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 In addition, he was the primary author of the recently published draft guidance on Nonclinical Testing of Individualized ASOs for Severely Debilitating or Life-Threatening Diseases.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

Laura Sepp-Lorenzino, Ph.D. joined Intellia Therapeutics in 2019 as Chief Scientific Officer and is responsible for Research and Early Development. Intellia’s leading CRISPR platform supports a full-spectrum therapeutic strategy. Laura previously held leadership positions at Vertex, Alnylam and Merck. She is on the BOD of Taysha Gene Therapies, the Alliance for Regenerative Medicine and the Oligonucleotide Therapeutics Society, and on the SABs for Thermo Fisher Scientific, the U.K. Nucleic Acid Therapies and Arsenal Capital Partners. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina and both her M.S. and Ph.D. in Biochemistry from New York University.

Andrew began his career in industry over 20 years ago and has spent the last 17 years in Regulatory Affairs. He’s had the 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 four RNAi therapeutics. Andrew holds degrees from Bates College, Massachusetts College of Pharmacy and Health Sciences, and Babson College.

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

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 a 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.

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 Supervisory Pharmacologist in the Office of Cardiology, Hematology, Endocrinology and Nephrology, CDER FDA. Prior to that, she was a senior consultant in the Federal Healthcare Practice at Deloitte Consulting LLP and her projects were focused on post-marketing drug safety, data warehouse and review modernization effort. 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 molecular genetics from Baylor College of Medicine and had postdoctoral training in molecular genetics and pathology at Columbia University Medical Center.

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.

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. Andrew Adams is the Co-Director for the Lilly Institute for Genetic Medicine. In this role, Andrew is responsible for leading the discovery of new types of therapies, such as genetic medicines, at Lilly via both internal research and robust collaborations with external partners. Andrew holds a degree in biology and a doctorate in zoology from the University of Aberdeen, Scotland. Prior to joining Lilly in 2011, Andrew was a postdoctoral fellow at Harvard Medical School studying the neurobiology of anorexia nervosa. During his time at Lilly Andrew has served in roles in early discovery, external innovation and as a leader of Lilly’s early trailblazer teams, championing new ways to bring Lilly science to patients with speed.

Nedim Emil Altaras SVP of Technical Development leads all CMC development activities at Moderna . He was previously the Head of External Manufacturing and the CMC lead of Moderna’s vaccine portfolio. Dr. Altaras has over 20 years of extensive experience in vaccines and biologics leading Global Product, Development, Manufacturing, Supply Chain and Operation teams. Dr. Altaras received a B. Eng from McGill University (1994), a Master’s in Organizational Dynamics from the University of Pennsylvania (2014) and a Ph. D. in Chemical Engineering from the University of Wisconsin-Madison (1999).

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. Sebastien Burel is currently Executive Director of Toxicology at Ionis Pharmaceuticals, Inc. Sebastien joined Ionis in 2007. Sebastien is leading efforts to characterize the various mechanisms of oligonucleotide mediated toxicity and translating those into safety screening models. Sebastien hold a Ph.D. in oncology and hematology from the Paterson Institute for Cancer Research in Manchester, UK, and an M.S. in biotechnology from the University of Strasbourg, France. .

Rima is a Senior Director of Regulatory Affairs CMC at Aadi Bioscience with 16 years of pharmaceutical industry experience in product development functions including CMC Regulatory Affairs and Formulation Development. Most recently, she worked at PTC Therapeutics as Director RA CMC and led several small molecule programs. Prior to that she was at Alnylam and supported NDA, MAA CMC filing strategy for first approved siRNA therapy. Rima also has technical expertise in manufacturing and analytical processes of drug products including parenteral lipid nano particle formulation. She received her Masters in Pharmacy degree from Massachusetts College of Pharmacy.

Dr. Rentel is currently Vice President, Analytical Development and Quality Control at Ionis Pharmaceuticals, Inc., Carlsbad, California. Prior to joining Ionis in 2001 he worked in Quality Control and Special Analytics at CarboGen in Switzerland. He received his Ph.D. (summa cum laude) from the University of Tuebingen, Germany. Dr. Rentel has 20 years of experience in Quality Control. He has extensive expertise in the CMC development of oligonucleotide therapeutics and is an expert in mass spectrometric techniques. He has been responsible for IND filings of more than 60 oligonucleotides and participated in the NDA filings for KYNAMRO® (mipomersen), SPINRAZATM (nusinersen), WAYLIVRA® (volanesorsen), and TEGSEDITM (inotersen).

