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DIA/FDA Oligonucleotide-Based Therapeutics Conference


  • Hobart  Rogers, PharmD, PhD

    Hobart Rogers, PharmD, PhD

    • Pharmacologist
    • FDA, United States

    Dr. Bart Rogers is a reviewer in the Genomics and Targeted Therapy Group in the Office of Clinical Pharmacology (OCP) at the FDA. Dr. Rogers also serves as an active duty officer with the United States Public Health Service. He serves as the lead for OCPs review of all synthetic oligonucleotides. His research interests are focused on the pharmacology of synthetic oligonucleotides, orphan disease drug development, and pharmacogenomics. Dr. Rogers completed his Pharm.D. degree from the University of Maryland, School of Pharmacy in 2004. He went on to obtain his Ph.D. in Clinical Pharmaceutical Sciences with a focus on cardiovascular pharmacogenomics from the same institution.

  • Sudip  Parikh, PhD

    Sudip Parikh, PhD

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

    Sudip S. Parikh, PhD, serves as Senior Vice President and Managing Director for DIA Americas. He previously served as Vice President and General Manager of Health & Consumer Solutions at Battelle, leading its Healthcare Improvement, Public Health Research, and Consumer, Industrial, and Medical Technology Development teams in R&D supported by government and commercial clients. Dr. Parikh is a board member of Research America, the Friends of Cancer Research, and the Food Innovation Center. He was a Presidential Management Fellow at the National Institutes of Health, and was awarded a National Science Foundation Graduate Research Fellowship while earning his PhD in Macromolecular Structure and Chemistry from The Scripps Research Institute

  • 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.

  • Paul C. Brown, PhD

    Paul C. Brown, PhD

    • ODE Associate Director for Pharmacology and Toxicology, OND, CDER
    • FDA, United States

    Dr. Brown's responsibilities include development and implementation of guidance and policy related to the nonclinical assessment of human pharmaceuticals. He has been at the FDA since 1996 when he joined the Division of Dermatology and Dental Drug Products as a Pharmacology/Toxicology reviewer. He was supervisor for Pharmacology/Toxicology in this Division from 2003 to 2008. Prior to coming to the FDA he was a Pharmacology Research and Training Fellow in the National Cancer Institute from 1991 to 1996. He worked on multidrug resistance gene structure and function in the Laboratory of Experimental Carcinogenesis. He received his Ph.D. in toxicology from the University of Maryland in 1991.

  • Arthur L. Caplan, PhD

    Arthur L. Caplan, PhD

    • Drs. William F. and Virginia Connolly Mitty Professor of Bioethics
    • NYU School of Medicine, United States

    The Drs. William F and Virginia Connolly Mitty Professor and founding head of the Division of Medical Ethics at NYU School of Medicine in New York City. Dr. Caplan is the author or editor of thirty-five books and over 725 papers in peer reviewed journals. His most recent books are The Ethics of Sport, (Oxford University Press, 2016 with Brendan Parent), Vaccination Ethics and Policy, (MIT Press, 2017 with Jason Schwartz) and Getting to Good (Springer, 2019) with Barbara Redman.

  • Lois M. Freed, PhD

    Lois M. Freed, PhD

    • Supervisory Pharmacologist, CDER
    • FDA, United States

    Lois Freed earned her undergraduate and Master’s degrees from the University of Kansas and her Ph.D. from the University of Maryland. Prior to working at the FDA, Lois worked at the National Institute on Aging/NIH in the Laboratory of Neurosciences. Lois has been at the FDA since 1992, joining the Division of Neuropharmacological Drug Products as a nonclinical reviewer. She was the Supervisory Pharmacologist in this Division from 2003 to 2005 and in the Division of Neurology Products from 2005 to the present.

  • Phil  Gatti, PhD

    Phil Gatti, PhD

    • Pharmacologist
    • FDA, United States

    Philip Gatti received the Ph.D. in Pharmacology from the University of Medicine and Dentistry of New Jersey. He did postdoctoral work at Georgetown University School of Medicine and then moved to a tenure-track position at Howard University College of Medicine where he rose to Associate Professor. In 2008, he came to the FDA as a Pharmacologist reviewer in the Division of Cardiovascular and Renal Products. He has published 50 peer-reviewed publications and has received numerous research grants. He has presented seminars internationally.

  • Silke  Klick, PhD

    Silke Klick, PhD

    • Regulatory Director CMC
    • AstraZeneca, Sweden

    Silke Klick is an analytical chemist with a degree from University of Münster, Germany and a PhD from University of Hannover, Germany. After a post-doctoral fellowship at University of Gothenburg, Sweden she joined AstraZeneca in 1991 and held manager positions within Pharmaceutical Development before moving into Regulatory Affairs in 2010. She is currently a Director in Regulatory CMC based in Gothenburg. With this background she has participated in the development of a considerable number of drugs.

  • Arthur M. Krieg, MD

    Arthur M. Krieg, MD

    • CEO and CSO
    • Checkmate Pharmaceuticals, United States

    Arthur M. Krieg, MD has worked in the oligonucleotide field since the 1980s. Most recently he founded Checkmate Pharmaceuticals to develop novel oligonucleotides for cancer immunotherapy, where he currently serves as CSO. Prior to that role Art was CSO at Sarepta until July 2014; co-founder and CEO at RaNA Therapeutics from 2011 to 2013; CSO of Pfizer’s Oligonucleotide Therapeutics Unit from 2008 to 2011; and co-founder, CSO of Coley Pharmaceutical Group from 1997 until its acquisition and incorporation into Pfizer in 2008.

