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.

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 In addition, he was the primary author of the recently published draft guidance on Nonclinical Testing of Individualized ASOs for Severely Debilitating or Life-Threatening Diseases.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.

As Chief Medical Officer Dr. Ticho is responsible for Stoke’s efforts to develop first-in-class RNA based disease-modifying medicines to treat severe genetic diseases. He is also co-founder and former CEO of Verve Therapeutics which is developing therapies to edit the genome and confer protection from cardiovascular disease. Prior to joining Stoke Barry was Head of R&D for Cardiovascular and Metabolic Diseases at Moderna Therapeutics. He was previously Head of External R&D Innovation for Cardiovascular and Metabolic Diseases at Pfizer and was Vice President of Clinical Development at Biogen. Barry obtained his MD and PhD degrees from the University of Chicago. He was on staff at Harvard Medical School and Massachusetts General Hospital

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.

Dr. Bart Rogers is a reviewer in the Division of Translational and Precision Medicine 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.

Andrew began his career in industry over 20 years ago and has spent the last 17 years in Regulatory Affairs. He’s had the great fortune of being a part of many great teams who have brought seven novel therapies to market for a variety of conditions. This includes the first three RNAi therapeutics. Andrew holds degrees from Bates College, Massachusetts College of Pharmacy and Health Sciences, and Babson College.

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. 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.

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.

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

Ramin Darvari is an Associate Research Fellow in Drug Product Design & Development group at Pfizer; contributing to the strategic and tactical planning for evaluation of external delivery technologies and internal delivery formulation & process development, with a focus on collaborative partner engagement. Ramin has lent his expertise in particle engineering and matrix-based drug delivery systems to evaluation and development of variety of applications, including his role as the drug product project lead for Pfizer-BioNTech Covid-19 Vaccine.

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. 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

Christian Wetter is an organic chemist and holds a Ph.D. from the University of Marburg. He started his professional career at Roche in 2004 in Chemical Development before moving to Novartis to Regulatory CMC in 2009. Since 2020 he has been a Regulatory Advisor in Small Molecule Development at Roche. Christian has worked on small molecule, peptide, oligonucleotide and device development projects in various phases of development and commercial lifecycle. He is part of the European Pharma Oligonucleotide Consortium (EPOC) and is currently leading its regulatory subteam.

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.

Dr. Dominik Altevogt is an experienced professional in the pharmaceutical industry, with over 15 years of experience leading regulatory submissions and health authority interactions for small molecule drugs, with a special focus on synthetic peptides and oligonucleotides. He started his career in CMC regulatory affairs at Bachem AG and has since worked for F. Hoffmann-La Roche AG and Novartis AG. Dominik holds a Ph.D. in organic chemistry from the University of Freiburg, Germany, and is an active member of the European Pharma Oligonucleotide Consortium (EPOC), where he currently leads the platform strategies subteam.

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. Xing Jing is an expert in oligonucleotide-based therapeutics. He possesses a broad range of experiences from biology to clinical trials. He was trained as a structural biologist in academia. After training, he worked in the field of R&D and bioinformatics in industry. Dr. Jing has been a clinical pharmacology reviewer at FDA since 2020. Initially, Dr. Jing was a reviewer in CDER at the FDA, where he reviewed oligonucleotide therapeutics and published a summary of clinical pharmacology for siRNA therapeutics. Currently, he is a reviewer of cell and gene therapies in CBER at the FDA. Dr. Jing's regulatory review experience covers a wide range of therapeutic areas including oncology, CNS, autoimmune, virology, etc.

Dr. Sydney Stern is a clinical pharmacology reviewer in the Division of Translational and Precision Medicine (DTPM) in the Office of Clinical Pharmacology (OCP) at the FDA. She is a primary reviewer for oligonucleotide programs and rare diseases in OCP and she has extensive experience with in vitro/in vivo extrapolation. Dr. Stern has led several data projects in the rare disease space and research projects investigating strategies for selecting safe starting doses in oligonucleotide-based therapeutic. Her research interests are focused on the pharmacology of synthetic oligonucleotides and rare diseases. She received her Master of Science in Clinical Research and a Ph.D. in Pharmaceutical Sciences at University of Maryland Baltimore.