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.

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.

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.

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.

I have been working as a Pharmacology/Toxicology reviewer in the Antiviral Division of OND/CDER/FDA. My job responsibilities include review animal and human data submitted to INDs, NDAs, and/or BLAs to evaluate the mechanism of action (MOA) as proof-of-activity or drug actions for use of medicinal products in human subjects,?and the safety of medicinal products for use in patients and healthy subjects, and provide regulatory recommendation such as clinical hold (INDs), need for clinical monitoring,?and drug approval (NDAs/ANDAs/BLAs) decisions, based on in vitro and/or nonclinical and/or clinical data. Before FDA, I worked as toxicologist in EPA to review regulatory documents on environmental chemicals.

Rohit is a Director at Eli Lilly & Company and is responsible for developing CMC regulatory strategies for oligonucleotides and oligonucleotide conjugates. Previously, he was a senior CMC reviewer at FDA for 5 years where he reviewed small molecules, oligonucleotides and ADCs. Rohit received 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 he 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.

Dan Hill is an Associate Director of PAT in Biogen’s Product & Technology Development organization. Dan brings 14 years of experience in PAT, and has a strong track record of developing and implementing process analytical technologies across non-GMP and GMP spaces in both Pharmaceuticals and Biopharmaceuticals. He leads a team of PAT experts responsible for advancing Biogen's process control initiatives, including the establishment of regulatory pathways for PAT acceptance. He also manages strategy execution of Biogen Product & Technology Development's Digital Evolution portfolio. Dan holds an MBA in Entrepreneurship from North Carolina State University and a Bachelors of Science in Biochemistry from Ball State University.

Dr. Kosuke Ito is principal reviewer at PMDA. Dr. Ito has experience in academia and government, including areas of review of oncology drugs, regulatory science of oligonucleotide therapeutics and regulatory affairs. Dr. Ito holds a PhD in Pharmaceutical Sciences from Osaka University.

Nina S. Cauchon, PhD, leads external engagement activities within RA-CMC. She has experience leading both early phase & commercial programs, including small molecules and biologics. Her areas of interest are regulatory challenges for innovative modalities and emerging technologies, CMC aspects of expedited review pathways, regulatory harmonization, and science and risk-based approaches to regulations. Nina is active in several external organizations which provide a strong network and knowledge base, including being a speaker/committee member for ISPE, CASSS, PQRI, AAPS, IQ, and DIA. She is a member of the ISPE International Board of Directors, the PhRMA Global Quality and Manufacturing group, and the ICH Q2(R2)/Q14 Expert Working Group.

Andreas Kuhn, Senior Vice President RNA Biochemistry & CMC Development, has worked in the field of RNA biochemistry and molecular biology for about 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 and vaccine.

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.

Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016. He is a Fellow of the American College of Physicians and in 2022 he was elected to the National Academy of Medicine.

Melissa has over 20 years of industry experience in QA, CMC and RA, across a broad range of lifecycle stages and therapeutic modalities, including small/large molecules, microbiome, and most recently oligonucleotides. She is currently Director of Regulatory Affairs and the Global Regulatory Lead for Alnylam Pharmaceuticals first approved GalNAc conjugate, Givlaari®. She initially led the regulatory CMC strategy for the NDA, MAA and JNDA, and is now overseeing global regulatory strategy for the commercial lifecycle. Previously, Melissa worked at Seres Therapeutics, Biogen, Sanofi (formerly Genzyme) and Alkermes. She received her B.S in Biology from Dickinson College, and her M.S in Regulatory Affairs and Health Policy from Northeastern.

Peter Stein, MD, is the Director of CDER’s Office of New Drugs (OND). OND is responsible for the regulatory oversight of investigational studies during drug development and decisions regarding marketing approval for new (innovator or non-generic) drugs, including decisions related to changes to already marketed products. OND provides guidance to regulated industry on a wide variety of clinical, scientific, and regulatory matters. A nationally-recognized leader in pharmaceutical research and development, Dr. Stein joined CDER in 2016 as the OND Deputy Director. Before coming to FDA, he served as Vice President for late stage development, diabetes, and endocrinology at Merck Research Laboratories.