  • Arthur A. Levin, PhD

    Arthur A. Levin, PhD

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

  • Saraswathy V. Nochur, PhD, MSc

    Saraswathy V. Nochur, PhD, MSc

    • Chief Regulatory Officer
    • Alnylam Pharmaceuticals, 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.

  • Nigel R. Richardson, PhD

    Nigel R. Richardson, PhD

    • Head Analytical Technology & Automation, CMC Analytical
    • GlaxoSmithKline, United Kingdom

    Dr Nigel Richardson is a senior director within the CMC Analytical Division of GlaxoSmithKline based in the UK. He completed his first degree in chemistry and PhD in synthetic organic chemistry at Exeter University, UK. Within GSK Dr Richardson has led the CMC activities for small molecules and led GSK’s oligonucleotide therapy for Duchenne Muscular Dystrophy into the final stages of development and regulatory submission. He has worked as chemistry lead in a team focused on novel platforms for oligonucleotide delivery. More recently he has supported the GSK / Ionis oligonucleotide collaborations and is representing GSK on the European Pharma Oligonucleotide Consortium (EPOC) Steering Team.

  • G. Susan  Srivatsa, PhD

    G. Susan Srivatsa, PhD

    • President
    • ElixinPharma, 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.

  • Christine  Swenson

    Christine Swenson

    • Head, Global Regulatory Affairs
    • Moderna Therapeutics, 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.

  • Kim  Tyndall

    Kim Tyndall

    • CMC Regulatory Consultant
    • CMC Tyndall Consultant LLC, 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.

  • Ronald L. Wange, PhD

    Ronald L. Wange, PhD

    • Associate Director for Pharm/Tox ODE3, CDER
    • FDA, Office of New Drugs, 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.

  • 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

  • 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.

  • Jessica  Hawes, PhD

    Jessica Hawes, PhD

    • Deputy Director
    • FDA, United States

    Dr. Jessica Hawes has been at the FDA since 2010 and is currently serving as Deputy Director in the Division of Systems Biology at the National Center for Toxicological Research. She served as Acting Team Leader and primary reviewer for Pharmacology and Toxicology for 9 years in the Department of Metabolism and Endocrinology Products (DMEP), Center for Drug Evaluation and Research (CDER). Dr. Hawes is a participant in the FDA Leadership Development Program and numerous CDER subcommittees and working groups. Dr. Hawes was a Postdoctoral Fellow at the National Cancer Institute and received her Ph.D. in Pharmacology from Yale University.

  • 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.

  • Donald N. Jensen, DVM, MS

    Donald N. Jensen, DVM, MS

    • Pharmacologist
    • FDA, United States

    Donald N. Jensen, D.V.M, M.S., is a nonclinical pharmacology/toxicology reviewer in the Division of Cardiovascular and Renal Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration.

  • Edward M. Kaye, MD

    Edward M. Kaye, MD

    • Chief Executive Officer
    • Stoke Therapeutics, United States

    I am a Pediatric Neurologist/Biochemical Geneticist and a long academic experience most recently as Chief of Biochemical Genetics at Children's Hospital of Philadelphia and industry experience as Group VP Clinical Research in charge of genetic diseases at Genzyme, CMO and CEO at Sarpeta Therapeutics, and most recently CEO of Stoke Therapeutics.

  • Imran  Khan, PhD

    Imran Khan, PhD

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

    I am a Pharmacologist at FDA with several years of experience in regulatory science and bench research in the fields of pain neurotransmission and cardiovascular regulation. My research interests are in receptor-ligand interactions including off-target binding, immunohistochemistry and antisense oligonucleotide treatment in experimental animal models.

  • Gabriel J. Robbie

    Gabriel J. Robbie

    • VP Clinical Pharmacology and Pharmacometrics
    • Alnylam Pharmaceuticals, Inc., United States

    Gabriel J. Robbie, Ph.D. Vice President, Head of Clinical Pharmacology and Pharmacometrics Gabriel Robbie is Vice President and Head of Clinical Pharmacology, and Pharmacometrics at Alnylam Pharmaceuticals in Cambridge, Massachussets. In this role, he leads a team providing development strategies for novel siRNA based therapeutics. Prior to joining Alnylam, Dr. Robbie spent ten years as Senior Director and site head for Clinical Pharmacology, Bioanalysis and Clinical testing laboratory at MedImmune, Gaithersburg, where he was primarily involved with monoclonal antibody and biologics development.

  • Brian  Stultz, MS

    Brian Stultz, MS

    • Staff Scientist, Division of Cellular and Gene Therapies
    • FDA, United States

    Brian Stultz is a Staff Scientist and CMC reviewer in the Division of Cellular and Gene Therapies, Office of Tissues and Advanced Therapies at CBER, FDA. He received a Master of Science with training in Biochemistry and Molecular Biology from the University of Virginia. At the FDA, Brian evaluates CMC submissions for gene therapy products and participates in policy development for gene therapy product safety. In addition to his regulatory activities, he is involved in laboratory research studying issues relevant to the safety and effectiveness of gene and cell therapy products.