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.

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.

Dr. Katherine Windsor is a Senior Pharmaceutical Quality Assessor (Drug Substance Lead) in the Office of Pharmaceutical Quality within the Center for Drug Evaluation and Research (CDER) at FDA. Katherine has 10 years of experience assessing CMC aspects of drugs in several therapeutic areas, particularly anti-infectives and antivirals, and a wide variety of APIs, including oligonucleotides, peptides, antibody-drug conjugates, and small molecules. Katherine conducted postdoctoral research at Vanderbilt University and obtained her Ph.D. in Organic Chemistry from the University of Wisconsin-Madison and her B.S. in Chemistry from the University of Notre Dame.

Sorcha McCrohan is a Specialist of Scientific Programs for the Americas Region at DIA. In her current role, she focuses on content development and strategy for DIA's meetings to improve and facilitate innovation in clinical research, drug development, and the fields of devices and diagnostics. Before joining DIA, she conducted COVID-19 research in Chiapas, Mexico, and worked in marketing within Pfizer's Global Vaccines Meningococcal franchise. Sorcha holds a BA in Sociology from Mount Holyoke College and an MSc in Global Health, Disease Prevention & Control from Georgetown University.

Bio – Carolien Versantvoort I have over 20 years experience as senior clinical pharmacology assessor and scientific expert at Medicines Evaluation Board in the Netherlands for new medicines and generics. Since 2014, I am a member of the Pharmacokinetic Working Party / Product Specific Bioequivalence Guidance Drafting Group at EMA, currently as Chair. In addition, I am member of the leadership team for the Clinical Pharmacology Special Interest Area group at EMA’s Methodology Working Party. Further, I was member of the ICH-M12 team as EU expert on the harmonisation of the drug interaction guideline.

Katharine is a pharmacologist and toxicologist with more than 30 years experience in nonclinical research and drug development. She has a proven track record in leading and empowering teams in large strategic initiatives e.g. immunogenicity. Her comprehensive experience spans various modalities (including large molecules, small molecules, and oligonucleotides), routes of administration, and disease areas including oncology. More recently she has been involved in the development of oligonucleotides in a number of neurological and rare diseases.

Eric is a biological reviewer in the Office of Gene Therapy (OGT) / CBER/ FDA. He reviews a wide range of direct acting gene therapy products including DNA or RNA therapeutics delivered by nanoparticles carriers, including lipid nanoparticles. An interdisciplinary scientist, he has degrees in biochemistry (M.S., Ph.D. University of Delaware) with extensive training in virology, immunology, and chemical biology. His dissertation included developing novel polymer-oligonucleotide conjugates to study innate cellular response. Postdoctoral studies included whole genome siRNA and miRNA screens to find host gene targets of noroviral infection. Prior to joining OGT, Eric was a reviewer of allergenic products and studied host antiviral response.

Lori is the Director of Analytical Development for Novo Nordisk Global Nucleic Acid Therapies, where her team is responsible for method development and validation activities for custom starting materials, drug substance and drug product, as well as drug substance characterization, drug substance and drug product release and stability studies, and CMC regulatory authoring. Prior to joining Novo Nordisk (formerly Dicerna Pharmaceuticals) in 2018, Lori spent 12 years at Agilent Technologies in a number of different roles within Quality Control, Analytical Development, and Analytical Services. Lori holds a B.S. in Chemistry from Abilene Christian University.