Dr. Janet Woodcock began her long and distinguished FDA career in 1986. In 1994, Dr. Woodcock was named Director of the CDER. In that position, she has led many of the FDA’s groundbreaking drug initiatives. In 2020 Dr. Woodcock was asked to lend her expertise to “Operation Warp Speed” the initiative to develop therapeutics in response to the pandemic. Dr. Woodcock was named Acting Commissioner of Food and Drugs on January 20, 2021-February 17, 2022. Dr. Woodcock is now the FDA's Principal Deputy Commissioner. In this role she works closely with the Commissioner of Food and Drugs to develop and implement key public health initiatives and helps oversee the agency’s day-to-day functions.

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.

Tracey Burr is a Director of CMC Regulatory Affairs at Ionis Pharmaceuticals, Inc. and is responsible for Ionis’ early and late phase, as well as/commercial compounds. She has 20 years of drug development and global CMC regulatory experience, and has spent her career working at small to mid-size pharmaceutical and biotechnology companies. Tracey received a Ph. D. in Materials Science and Engineering from MIT, M.Sc. in Chemistry from Caltech, and BA in Chemistry from Wellesley College.

Dr. Carmi-Levy is Chief Scientific Officer and VP R&D at Aummune, a biotech company developing a personalized therapy for cancer. Prior to joining Aummune, she served as a Project Director in BioLineRx. She is a drug development expert and an immunologist by training. Dr. Carmi-Levy performed postdoctoral research at the Curie Institute and earned her PhD in Biochemistry and Molecular Biology from the Hebrew University.

Adam Dinerman, Ph.D., is Executive Director and CMC Head at Aro Biotherapeutics responsible for process, product, analytical development and manufacturing. Adam has over twenty years of experience in pharmaceutical development, technology transfer, and manufacturing. Prior to Aro Biotherapeutics, Adam was CMC Leader at Johnson & Johnson (Janssen R&D), leading multiple early and late stage CMC programs for a variety of biotechnology products. He also led a biologics drug product development group at Janssen R&D and contributed to multiple IND/CTA and BLA submissions. Prior to Johnson & Johnson, Adam worked at Genzyme in bioprocess development. Adam received his Ph.D. in Pharmaceutical Sciences from the University of Maryland at Baltimore.

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.

Dr. Kinoshita received a PhD in Pharmacology form Tokyo University. He has worked in former Tanabe Pharmaceuticals until 2000 as a CNS pharmacologist in the Discovery phase and moved to GSK as a Sr researcher supporting genomics projects for clinical materials. After starting working for Banyu/Merck as a research director, since 2006, he has begun an interface role as a regulatory specialist between research people in US/EU of Merck and Japanese authority for preclinical matters. Since 2014, he has lead the discussion group regarding preclinical safety for oligonucleotide therapeutics in JPMA and has supported to create Japanese guidelines. He has also been focusing on technical writing and translation efficiency during the above period.

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.

Anuradha Ramamoorthy, Ph.D. is a Policy Lead at the Office of Clinical Pharmacology (OCP), Food and Drug Administration (FDA). She received her Ph.D. in Medical and Molecular Genetics from Indiana University and was a postdoctoral fellow at the NIH and FDA. In her current role, she contributes to regulatory policy development, stakeholder engagement, and regulatory research focused on clinical pharmacology. Prior to this role, she was a Reviewer in the Genomics and Targeted Therapy, OCP contributing to the regulatory review of investigational new drug (IND), new drug application (NDA), and biologic licensing application (BLA).

Sriram Sathy is the Chief Scientific Officer (CSO) at Codiak Biosciences and a distinguished scientist with more than 23 years of research experience in cancer biology, immunology and immuno-oncology. He is an experienced drug developer and is responsible for bringing multiple drug candidates from concept to clinic across multiple therapeutic modalities including monoclonal antibodies, complex biologics, small molecules and antisence oligoneucliotides. His research team designed and led the preclinical evaluation of all of Codiak’s development programs in oncology/immuno-oncology, including exoIL-12™ and exoSTING™, which are currently in Phase 1/2 clinical trials, and exoASO™-STAT6, which has cleared the IND and will enter clinical develop.