  • Laurence  Whiteley, DVM, PhD

    Laurence Whiteley, DVM, PhD

    • Senior Director, Global Pathology Team Leader
    • Pfizer Inc, United States

    Larry Whiteley DVM, PhD, ACVP is a Veterinary Pathologist with 27 years of experience in the safety assessment of small molecules, biologics, oligonucleotides and gene therapy. He started his career in toxicology as a postdoctoral associates at the Chemical Industry Institute of Toxicology where he studied mechanisms of mineral fiber induced pulmonary carcinogenesis. He then joined the Procter and Gamble company where he worked as study pathologists and lead an investigative pathology laboratory. Larry is currently a Sr. Director at Pfizer where he is a Global Pathology Team Leader and subject matter expert on the nonclinical safety and risk assessment of oligonucleotide therapeutics and gene therapy modalities.

  • 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.

  • C. Frank  Bennett, PhD

    C. Frank Bennett, PhD

    • Senior Vice President, Research
    • Ionis Pharmaceuticals, Inc., United States

    Dr. Bennett is senior vice president of Research at Ionis Pharmaceuticals and one of the founding members of the company. He is responsible for preclinical antisense drug discovery and antisense technology research and is the franchise leader for neurological programs at Ionis. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry. Dr. Bennett has published more than 200 papers in the field of antisense research and development and has more than 175 issued U.S. patents.

  • Daniel  DeCollibus, MS

    Daniel DeCollibus, MS

    • Scientist I
    • Biogen, United States

  • Jennifer A. Franklin, PhD

    Jennifer A. Franklin, PhD

    • Director, CMC RegulatoryAffairs
    • Ionis Pharmaceuticals, Inc., United States

    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, USA. She earned her B.S in Biology and French from the University of Wisconsin.

  • Yoko  Hirabayashi, MD

    Yoko Hirabayashi, MD

    • Director, Center for 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.

  • Veronika  Jekerle, PhD, RPh

    Veronika Jekerle, PhD, RPh

    • Head of Pharmaceutical Quality
    • European Medicines Agency, Netherlands

    Veronika Jekerle joined the European Medicines Agency in 2006. She has been a Product Team Leader for numerous Biological medicinal products applications including Recombinant proteins, Vaccines, ATMPs and Biosimilar applications. In 2014 she became a Quality Specialist within the Specialised Scientific Disciplines Department where she provides specialised scientific input to various procedures including ITF, scientific advice, PRIME, marketing authorisation and post-marketing procedures. She has also coordinated the development of a number nt guidelines in the area of Advanced Therapies, Recombinant proteins and Biosimilars.

  • William F. Kiesman, PhD

    William F. Kiesman, PhD

    • Senior Director
    • Biogen, Inc., United States

    William Kiesman, Ph.D. received his B.S. (1990) and Ph.D. (1995) degrees in chemistry from the University of Connecticut, and pursued postdoctoral studies at Duke University (1995-1997). He then joined Biogen and worked in a variety of roles in the medicinal chemistry and chemical development groups. In 2016, Will took over leadership of the Antisense Oligonucleotide Development and Manufacturing team. He has published twenty-three scientific articles, three book chapters, and has nine patents. He was a member of the API Leadership group within the IQ Consortium (2011-2017) and is currently a co-chair of the Starting Material Working group within the European Pharma Oligonucleotide Consortium.

  • Olen M. Stephens, PhD

    Olen M. Stephens, PhD

    • Chemist Reviewer, CMC Reviewer, OND
    • FDA, United States

    Olen Stephens is a chemistry reviewer for the CMC branch that supports the Office of Hematology and Oncology Products. Over the past 11 years, he has served as a reviewer, CMC lead, and acting branch chief to support over half the clinical division in the Office of New Drugs at CDER. His formal training began as a bioorganic chemist at the University of Utah for his Ph.D., where he studied double stranded RNA ? protein interactions and continued as a post-doc at Yale in biophysical chemistry, designing de novo secondary structures using ?-peptides.

  • Robert  Ward

    Robert Ward

    • Chairman and CEO
    • Eloxx Pharmaceuticals, United States

    Mr. Robert (Bob) E. Ward is the Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals, Inc. (“Eloxx”). He previously served as the Chief Executive Officer and President at Radius Health, Inc. (NASDAQ: RDUS) successfully completing the initial public offering that became the top performing IPO; raised over $780M from private and public sources; achieved FDA approval and launch of the new drug TYMLOStm injection while gaining Fast Track development status for the Elacestrant oncology program.

  • Nadim  Akhtar, PhD

    Nadim Akhtar, PhD

    • Principle Scientist
    • AstraZeneca, United Kingdom

    Nadim Akhtar has a PhD degree in chemistry from Manchester and has been working for AstraZeneca since 2005. During this time Nadim has worked across different fields including new modalities, advanced drug delivery and predictive science supporting both drug substance and drug product activities. Nadim currently sits in the Global Product Development and has been involved with oligonucleotides since 2012, supporting both early and late stage portfolio. Nadim has been involved with several regulatory filing involving small molecules, oligonucleotides and nanomedicines.

  • Nicole  Del Canto

    Nicole Del Canto

    • Director, Global Regulatory CMC
    • Biogen Inc., United States

    Nicole del Canto is a Director of Global Regulatory CMC at Biogen, a Massachusetts-based biotechnology company focused on neurological diseases. Her regulatory team is responsible for leading clinical and commercial oligonucleotide and small molecule programs including global Agency engagement and registration of clinical trials and marketing authorization applications. Prior to Biogen, she worked in Regulatory CMC at Genentech, a member of Roche and has previous experience in QC analytical. She received a B.S in Biology from University of Massachusetts at Amherst.

  • 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.

