catalytic RNAs, and her bachelor’s degree from Brown University, where she investigated polymer drug delivery systems.

  • Jonathan  Yingling

    Jonathan Yingling

    • Senior Vice President, Early Developement
    • Idera, United States

  • Richard Stephen Geary, PhD

    Richard Stephen Geary, PhD

    • Executive Vice President, Drug Development
    • Ionis Pharmaceuticals, Inc., United States

    Dr. Geary is Senior Vice President of Drug Development at Ionis Pharmaceuticals. He is responsible for preclinical and clinical development of antisense drugs. 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 two successful CTD/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.

  • Ulrike  Gnad-Vogt , MD

    Ulrike Gnad-Vogt , MD

    • Chief Medical Officer
    • CureVac AG , Germany

    She is board certified in internal medicine, medical oncology and hematology and worked as attending oncologist at the National Center for Tumor Diseases in Heidelberg/Germany before joining CureVac in 2011. From 2005-2009 she was medical leader, responsible for several early phase clinical programs in immunooncology at Merck Serono in Darmstadt/Germany. At CureVac Ulrike oversees the clinical development and regulatory affairs of CureVacs RNA therapeutics and prophylactic vaccines. She earned her medical degree from the University of Homburg/Germany and completed training in internal medicine, hematology and medical oncology at the Universities of Mannheim and Heidelberg.

  • Ying  Huang, PhD

    Ying Huang, PhD

    • Pharmacologist, Office of Cellular, Tissue and Gene Therapies, CBER
    • FDA, United States

    Dr. Huang is a Pharm/Tox reviewer at CBER/FDA conducting reviews for regulatory submissions for cell and gene therapies, immunotherapy, viral therapy and tumor vaccines. Before joining FDA, she was a scientist at Genetic Therapy Inc., a Novartis Company, and received her Ph.D. degree in 1995.

  • Ryan  McGowan

    Ryan McGowan

    • Associate Director, Combination Products
    • AstraZeneca, United States

    Ryan McGowan is an Associate Director in Regulatory Affairs at AstraZeneca where he has responsibility for developing regulatory strategies for the approval of combination products and medical devices. Prior to joining AstraZeneca, Ryan was a premarket reviewer and combination products team leader at FDA’s Center for Devices and Radiological Health where he evaluated and influenced regulatory policy for drug delivery constituent parts of combination products.

  • Laurence  Mignon, PhD

    Laurence Mignon, PhD

    • Director, Clinical Development
    • Ionis Pharmaceuticals, Inc., United States

  • Matthias  Miller, PhD

    Matthias Miller, PhD

    • Project Manager
    • BioNTech RNA Pharmaceuticals GmbH, Germany

    Matthias Miller started to work as a clinical project manager for the BioNTech AG (Mainz, Germany) in August 2014. He is managing the first-in-human clinical trial utilizing polytopic RNA-vaccines targeting mutation-derived neo-antigens. Currently, he focuses on projects that aim at taking the next steps in the clinical development of this personalized vaccine and investigates the implementation of novel technologies into the manufacturing process. He acquired his Ph.D. at the German Cancer Research Center (DKFZ), where he investigated adaptive features of innate immune cells in the context of tumor immunotherapies. Matthias Miller studied biochemistry at the Eberhard Karls University in Tübingen.

  • Maria  Montiel-Gonzalez, PhD

    Maria Montiel-Gonzalez, PhD

    • Post Doctoral Fellow
    • The Marine Biological Laboratory, University of Chicago, United States

    In 2006 I started graduate school at the University of Puerto Rico and joined Dr. Joshua Rosenthal’s lab to study engineering a site-directed RNA editing strategy to correct genetic mutations at the level of the RNA. We designed a synthetic editing enzyme that is guided by an RNA oligonucleotide to a specific adenosine within an RNA molecule. After obtaining a PhD in Biochemistry in 2014, I continued in Rosenthal lab as a postdoc. My project is to improve the site-directed RNA editing strategy by making modifications to the editing enzyme and the guide oligonucleotide. Recently, Dr. Rosenthal’s lab moved to the Marine Biological Laboratory in Woods Hole, MA, where I am testing the ability of our strategy to correct mutations in CFTR mRNAs.

  • William  Querbes, PhD

    William Querbes, PhD

    • Director, Research
    • Alnylam Pharmaceuticals, United States

  • Olen M. Stephens, PhD

    Olen M. Stephens, PhD

    • Chemist
    • FDA, United States

  • James M. Willard

    James M. Willard

    • Pharmacologist, DCaRP, CDER
    • FDA, United States

  • Cathaline  Den Besten

    Cathaline Den Besten

    • Senior Director, Head Toxicology, ADME, PK
    • Proqr Therapeutics, Netherlands

  • Troels  Koch, PhD, MSc

    Troels Koch, PhD, MSc

    • VP, Head of Research, RNA Therapeutics
    • Roche Innovation Center Copenhagen, Denmark

    Troels Koch is PhD in bio-organic chemistry from the University of Copenhagen. He has worked in the area of nucleic acid chemistry and biology for 20 years. He co-founded Santaris Pharma A/S and pioneered the LNA technology platform and LNA Therapeutics. Santaris was acquired by Roche in August 2014. In his present position at Roche Innovation Centre Copenhagen the main responsibilities are to develop the chemical and biological properties of LNA, improve and upgrade the LNA platform, refine LNA antisense drug discovery processes, and establish a RNA therapeutics drug pipeline in Roche.

  • Keith  Peden

    Keith Peden

    • Microbiologist, Laboratory of Retroviruses, CBER
    • FDA, United States

  • Mike  Stelmah

    Mike Stelmah

    • Director, Regulatory Affairs, CMC & Combination Products
    • Alnylam Pharmaceuticals, United States

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