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 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

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 AstraZeneca as well as different mRNA applications.

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.

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 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 and then the Division of Neurology Products. Lois is currently the Director of the Division of Pharmacology/Toxicology in the Office of Neuroscience (OND/CDER).

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 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 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.

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.

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.

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.

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 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.

Dr. Wange is an Associate Director for Pharmacology & Toxicology within the Office of New Drugs in CDER at the FDA, and has over 15 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. In addition, he was the primary author of the recently published draft guidance on Nonclinical Testing of Individualized ASOs for Severely Debilitating or Life-Threatening Diseases. Prior to joining FDA, he was the head of the T-lymphocyte Signaling Unit at the National Institute on Aging at the NIH.

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

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.

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.

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, 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.

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.

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, Ph.D. Senior Vice President, Head of Clinical Pharmacology and Pharmacometrics Gabriel Robbie is Senior 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 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.

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 is a co-chair of FDA’s Pharmacology/Toxicology Oligonucleotide Subcommittee. She received a BS in Biology from State University of New York, a PhD in Cell Biology from University of Connecticut, and her MPH in Epidemiology at University of California at Berkeley. She was an Associate Investigator at San Francisco VA Medical Center where her research focused on miRNA dysregulation in prostate cancer. She completed post doctoral research at Stanford School of Medicine on miRNAs in NHL and was a Cancer Prevention Fellow at the National Cancer Institute in the Laboratory of Human Carcinogenesis where her research involved examining the role of extracellular plant small RNA communication in human carcinogenesis.

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.

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, PhD is Director of Center for Biological Safety and Research (CBSR), National Institute of Health Sciences (NIHS), Japan. Dr. Hirabayashi is responsible for research on the safety of various substances including pharmaceuticals as well as comprehensive safety assessment including toxicity prediction methods. Dr. Hirabayashi was a topic leader of the MHLW of the ICH S6 (R1) EWG and is currently the representative of the Japanese research group on ICH-related issues funded by AMED, specifically responsible for issues relating the preclinical safety assessment for biopharmaceuticals and oligonucleotide therapeutics. Dr. Hirabayashi graduated Yokohama City University School of Medicine and hold PhD in Medical Sciences.

Veronika Jekerle hold the position as Head of Office for EMA’s Pharmaceutical Quality Office in the Human medicines Division at European Medicines Agency. The office manages pharmaceutical quality aspects for all human medicines across the lifecycle from early development to post-authorisation. A pharmacist by training, she joined European Medicines Agency in 2006 as a Product Team Leader for Recombinant proteins, Vaccines, ATMPs and Biosimilar applications. She held various positions including Quality Specialist and Scientific secretary for the Biologics working party and coordinated several guidelines in the area of Biologicals, ATMPs and vaccines as well as PRIME toolbox guidance and prior knowledge.

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 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 beta-peptides. He is currently the CDER representative on the FDA Nanotechnology Taskforce and a member of the OPQ-OGD Oligonucleotide Product Specific Guidance working group.

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 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 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.

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 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 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.

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 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 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.

Ben Stevens is a Director of CMC Policy and Advocacy at GlaxoSmithKline and has nearly 15 years of drug discovery and regulatory experience. Prior to GSK, Ben was a Director of Regulatory Affairs CMC at Alnylam, a Principal Consultant at PAREXEL and an acting Branch Chief in the Office of New Drug Products (ONDP) at the FDA. 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 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 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.

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.

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.

Dr. Tim Yu, MD, PhD is a neurologist in the Division of Genetics and Genomics at Boston Children’s Hospital, Associate Professor at Harvard Medical School, and an Associate Member of the Broad Institute. His research group 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 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 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.

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.

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.

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.

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.

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 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 “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.

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

Rohit is a Senior Advisor at Eli Lilly & Company and is responsible for developing CMC regulatory strategies for small molecules, oligonucleotides and peptides. Previously, he was a senior CMC reviewer at FDA for 5 years where he reviewed small molecules, oligonucleotides and ADCs. Rohit obtained his Ph.D. in Medicinal Chemistry from The Ohio State University working on the design and syntheses of nucleoside analogues. This was followed by a post-doctoral work at University of Notre Dame and ORISE research fellowship at FDA where learned about oligonucleotide chemistry.

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.

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

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 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, 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 works as a regulatory consultant for CNS drugs and Rare Diseases. He has 8 years of experience in drug review, development, and regulatory experience at the U.S. Food and Drug Administration most recently in the role of Associate Director of Rare Diseases in CDER. He was responsible for all Divisions performing the scientific review and evaluation of rare disease therapeutic drugs and biologics subject to regulation by the CDER/OND. He has strong knowledge of review and development of small molecules and biologics targeting the central nervous system.

After Ribozyme Pharmaceuticals (RPI) and the Nucleic Acids Synthesis unit of Transgenomic, Inc., in March 2004 Dr. Nechev joined Alnylam Pharmaceuticals. In the last 18 years, he has led the development and implementation of the CMC strategy for siRNA therapeutics used in four approved products – ONPATTRO®, GIVLAARI®, OXLUMO® and Leqvio®(Novartis). ONPATTRO® is the first approved siRNA therapeutic and the first approved lipid nanoparticle (LNP) formulated oligonucleotide. GIVLAARI® is the first approved GalNAc-conjugated siRNA. Dr. Nechev received his Ph.D. degree in Organic Chemistry from St. Kl. Ohridski University, Sofia, Bulgaria and completed his post-doctoral training at Vanderbilt University, Nashville, Tennessee.

Lawrence Perez has been a CMC Reviewer for new drugs with the FDA since 2015 and in 2021 he became a Senior Pharmaceutical Quality Assessor for API New Drugs. 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 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.

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 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.

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.

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, Senior Vice President RNA Biochemistry & Manufacturing, has worked in the field of RNA biochemistry and molecular biology for almost 30 years. His work on RNA-based immunotherapies began in 2007 with Ugur Sahin and Andreas joined BioNTech SE 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 therapeutic agent.

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.

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 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.

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 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.

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.

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 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.