  • Martin  Lamb

    Martin Lamb

    • Principal Investigator, Comparative Pathology
    • Biogen, United States

    Martin is a toxicologic pathologist in the preclinical safety group at Biogen in Cambridge, Massachusetts. He gained his degree in veterinary medicine at the Royal Veterinary College, London, UK, after which he completed a residency in veterinary pathology at the University of Pennsylvania School of Veterinary Medicine and became a Diplomate of the American College of Veterinary Pathologists. He then spent several years working as a toxicologic pathologist for Roche in New Jersey, before moving to Biogen in 2010 where he has recently been involved with a number of antisense oligonucleotide programs.

  • Bianca  Matthee, PharmD, MPharm, MSc

    Bianca Matthee, PharmD, MPharm, MSc

    • Vice President CMC
    • ProQR Therapeutics, Netherlands

    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.

  • Tracy  McGregor, MD

    Tracy McGregor, MD

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

    Dr. McGregor is a Director of Clinical Research at Alnylam Pharmaceuticals in Cambridge, Massachusetts, USA. She serves as the global clinical lead for the lumasiran program, an investigational RNAi therapeutic for the potential treatment of Primary Hyperoxaluria Type 1. Previously she was on faculty as a Clinical Geneticist at Vanderbilt University Medical Center. She trained in Pediatrics and Clinical Genetics at St. Louis Children’s Hospital in St. Louis, Missouri. Dr. McGregor obtained her medical degree at Washington University in St. Louis and her Master of Science in Clinical Investigation at Vanderbilt.

  • Jennifer  Panagoulias, RAC

    Jennifer Panagoulias, RAC

    • Vice President, Regulatory Affairs
    • WAVE Life Sciences, United States

    Jennifer Panagoulias, RAC is the Vice President of Regulatory Affairs at Wave Life Sciences. Jennifer has worked in drug development for over 20 years, primarily focused on advancing global development programs for the treatment of rare neurological diseases. She spent 16 years in Regulatory Affairs at Genzyme where she held various roles including Global Therapeutic Head, Regulatory Affairs Neurology supporting global registration efforts for Myozyme (alglucosidase alfa), an enzyme replacement therapy for children and adults with Pompe disease, and Lemtrada (alemtuzumab) for MS.

  • Martina  Sahre, PhD

    Martina Sahre, PhD

    • Staff Fellow
    • FDA, United States

    Martina is a Policy Lead in the Office of Clinical Pharmacology Guidance and Policy Team at the US Food and Drug Administration. Her interests are related to intrinsic factors, such as renal and hepatic impairment, obesity, age and others. Prior to that, she was a reviewer in the Office of Clinical Pharmacology, reviewing cardiovascular and renal drug programs.

  • Benjamin  Stevens, PhD, MPH

    Benjamin Stevens, PhD, MPH

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

    Ben Stevens is an Associate Director of Regulatory Affairs, CMC at Alnylam and has nearly 13 years of drug discovery experience. Prior to Alnylam, Ben was a Principal Consultant at PAREXEL and also spent nearly 4 years in the Office of New Drug Products (ONDP) at the US FDA where he was an Acting Branch Chief. Before FDA, Ben spent seven years in pharmaceutical R&D at Pfizer and Merck. Ben received a Ph. D. in Chemistry from the University of Pittsburgh, a M.P.H. from the Johns Hopkins and is a co-author of over 20 publications and patents.

  • Hiroshi  Takeda, PhD, MS

    Hiroshi Takeda, PhD, MS

    • Technical Officer
    • Pharmaceuticals and Medical Devices Agency (PMDA), Japan

    Hiroshi Takeda is a reviewer of Office of New Drug III, Pharmaceuticals and Medical Devices Agency (PMDA). He is currently engaged in review of drugs for neurology and psychiatry.

  • Yann  Tessier, DVM

    Yann Tessier, DVM

    • Senior Toxicology Project Leader
    • Roche, Denmark

    Yann Tessier is currently a senior toxicology project lead at Roche in Basel, Switzerland. Yann has supported antisense oligonucleotide drug discovery and development since 2011 when he joined Santaris Pharma and then got mentored by Art Levin. Prior to this, he had spent 12 years in project toxicology and comparative medicine roles in Europe in pharmaceutical companies and CRO. Initially trained as a DVM, he gained postgraduate qualifications in toxicology, veterinary ophthalmology and translational medicine. He is a European Registered Toxicologist.

  • James D. Thompson, PhD

    James D. Thompson, PhD

    • CMC Therapeutic Area Lead
    • 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.

  • Mathias  Vormehr, PhD

    Mathias Vormehr, PhD

    • Head of Cancer Vaccines
    • BioNTech RNA Pharmaceuticals GmbH, Germany

    Dr. Mathias Vormehr is an immunologist heading the research group Cancer Vaccines at BioNTech, Mainz, Germany. He performed his diploma thesis on immunotherapy at the German Cancer Research Center Heidelberg and received a doctorate in pharmacy from the University of Mainz. His work on neoantigens and personalized cancer vaccines was published in high impact journals including Nature.

  • Wendy  Wu, PhD

    Wendy Wu, PhD

    • Pharmacologist
    • FDA, United States

  • Timothy W. Yu, MD, PhD

    Timothy W. Yu, MD, PhD

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

    Dr. Tim Yu, MD, PhD is a neurologist in the Division of Genetics and Genomics at Boston Children’s Hospital, Assistant Professor at Harvard Medical School, and an Associate Member of the Broad Institute An early pioneer in high throughput sequencing, he has identified over a dozen new human disease genes. His laboratory applies genetics, neurobiology, and bioinformatics to study neurodevelopmental disorders and advance genomic medicine, ranging from autism gene discovery, genome sequencing for neonatal care, and the development of therapeutic approaches to orphan pediatric neurogenetic diseases.