I am an Executive Director at Amgen, and lead the Safety Pharmacology & Animal Research Center (SPARC) department. My team includes 60+ staff (Thousand Oaks, San Francisco, Burnaby BC) and we contribute actively and extensively to the entire drug discovery and development pipeline. My scientific responsibilities include preclinical safety pharmacology, profiling of preclinical candidates for treatment of cardiovascular disease, oversight for animal welfare, and strategic leadership responsibilities. I have a PhD (Pharmacology, Rutgers Grad Sch of Biomed Sci, 1983-88) and was a post-doctoral fellow (UCLA Sch of Med, 1988-91). I have held lead the Safety Pharmacology Society, and actively contribute to several drug safety consortia.

Dr. Deyi Zhang is a senior chemist in the Office of Research and Standards (ORS), Office of Generic Drugs (OGD) at FDA specializing in complex active ingredients, including peptides, oligonucleotides and complex mixtures. He provides scientific inputs for regulatory policy and actively involves in pre-ANDA meetings, product-specific guidance development of such products, and manages related research activities. Dr. Zhang received his Ph.D. in organic chemistry from the University of Notre Dame. He had a two-year NIH postdoctoral fellowship training at the University of Pennsylvania before joining Eli Lilly in 2000. After 15 years in pharmaceutical industry, he joined FDA. He has over 50 publications and presentations.

Jessica Grieves is a toxicologic pathologist at Ionis Pharmaceuticals where she is involved with the nonclinical development of ASOs, siRNAs, and gene editing modalities with a focus on pulmonary, neurology, and cardiometabolic therapeutic areas. Prior to Ionis, Jessica was a toxicologic pathologist at Takeda Pharmaceuticals.

Meena is the Senior Vice President of Translational DMPK and Clinical Pharmacology at Stoke Therapeutics. Prior to joining Stoke in 2018, Meena served as senior director of bioanalytical, pharmacology and biomarker development at Wave Life Sciences. She played a pivotal role in building Wave’s stereopure oligonucleotide chemistry platform and in guiding the clinical entry of three antisense oligonucleotide programs. Earlier in her career, Meena worked at Alnylam Pharmaceuticals on siRNA chemistry and targeted siRNA delivery. Meena received her Ph.D. in chemistry with Dr. K.N. Ganesh at the National Chemical Laboratory in Pune, India, and did her postdoctoral research on nucleic acid analogs with Professor Larry W. McLaughlin at Boston Colle

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®.

Chris is the CEO and co-founder of Creyon Bio and an experienced leader leveraging computational methods, ML/AI, and deep biological insights to solve problems. He has extensive experience in pharma and biotech, including building and leading the functional genomics department at Ionis where he was responsible for company-wide genomics and bioinformatics efforts as well as execution and strategic leadership of exploratory drug discovery programs for rare and common diseases. He also worked at the Science and Technology Policy Institute advising the White House Office of Science and Technology Policy on biomedical research and health policy issues. Chris earned his PhD from Caltech and conducted post-doctoral training at Yale University.

Dr. Jinhee Yang is a Director in the New Drug Development Division at BIORCHESTRA, contributing to the preclinical development of antisense therapeutics with CNS drug delivery systems and clinical biomarker studies. Prior to joining, she led a CNS pharmacology team at CKD Pharmaceutical Company, evaluating clinical candidates and lead molecules for the treatment of various CNS diseases. She received her Ph.D. in molecular neurobiology from KAIST, Korea, and had postdoctoral training in the pathophysiology of Alzheimer's disease at Yale University School of Medicine and Seoul National University School of Medicine.

Dr Hughes trained in general internal medicine and has been in the biotech/pharma sector for over 25 years. He has been involved in more than 60 clinical trials with more than 30 drugs across multiple therapeutic areas including multiple rare diseases, neurology, cardiac and oncology. Dr Hughes is currently Chief Medical Officer at Avidity BioSciences, an RNA focused company developing treatments for rare neuromuscular and cardac diseases. Prior to joining Avidity he has held senior drug development positions at several other RNA therapeutics companies.