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

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

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

Arthur M. Krieg, MD 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.

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

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

Kim 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. 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

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

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

Sudip 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

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

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

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

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

Tal Zaks, M.D., Ph.D., is a Venture Partner with OrbiMed. Dr. Zaks was recently the Chief Medical Officer at Moderna, where he led the development of the company’s COVID-19 vaccine and other key programs. Previously, Dr. Zaks held senior leadership positions in drug development at major pharmaceutical companies, including Sanofi and GlaxoSmithKline. Dr. Zaks received his M.D. and Ph.D. from the Ben Gurion University and conducted post-doctoral research at the U.S. National Institutes of Health.

A PhD Chemical Engineer by training, Dr. Antia has spent over 30 years in the pharmaceutical industry carrying out process development with roles at Sandoz, J&J, Merck and Palatin Technologies, before joining Biogen in 2012, where he is now Head of Oligonucleotide Development

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

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

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

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

Lois 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).

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.

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.

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

Emily 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.

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

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.

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.

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

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

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

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.

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

Philip J. (P.J.) Brooks is the deputy director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC). He also is the working group co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project and the co-chair of the Bespoke Gene Therapy Consortium.

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.

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

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

CDR QuynhNhu Nguyen is an engineer officer and has been with FDA for more than 13 years. She began her work at FDA evaluating human factors (HF) and use safety of medical products with the MedSun project in Office of Surveillance and Biometrics, then with the Human Factors Premarket Evaluation Team in Office of Device Evaluation within the Center for Devices and Radiological Health, and now with the Division of Medication Error Prevention and Analysis (DMEPA).She currently serves as the Associate Director for Human Factors for DMPEA and her current responsibilities include developing policy and best practices for HF reviews to ensure clarity and consistency within the Division.

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

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

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

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

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.

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

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

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

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

Ryan McGowan is the Director of Digital Devices and Combination Products in Regulatory Affairs at AstraZeneca. He has responsibility for developing regulatory strategies for the approval of combination products and digital health products including software as a medical device. Prior to joining AstraZeneca, Ryan was a premarket reviewer and combination products team leader at FDA’s Center for Devices and Radiological Health where he evaluated and influenced regulatory policy for drug delivery constituent parts of combination products.

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

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

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.

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

Dr Peden obtained his PhD degree in molecular biology from the University of Edinburgh, UK. He did post-doctoral work in molecular virology at Johns Hopkins University School of Medicine studying the DNA tumor virus SV40. In 1988, he spent a year at the Pasteur Institute studying the molecular genetics of HIV-1 and HIV-2. Dr Peden then spent 5 years at the NIH before moving to CBER/FDA in 1994 as a senior investigator. His research at CBER has been in the area of cell substrates, adventitious agent detection, and vaccine safety. Dr Peden is a product reviewer for several types of vaccines, including adenovirus-vectored vaccines, DNA vaccines, RNA virus-vectored vaccines, lentivirus-vectored vaccines, and mRNA vaccines.