  • Jeff  Allen, PhD

    Jeff Allen, PhD

    • Associate Director, Toxicology
    • Alnylam Pharmaceuticals, Inc., United States

    Jeff Allen is an Associate Director of Toxicology at Alnylam Pharmaceuticals. He has been involved in the direct delivery of a wide range of therapeutics to the CNS for the past 20 years with an emphasis on intrathecal and intraparenchymal delivery in large animal models. This work has included positions within academia, a medical device company, a CRO, and currently at Alnylam where he is leading the preclinical CNS toxicology efforts.

  • Matt R. Buck, JD

    Matt R. Buck, JD

    • Vice President, Regulatory Affairs
    • Ionis Pharmaceuticals, Inc., United States

    Matt is an experienced regulatory affairs professional with over 15 years working on therapeutics for rare diseases. At Ionis he has successfully led the global regulatory strategy for Spinraza (nusinersen) and Tegsedi (inotersen). Now as Head of Regulatory Affairs at Ionis he oversees all regulatory activities for the companies diverse pipeline which contains numerous investigational products in development for orphan indications.

  • Jonathan  Burgos, PhD

    Jonathan Burgos, PhD

    • Lieutenant Commander, U.S. Public Health Service Commissioned Corps
    • CDER, FDA, United States

    LCDR Jonathan Burgos is a microbiology reviewer at FDA/CDER Division of Microbiology Assessment (DMA). His analysis of scientific information presented in ANDAs, NDAs, and INDs ensures FDA-regulated drug products meet the standards of microbial safety as required by Federal law. Prior to his transition to DMA, LCDR Burgos held a 6-year tenure at FDAs Center for Biologics Evaluation and Research (CBER) with the Office of Vaccines Research and Review. During his time at CBER, LCDR Burgos balanced his time between reviewing Biologic drug product applications and conducting microbial pathogenesis research on Corynebacterium diphtheriae.

  • Sagar  Damle, PhD

    Sagar Damle, PhD

    • Associate Director, Functional Genomics
    • Ionis Pharmaceuticals, Inc., United States

  • Diana  Escolar, MD

    Diana Escolar, MD

    • Senior Vice President Medical Science
    • Miragen, United States

    Dr. Diana Escolar, M.D serves as the Senior Vice President of Medical Sciences at miRagen Therapeutics, Inc., where she leads the clinical development of microRNA therapeutics. Dr. Escolar has applied nearly two decades of research in rare disorders to help guide the development of new therapies. She has provided medical oversight and guidance for all phases of the drug discovery process as well as target identification for various drug development companies, including Acceleron, Genzyme, Wyeth and Shire. With a long academic career at George Washington University and Johns Hopkins, she received her M.D. from the University of Buenos Aires and her post-graduate training from Boston University- and Harvard Medical School affiliated-programs.

  • James  Hamilton, MD, MBA

    James Hamilton, MD, MBA

    • Vice President, Clinical Development
    • Arrowhead Pharmaceuticals, United States

    Dr. Hamilton currently serves as Vice President, Clinical Development at Arrowhead Pharmaceuticals and previously served as Medical Director at Arrowhead. In these roles, he has designed and managed multiple clinical studies with a wide range of siRNA compounds and has led clinical programs in various disease areas including hepatitis B, alpha-1 antitrypsin deficiency and dyslipidemia. He is the global medical lead on the AROAAT2001 (SEQUOIA) study evaluating the siRNA compound, ARO-AAT for the treatment of liver disease in the setting of alpha-1 antitrypsin deficiency. Dr. Hamilton holds an MD and an MBA from The Ohio State University and is board certified in Emergency Medicine.

  • James  Kramer, PhD

    James Kramer, PhD

    • Principal Scientist
    • Charles River Laboratories, United States

    Jim Kramer is a Principal Scientist at Charles River Laboratories’ Cleveland Location. He earned a Ph.D. in Biomedical Science from Wright State University (1995) and did his post-doctoral training with Glenn Kirsch and Buzz Brown at Case Western Reserve University School of Medicine. Jim joined ChanTest Corporation in 1999 as a Study Director and has devoted his 20 year career determining the effects of drugs on various cardiac models (in vitro, ex vivo and in vivo) but primarily on the conductance of cardiac ion channels, the proteins that underlie the electrical activity of the heart.

  • Ben-Fillippo  Krippendorff, PhD

    Ben-Fillippo Krippendorff, PhD

    • DMPK/PD Project Leader, Pharmaceutical Sciences
    • Roche Pharma Research and Early Development, Roche Innovation Center, Switzerland

    Dr Ben-Fillippo Krippendorff gained his PhD from the Max-Planck Graduate School for Computational Biology and Scientific Computing in Berlin and the Hamilton Institute, National University of Ireland. For the PhD he used quantitative data and computational modeling approaches to optimize the PK/PD of therapeutic proteins in cancer therapy. Afterwards, Ben-Fillippo accepted a postdoc at the University of Cambridge (UK), where he was part of the Pharmacology and Drug Development Group and based in the Department of Oncology. The postdoc offered the possibility to apply the theoretical knowledge on actual newly developed therapies for mice and patients. During his postdoc he also worked part time as a consultant for Genentech.