Tom Thuren MD PhD Tom joined Novartis Pharmaceuticals in 2007 and is currently the Clinical Development Head for Metabolism, Lipids and Atherosclerosis. During his tenure at Novartis, Tom has held positions of increasing responsibilities in the Galvus (DPPIVi) team, new products development and as clinical lead for the CANTOS team to establish the role interleukin-1beta driven inflammation in atherosclerotic cardiovascular disease. Prior to joining Novartis, Tom was Senior Director of Cardiovascular Medicine Clinical R&D with Pfizer Global R&D where he led global clinical development for torcetrapib/atorvastatin. Tom is board certified in Internal Medicine and in Endocrinology and Metabolism. He did his internal medicine as we

Dr. Tsai is an Assistant Member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab’s research focuses on genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies such as sickle cell disease and T-cells for cancer immunotherapy. His group has recently developed CHANGE-seq, a state-of-the-art, sensitive, unbiased, high-throughput method for defining the genome-wide activity of genome editors. Previously, he has developed methods for high-throughput genome editing with TALENs, and for defining and improving the genome-wide specificity of CRISPR-Cas nucleases such as GUIDE-seq and CIRCLE-seq. Dr. Tsai completed a postdoctoral fellowship at Mass

Kristy Wood is currently VP of Nucleic Acid Therapeutics Development & Manufacturing at Intellia Therapeutics, which focuses on process development and manufacturing of our sgRNA, mRNA, and LNP platforms. Kristy started in 2017 at Intellia Therapeutics and was initially responsible for developing Intellia’s LNP platform and over-seeing delivery technologies for therapeutic applications of CRISPR/Cas9. She has more than 15 years of industrial experience in developing novel drug delivery systems for the delivery of nucleic acids. She received her Ph.D. in Biomedical Engineering from the University of Texas at Austin working with Professor Nicholas Peppas and her B.S. in Biomedical Engineering from the University of Wisconsin-Madison.

Annemieke Aartsma-Rus is a professor of translational genetics at the Leiden University Medical Center, the Netherlands. She is one of the pioneers of antisense oligonucleotide mediated exon skipping development for Duchenne muscular dystrophy. She currently applies the expertise she gained during 20+ years of exon skipping development to optimize this approach for Duchenne and to develop mutation-specific, N-of-1 exon skipping therapies for patients with progressive brain diseases.

Michael is a board certified medical doctor and internist with research interest in hepatology and viral hepatitis. He served as investigator in more than 100 clinical studies at University Hospitals of Hamburg, Berlin and Leipzig (Germany) before he joined Janssen in 2013. After positions in medical affairs, R&D programs of early and late development, Michael is the medical leader of the siRNA, JNJ-3989 related development program.

Anna holds a PhD in Biochemistry at Lund University together with a MBA from Gothenburg University and joined AstraZeneca in 2001 recruited as a molecular and cell biologist. Before joining AZ Anna spent time at a small biotech in Copenhagen, ALK with antigen production for immunization of humans and one of the world leading technology institutes, VTT in Helsinki, Finland with large scale protein production in collaboration with biotech Genencore, US. Anna has held a number of increasingly senior positions within AstraZeneca, working in all stages of the Drug Discovery and Development value chain and being part of marketed block buster successes such as Crestor, AstraZeneca´s own statin. Anna now leads the frontrunner mRNA VEGF program.

Brian Dooley has worked as a quality specialist in the Pharmaceutical Quality Office of EMA since 2016, working mostly on centralised marketing authorisations and scientific advice, and supporting the development of scientific guidelines by the CHMP, QWP and BWP. From 2008 to 2016, Brian worked as a pharmaceutical assessor in the IMB/HPRA (Ireland). He holds a B.Sc. in Pharmacy (2005) and M.Sc. in Pharmaceutical Medicine (2015) both from Trinity College Dublin, Ireland. Areas of interest: lifecycle management, assessment-inspection interface, synthetic peptides, oligonucleotides, mRNA technology, sterilisation processes, radiopharmaceuticals.

Mr. Duke has held leadership positions at several rare disease and immuno-oncology companies including Amicus Therapeutics, Advaxis, Inc., and NPS Pharma. Most recently, Mr. Duke led operations for Amicus K.K. in Japan including commercialization efforts for Galafold™ (migalastat) for Fabry disease, after previously serving as the company’s vice president of global commercial operations. He also was chief operating officer for Advaxis, Inc., and executive director of international commercial operations for NPS Pharma. Earlier in his career, Mr. Duke held clinical and scientific positions at Merck & Co. and The Schering-Plough Corporation.