  • Lauren  Mihalcik, PhD

    Lauren Mihalcik, PhD

    • Senior Scientist, Toxicologist | Comparative Biology and Safety Sciences
    • Amgen, United States

    Lauren Murphree Mihalcik, PhD, DABT, is a Senior Scientist at Amgen in the department of Comparative Biology and Safety Sciences. She received her PhD in Pharmacology from the University of Virginia under Joel Linden. She spent eight years as a pharmacology/toxicology reviewer in the Division of Metabolism and Endocrinology Products at FDA/CDER, where she was also the FDA scientific liaison to the CDSIC/SEND team. At Amgen, Mihalcik is a project team toxicologist for several programs and leads Amgen's nonclinical safety strategy for siRNA molecules.

  • Padma Kumar  Narayanan, DVM, PhD, MS

    Padma Kumar Narayanan, DVM, PhD, MS

    • Executive Director
    • Ionis Pharmaceuticals, Inc., United States

    Padma-Kumar “Padma” Narayanan holds a BVSc and Ph.D. degrees and a post-doctoral fellowship (Los Alamos National Laboratories, NM). Padma is currently Executive Director of Toxicology at Ionis Pharmaceuticals in Carlsbad, CA, following careers in the Nonclinical Safety Assessment Departments in SmithKline Beecham/GlaxoSmithKline and Amgen. His work in the field of cytometry, platelet pathophysiology, immunopharmacology, and mechanistic toxicology has been published in numerous peer-reviewed journals, books, newsletters, and newspapers. Padma is a member of SOT, STP, American Society for Hematology, and International Society for Advancement of Cytometry.

  • Ramesh  Raghavachari, PhD

    Ramesh Raghavachari, PhD

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

    Ph.D - Temple University, Philadelphia, PA Currently a Chemist at FDA/CDER, has been with FDA since 2003.

  • Rohit  Tiwari, PhD

    Rohit Tiwari, PhD

    • Chemist
    • FDA, United States

    Rohit joined FDA, Office of New Drug Products in the capacity of API (drug substance) reviewer in June 2016. Prior to the joining as a reviewer, he was an ORISE fellow in the office of biotechnology products (OBP) in FDA and learned about oligonucleotide chemistry. Rohit earned a Ph.D. in Medicinal Chemistry from The Ohio State University where he worked on the design and syntheses of nucleoside analogues. This was followed by a post-doctoral work at University of Notre Dame where he worked on the design and syntheses of small molecule and siderophores against tuberculosis and malaria. As an API reviewer, Rohit has reviewed complex drug substances such as oligonucleotides, peptides and antibody drug conjugates.

  • Yanfeng  Wang, PhD

    Yanfeng Wang, PhD

    • Executive Director
    • Ionis Pharmaceuticals, United States

    Yanfeng is the Head of the Pharmacokinetics and Clinical Pharmacology group at Ionis, a leading biopharmaceutical company specializing in antisense oligonucleotides drug development. Prior to joining Ionis, Yanfeng worked for Novartis and Merck for nearly 20 years and led DMPK and Clinical Pharmacology teams supporting oncology and neuroscience therapeutic areas, as well as various rare disease indications.

  • William E. Achanzar, PhD

    William E. Achanzar, PhD

    • Director, Toxicology
    • Bristol-Myers Squibb, United States

    Dr. Achanzar is currently the Drug Safety Evaluation Therapeutic Area Head for Cardiovascular Diseases at Bristol-Myers Squibb, where he oversees the nonclinical safety strategy for all CV assets. He received his PhD in Molecular and Cellular Biology from the University of Arizona. After graduating, he trained in toxicology as a postdoctoral fellow at the National Cancer Institute with Dr Michael Waalkes. He joined BMS in 2003 as a junior toxicologist and, over the past 16 years, has acted as the toxicology lead for a number of early- and late-stage drug development programs across multiple disease areas and modalities, including oligonucleotides.

  • Charlotte  Bon, DVM, PhD

    Charlotte Bon, DVM, PhD

    • Translational Modeling and Simulation Scientist
    • F. Hoffman-La Roche AG, Switzerland

    Charlotte is a translational PKPD modeler at Roche (Basel) since 2017 and supports the RNA therapeutics drug development up to Ph1. Charlotte is a trained veterinarian and holds a PhD in Pharmacology and modeling and simulation (M&S) from the National Veterinary School of Toulouse, France. Her PhD thesis focused on optimizing the targeting to hepatocytes via the asialoglycoprotein receptor through PKPD and M&S analysis. Her current areas of interest gravitate around RNA therapeutics and human early dose predictions. In her free time Charlotte enjoys playing sports with tennis on top of the list and traveling.

  • David  Carbone, PhD

    David Carbone, PhD

    • Toxicologist
    • FDA, United States

    David Carbone has a background in neurodegenerative disorders, and has been a reviewer in Division of Neurology Products since 2014.

  • Chantal  Ferguson

    Chantal Ferguson

    • MD/PhD Candidate
    • University of Massachusetts Medical School, United States

    Chantal Ferguson is a graduate of Wesleyan University with a BA in Neuroscience. At Wesleyan, she performed 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.

  • Nagy  Habib

    Nagy Habib

    • Head of Research and Development
    • MiNA Therapeutics Limited , United Kingdom

    Nagy is a founder of MiNA Therapeutics Limited, London, UK and acts as both Chairman, Head of R&D and Chief Medical Officer. Currently he is Lead Clinician and Head of the Department of HPB Surgery at Imperial College London. Previously, he pioneered the first clinical trial in the use of adenovirus and plasmid for the treatment of liver cancer, the use of plasmid gene therapy in hydrodynamic gene delivery and autologous stem cells for patients with liver disease and acute stroke. He was the founder and Chairman of EMcision Limited, medical device company that was acquired by Boston Scientific Inc in March 2018.