Toby is a neuromuscular neurologist and neuroscientist who joined Biogen in 2013. His professional experience has been focused on developing treatments for neuromuscular disease, with a focus on ALS. He is currently the Head of the Neuromuscular Development Unit at Biogen and leads the neuromuscular clinical development group. Toby plays a key role in developing ALS clinical trials, and in driving preclinical strategy within neurodegenerative and neuromuscular disease. Prior to Biogen, Toby had a clinical neuromuscular neurology practice and trained in neurology and neuromuscular disease at the University of Pennsylvania. He obtained an MD and PhD (Neuroscience) at the University of Florida.

Jo started her career in the Clinical Pathology lab at Sanofi, ultimately as Laboratory Head. Jo then took a role as study director with responsibility for Toxicology studies at Covance, whilst maintaining an interest and specialism in clinical pathology data interpretation and choice of biomarkers on Toxicology studies. After gaining experience with a wide range of study types and modalities, Jo accepted a position as Project Toxicologist at AstraZeneca initially focussing on the non-clinical safety assessment of compounds for oncology indications. More recently she moved to the Department of Cardiovascular, Renal and Metabolism and has a particular interest in the development of antisense oligonucleotides with regard to safety assessment.

Rachel Johns, Ph.D., is currently Senior Director, Analytics and Formulations at Avidity Biosciences, Inc., where she has been since 2013. Rachel has been active in the field of oligonucleotide delivery since 2000, starting out as a nanoparticle and liposomal formulation scientist. She received her Ph.D. in Bioengineering from the University of Washington and started immediately in the biotech industry. Over the last several years, Rachel transitioned to specialize in CMC and currently leads the Analytical Development efforts for Avidity’s novel therapeutics.

A.Robert MacLeod, Ph.D- BIOSKETCH: Dr. MacLeod is currently CSO of Flamingo Therapeutics. Previously R. MacLeod held several positions at Ionis Pharmaceuticals and most recently Vice President and Franchise Head of Oncology. He and has contributed to the discovery and development of clinical drug candidates in the several therapeutic areas including, oncology, thrombosis, inflammatory and musculoskeletal diseases. Prior to joining Ionis Dr. MacLeod was Senior Director of Discovery Biology at Takeda Pharmaceuticals and was a founding scientist of MethylGene Inc. (now Mirati Therapeutics). He has led the strategic and scientific efforts of several multidisciplinary drug discovery teams leading to the discovery and clinical development of 15

Stefan McDonough, Ph.D., is vice president and head of neuroscience at Atalanta Therapeutics. Previously he served for five years as executive director, genetics at Pfizer, prior to which he spent 12 years at Amgen in scientific roles of increasing responsibility in the neuroscience department and as head of Amgen’s genome analysis unit. He holds a Ph.D. in biology from Caltech and completed postdoctoral studies at Harvard Medical School and the University of Maryland Medical School. He is author on over 40 peer-reviewed publications and is adjunct professor at the Carney Institute for Brain Science of Brown University.

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

Dr. Norris has over 23 years of experience with the past 15 years at Ionis Pharmaceuticals in Carlsbad, CA as Executive Director of Pharmacokinetics and Clinical Pharmacology with supervisory management responsibility for PK/ClinPharm, bioanalytical, and ADME scientists. In this capacity, he has project responsibilities on many development programs within the neurodegenerative franchise. He has authored nearly 30 papers and/or poster presentations as primary or co-author and presented at scientific conferences across the United States. His research interests include PK modeling of ASOs in the CNS. Dr. Norris received his Bachelor of Science in Pharmacy degree from Butler University and his Ph.D. in Pharmaceutics from Rutgers University.

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®.

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.

Paul Rohlfing is Exec. Director, Reg Affairs CMC, Pfizer, accountable for Global Regulatory CMC strategies for registration and post-approval of vaccines and other biologics including mRNA COVID vaccine, a 20-valent pneumococcal conjugate vaccine, >7 other development programs and 8 licensed products. Previously he developed production process improvements and new applications of technology, as well as transferred technology into production facilities, including processes for the licensure and commercial support of complex conjugate vaccines. He has also held various production and quality assurance roles with biologicals and classical pharmaceuticals in >25 years of experience with Pfizer, Novartis and Allergan.