  • Bahru  Habtemariam, PharmD

    Bahru Habtemariam, PharmD

    • Senior Director, Clinical Pharmacology
    • Alnylam Pharmaceuticals, United States

    Bahru Habtemariam, Pharm.D., is a senior director of Clinical Pharmacology at Alnylam Pharmaceuticals. Prior to joining Alnylam in October 2017, Dr. Habtemariam served as a Clinical Pharmacology reviewer and Team Leader at the United States FDA.

  • Lucas  Kempf, MD

    Lucas Kempf, MD

    • Medical Officer
    • FDA, United States

    Dr. Lucas Kempf is the Acting Associate Director for the Rare Disease program in the OND immediate office. Prior to joining FDA in 2012, Lucas spent 8 years at NIH with a focus on neuroscience research, working to understand the genetics of neuropsychiatric disease and developing translational approaches and therapeutics for these disorders. Lucas was trained at UC-Berkeley in molecular biology and genetics, received his MD degree from the University of Kansas, and did his post-graduate training in psychiatry at Georgetown and Johns Hopkins before moving to the NIH for fellowship.

  • Lubomir  Nechev, PhD

    Lubomir Nechev, PhD

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

  • Lawrence  Perez, PhD

    Lawrence Perez, PhD

    • Review Chemist
    • FDA, United States

    Lawrence Perez has been a CMC Reviewer for new drugs with the FDA since 2015. Before that, Lawrence was a discovery chemist with Novartis Oncology. Lawrence has been active in the areas of pharmaceutical regulations and medicinal chemistry, with his most notable work being the discovery and development of the oncology drug Farydak®.

  • Mark K. Schlegel, PhD

    Mark K. Schlegel, PhD

    • Principal Scientist, RNAi Discovery
    • Alnylam Pharmaceuticals, Inc. , United States

    Mark earned his PhD in Chemical Biology at the University of Pennsylvania in 2009 working on the elucidation of the structural and biophysical properties of glycol nucleic acid, an acyclic nucleic acid analog. He subsequently pursued postdoctoral studies at the Max Planck Institute of Colloids and Interfaces in Berlin working on automated oligosaccharide synthesis and DNA-carbohydrate conjugates. In 2014, Mark joined Alnylam Pharmaceuticals where he leads the company’s high throughput synthesis lab and works closely with research colleagues on lead development and siRNA design; most notably, the company’s ESC+ design strategy which features an improved specificity and therapeutic index.

  • Nan  Zheng, PhD

    Nan Zheng, PhD

    • Scientific Lead
    • Office of Clinical Pharmacology/CDER, FDA, United States

    Dr. Nan Zheng is a Scientific Lead for the Interdisciplinary Review Team for Cardiac Safety Studies (formerly QT-IRT) at US FDA. She is experienced in applying clinical pharmacology principles to the dose section, clinical trial design, and cardiac safety evaluation across different therapeutic areas. Previously she worked as a pharmacometrics reviewer at the Office of Clinical Pharmacology and a science reviewer at the Office of Generic Drugs. Dr. Zheng received her Ph.D. in Pharmaceutical Sciences from the University of Michigan.

  • Ivan  Zlatev, PhD

    Ivan Zlatev, PhD

    • Principal Scientist, Research
    • Alnylam Pharmaceuticals, Inc., United States

    Ivan Zlatev is a Principal Scientist at Alnylam’s Research Department. His focus of research is centered around the chemical synthesis and chemical modifications of synthetic oligonucleotides, and small interfering RNAs in particular. He has contributed to the discovery and development of two clinically validated delivery platforms for RNAi therapeutics and to the advancement of numerous therapeutic programs and platforms to clinical and pre-clinical development in the fields of RNAi and Antisense oligonucleotides. He is the author of over 25 peer-reviewed articles and chapters, and coinventor in 15 patent applications, with over 600 citations to date.

  • Panel  Discussion

    Panel Discussion

    • All Session Speakers, United States

  • Ian  Dobson

    Ian Dobson

    • Team Leader, Evaluator
    • Health Canada, Canada

    Ian Dobson is a Team Leader in the New Drugs Quality Division of Health Canada. Over a 20 year career with Health Canada he has been involved in regulatory review at all phases of product development and for a wide range of drugs, medical devices and combination products.

  • Eric F. Donaldson, PhD

    Eric F. Donaldson, PhD

    • Virology Reviewer, CDER/OND/OAP/Division of Antiviral Products
    • FDA, United States

    Dr. Donaldson is a virology reviewer at FDA working in the Division of Antiviral Products. He is an expert in Bioinformatics and Next Generation Sequencing and frequently performs independent analyses of regulatory data to support drug approvals. Prior to joining the FDA in 2012, Dr. Donaldson was an Assistant Professor at University of North Carolina where he worked in the areas of Coronavirus cross-species transmission and Norovirus evolution.

  • Andreas  Kuhn, PhD

    Andreas Kuhn, PhD

    • Vice President RNA Biochemistry
    • BioNTech RNA Pharmaceuticals GmbH, Germany

    Andreas Kuhn, Senior Vice President RNA Biochemistry & Manufacturing, has worked in the field of RNA biochemistry and molecular biology for more than twenty years. His work on RNA-based immunotherapies began in 2007 with Ugur Sahin and Andreas joined BioNTech RNA Pharmaceuticals GmbH shortly after its founding in 2008. His main focus is expanding BioNTech's proprietary technologies to increase the efficacy of RNA immunotherapies and to optimize GMP-compatible manufacturing processes for RNA. He has co-authored several publications and patents ranging from basic research on RNA to its application as a diagnostic and therapeutic agent.

  • Jing-Tao  Wu, PhD

    Jing-Tao Wu, PhD

    • Vice President, Early Development
    • Alnylam Pharmaceuticals, United States

    Jing-Tao is the Vice President of DMPK/Early Development at Alnylam Pharmaceuticals, where he oversees DMPK and investigative toxicology activities to support platform and pipeline. Prior to joining Alnylam, Jing-Tao was the Senior Director and the DMPK site head of Takeda Boston (formerly Millennium Pharmaceuticals), where he led the DMPK group to support the pipeline at all stages. Jing-Tao got his Ph.D. in chemistry from the University of Michigan. He is an associate editor of the Journal of Pharmacological and Toxicological Methods and a co-founder and the president of the Boston Pharmaceutical & BioSciences Society.

  • Oluseyi  Adeniyi, PharmD, PhD

    Oluseyi Adeniyi, PharmD, PhD

    • Reviewer
    • FDA, CDER, United States

    I received I graduate training from the University of Michigan where I studied ways to improve vaccine and gene delivery by cell-specific targeting and circumventing subcellular barriers. I joined the FDA as a Commissioner’s Fellow in 2015 to characterize the potential of biomarker-based drug development in the pharmaceutical pipeline for common chronic diseases. I am currently a reviewer in the Genomics & Targeted Therapy Group in the Office of Clinical Pharmacology where I contribute to advancing biomarker-based drug development strategies in the regulatory review of investigational new drugs and new drug/biologic applications

  • Rusty  Montgomery, PhD

    Rusty Montgomery, PhD

    • Director, Research
    • miRagen Therapeutics, Inc., United States

    Rusty Montgomery received his Ph.D. in 2008 in the laboratory of Eric Olson at UTSouthwestern Medical Center in Dallas. Following up on miRNA discoveries in the Olson lab, Rusty joined miRagen Therapeutics to translate these discoveries into novel therapeutics. He is currently Product Development Team Lead and Director of Research at miRagen Therapeutics.

  •   Panel Discussion

    Panel Discussion

    • All Session Speakers, United States

  • Faraz  Kazmi, PhD

    Faraz Kazmi, PhD

    • Senior Scientist, Drug Metabolism and Pharmacokinetics (DMPK)
    • Janssen Research & Development, United States

    Dr. Kazmi has over 13 years experience as a DMPK scientist and is currently a Senior Scientist in the DMPK PK/PD modelling group at Janssen Research and Development. Prior to that, he worked at the Sekisui XenoTech and has published several manuscripts on oligonucleotide in vitro drug-drug interactions. Dr. Kazmi received his BS (Cell and Molecular Biology) and MS (Biology) from Concordia University in Canada and a PhD from the University of Kansas Medical Center.

  • Venkateswaran  Chithambarampillai, PhD, MS

    Venkateswaran Chithambarampillai, PhD, MS

    • Senior Staff Fellow, Office of Clinical Pharmacology
    • CDER, FDA, United States

    Venkateswaran Chithambaram Pillai (Venki) received his PhD in Pharmaceutical Sciences from Texas Tech University Health Sciences Center and post-doctoral training at University of Pittsburgh. He has been a part of Office of Clinical Pharmacology, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA) and serving as Senior Staff Fellow since 2015.

  • Zhenrong  Yu, PhD

    Zhenrong Yu, PhD

    • Executive Director
    • Ionis Pharmaceuticals, Inc, United States

    Dr. Yu is an Executive Director of Pharmacokinetics and Clinical Pharmacology Group at Ionis Pharmaceuticals, Inc. Since joining Ionis in 1997, she has contributed to antisense platform advancement and drug development including multiple submissions. Being a pharmacokineticist by training, she is also an analytical expert, the primary inventor of the hybridization based-assays in quantifying antisense oligonucleotide in biological matrices. She has also developed extensive experiences in the areas of immunogenicity, evaluation of QT prolongation potential, pharmacokinetics, pharmacodynamics, and population-based modeling and simulations to support preclinical and clinical drug development.

  • Daniel  Mytych, PhD

    Daniel Mytych, PhD

    • Scientific Director, Clinical Immunology
    • Department of Translational Medicine, Amgen, Inc. , United States

    Dr. Daniel Mytych is a Scientific Director - Clinical Immunology in the Department of Translational Medicine at Amgen. He is an established subject matter expert in immunogenicity at both the preclinical and clinical development stage supporting various therapeutic modalities including proteins, cell therapies, siRNAs and biosimilars. Dan and his team are responsible for the implementation of predictive immunogenicity tools during lead selection, immunogenicity strategy and impact assessment of ADAs on PK, PD and safety.

  • John  Talian, PhD

    John Talian, PhD

    • Senior Vice President
    • CureVac, United States

    John Talian recently joined Curevac, Inc as the Senior VP of Global Regulatory Affairs. He previously worked with Hurley Consulting Associates directing the regulatory activities for small and mid-size Pharmaceutical clients. He served as US Head of Regulatory for Bayer Pharmaceuticals and had various regulatory roles with Hoffman-LaRoche both in the US and EU. John originally joined the industry with Johnson&Johnson following graduate and post-doctoral work in Cell and Molecular Biology.