Dr. Tsimikas is a practicing Board-Certified Cardiologist and Professor of Medicine and the Director of Vascular Medicine at the University of California San Diego School of Medicine, and Senior Vice President of Global Cardiovascular Development at Ionis Pharmaceuticals, Carlsbad, CA. He obtained his Medical Degree from the University of Massachusetts Medical School, Internal Medicine training at the University of Massachusetts Medical Center, and separate fellowships in Cardiovascular Medicine, Atherosclerosis and Molecular Medicine and Interventional Cardiology at the University of California San Diego (UCSD) Medical Center.

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.

Aniz Girach, is a qualified Ophthalmologist with 24 years industry experience in senior leadership roles with Lilly, Merck and Alcon, as well as being Chief Medical Officer at smaller biotech companies such as Oxurion and Nightstar Therapeutics. In addition to being an Honorary Professor at Wills Eye Hospital, Philadelphia, USA, he is currently the Chief Medical Officer at ProQR Therapeutics, leading the development of RNA therapies for genetic eye diseases. He is a member of 3 international Scientific Advisory Boards and a reviewer for 5 peer-reviewed journals. He has been Chief Editor of 4 books and published over 70 abstracts/manuscripts, with numerous invited lectures at national/international ophthalmology meetings.

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.

Erle is a computational biology research scientist at Deep Genomics, where she works on developing tools and methods to improve the design of steric-blocking oligonucleotides. She holds a master’s degree in statistics from the University of Waterloo.

David Lebwohl is the chief medical officer at Intellia Therapeutics, a genome editing company. Previously, David was the CMO at Semma Therapeutics and led drug development teams at Novartis and BMS. At Novartis, he was SVP and Program Head, CAR-T Teams, responsible for the breakthrough therapy Kymriah® (tisagenlecleucel), approved for leukemia and lymphoma. Under his leadership, the mTOR inhibitor Afinitor® (everolimus) was approved for metastatic breast, neuroendocrine, and kidney cancer and the rare genetic disease tuberous sclerosis. David received an AB in Biochemical Sciences from Harvard College and an MD from Yale. He was fellow in medical oncology and hematology at MSKCC and an internal medicine resident at the Brigham in Boston.

Dr. Donald Parsons is VP, Early Technical Development and Lipid Nanoparticle Process Development at Moderna Therapeutics. His team focuses on the process science behind the manufacture of lipid nanoparticles for mRNA delivery; and the development and scaleup of these processes. Additionally, he plays a matrix leadership role coordinating CMC activities for Moderna’s early-phase clinical pipeline; and leads Moderna’s small molecule process chemistry efforts. Prior to Moderna, Don spent six years with BIND Therapeutics, supporting the development of small molecule-loaded polymeric nanoparticles as Vice President, Pharmaceutical Development. Don has extensive experience in CMC development of complex drug delivery systems.

Branden Ryu is the founder, the chief executive officer, and the chairman of BIORCHESTRA. Branden earned his Ph.D. degree from the Graduate school of medicine at the University of Tokyo and he has experience as a researcher at Harvard medical school. After his research career, he found BIORCHESTRA to focus on developing RNA-based therapeutics in 2016. He has discovered the novel RNA biomarker and invented the novel ASO modality to treat neurodegenerative diseases. Under his leadership, BIORCHESTRA has expanded the new RNA modalities to cure neurological disorders including genetic rare diseases. Based on these scientific results, he has filed more than 70 patent applications since 2017.

Dr. Daniel J. Siegwart is an Associate Professor in the Department of Biochemistry and the Simmons Comprehensive Cancer Center (SCCC) at UT Southwestern Medical Center (Dallas, Texas, U.S.A.). He is also the co-director for the Chemistry and Cancer Program within the National Cancer Institute-designated SCCC. He received a B.S. in Biochemistry from Lehigh University (2003), and a Ph.D. in Chemistry from Carnegie Mellon University (2008) with University Professor Krzysztof Matyjaszewski. He also studied as a Research Fellow at the University of Tokyo with Professor Kazunori Kataoka (2006). He then completed a Postdoctoral Fellowship at MIT with Institute Professor Robert Langer and Professor Daniel G. Anderson (2008-2012).

Received B.Sc. (Hon) in Biochemistry/Biotechnology from the University of Ottawa, and Ph.D. in Immunology from the University of Glasgow. In 2001, joined Health Canada as a Quality reviewer at the Monoclonal Antibodies Division, and more recently at the Gene Therapies Division.

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.