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 Senior Director of Regulatory Affairs CMC Policy and Advocacy at Alnylam Pharmaceuticals with nearly two decades of experience across drug discovery, global regulatory CMC strategy, and policy development. He has held senior roles at Alnylam and GSK, served as an Acting Branch Chief in FDA CDER’s Office of New Drug Products, and worked in medicinal chemistry R&D at Pfizer and Merck. His expertise spans small molecules, biologics, oligonucleotides, combination products, and emerging technologies. Ben holds a PhD in Chemistry and an MPH, and is a co-author of 30+ publications and patents. He provides strategic leadership on global CMC advocacy, supporting RNAi therapeutics from development through lifecycle management widely.

Jeff Foy is the Executive Director of Toxicology at Nimbus Therapeutics. He has been involved in the oligonucleotide field for over 20 years with experience in the development of aptamers, siRNAs, and peptide-conjugated oligonucleotides. Jeff chaired the Oligonucleotide Safety Working Group (OSWG) for over 10 years. He received his bachelors degree from the University of Vermont and his PhD from Northeastern University.

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 25 years ago and has spent the last 20 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.

Dr. Sydney Stern is an independent consultant and the Associate Director of Medical Writing, Publications, and Medical Communications at BeOne Medicines, Ltd. She is a former clinical pharmacology reviewer in the Division of Translational and Precision Medicine in the Office of Clinical Pharmacology (OCP) at the FDA. She was a primary reviewer for oligonucleotide programs, surrogate endpoints, and rare diseases in OCP. Dr. Stern has led data projects in the rare disease space and research projects investigating strategies for selecting safe starting doses in oligonucleotide-based therapeutic. She received her Master of Science in Clinical Research and a PhD in Pharmaceutical Sciences at University of Maryland Baltimore.

Patrik received his PhD in toxicology from Karolinska Institutet, Stockholm in 2003. He 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 oligonucleotide platform in AstraZeneca with a focus on off-target assessment and regulatory guidelines.

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 at CDER, FDA. Before joining CDER, she served as a Senior Consultant in the Federal Healthcare Practice at Deloitte Consulting LLP. She holds a Ph.D. in Molecular Genetics from Baylor College of Medicine and completed her postdoctoral training in Developmental Biology at Columbia University Medical Center. Dr. Chi currently serves as the FDA Topic Leader in the Expert Working Group of ICH-S13.

Tae-Won Kim, PhD, is Vice President of Preclinical Development at Ionis Pharmaceuticals. He received his PhD in Environmental and Molecular Toxicology from North Carolina State University and completed postdoctoral training at the University of California, San Francisco, focusing on carcinogenesis and tumor metastasis. At SUGEN, he served as toxicologist and Group Leader on the SUTEN project. At Ionis, he led the nonclinical development of six approved therapies—mipomersen, inotersen, volanesorsen, eplontersen, olezarsen, and donidalorsen—and oversees antisense oligonucleotide and siRNA programs. His work focuses on nonclinical safety assessment and development strategy for oligonucleotide therapeutics.

Aimee is currently a CXO at Curie Bio. Prior to joining Curie, she was the Chief Scientific Officer for Atalanta Therapeutics where she was responsible for building and directing a fully integrated organization to identify and develop divalent siRNAs for CNS indications. Aimee has worked in the field of oligo therapeutics for ~25 years. She was the VP of Research for miRagen Therapeutics, advancing multiple microRNA programs from initial concept to FIH clinical trials. She was led preclinical research at Regulus Therapeutics, and was the Molecular Profiling Lead for siRNA therapeutics. Known for identifying siRNA off-target effects, she played a lead role in siRNA design and chemistry for enhanced activity and specificity.

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.

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.

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.

Rumi Young is the Director of Regulatory Policy at Novo Nordisk, ensuring future growth and innovation by promoting effective biopharmaceutical policies. Previously, she led BD’s Global Regulatory Policy team to shape future policies for medical devices, diagnostics, and combination products. As Assistant Director – Injection Devices at FDA, she managed reviewers and set technical and regulatory expectations for combination products and drug delivery devices. Before FDA, Rumi worked in R&D for a number of years at Genentech and AstraZeneca. She holds a Bachelor’s in Chemistry and Chemical Biology and a Masters in Material Science Engineering from Cornell University.

Physician, trained in Internal Medicine, Endocrinology, and Reproductive Endocrinology at McGill and Harvard universities, with over 30 years of industry experience including as founding CMO in a number of successful and now-public companies including Radius Health, Moderna, Akcea and Biorchestra. With multiple drug approvals in the fields of reproductive, metabolic and rare genetic diseases, he currently serves as board chairman of a UK biotech and board member and strategic advisor to other emerging US and UK companies.

Dan trained on the MD PhD programme at UCL, completing a PhD in genetic epidemiology. Thereafter he worked in London as a clinical academic in internal medicine and clinical pharmacology. In his academic research he led international human genetics consortia for drug target discovery in cardiometabolic disease, with findings published in the Lancet and Nature Communications. Before joining GSK in 2022, Dan led oligonucleotide clinical development programmes and a computational genomics group at Silence Therapeutics, prior to which he worked in AI-enabled translational and precision medicine at BenevolentAI. At GSK, Dan leads early oligo and cardiovascular clinical development programmes. He is an honorary associate professor at UCL.

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.

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.

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. In 2012 he joined Biogen and rose to be Head of Oligonucleotide Development. Since 2024, he has served as Vice President of Oligonucleotide and Small Molecule CMC at Denali Therapeutics.

Brian Doyle has over 15 years of experience in technical and CMC development for vaccines and therapeutics. Since joining Moderna in 2019, Brian has led organizations focused on late-stage and commercial process development and CMC technical writing, with a particular interest at the interface between process and regulatory science related to advanced manufacturing and platform-based approaches. Prior to joining Moderna, Brian held roles in roles in cell culture and fermentation process development at Gilead Sciences and Merck. Brian holds a B.S. in Chemical-Biological Engineering from the Massachusetts Institute of Technology.

Ami Mankodi is a lead physician in the Division of Neurology 2 in the FDA’s Office of Neuroscience in CDER and serves as a clinical and cross-discipline team lead for IND and NDA/BLA reviews for rare diseases and neuromuscular diseases. She serves as a subject matter expert for the CDER Center for Clinical Trial Innovation programs, rare disease endpoint advancement, clinical outcome assessment qualification and biomarker development, FDA representative to the Critical Path Institute Duchenne Regulatory Science, and internal and external grant reviews. Mankodi is a Board-certified neurologist and completed neurology residency at the Johns Hopkins Hospital and post-doctoral fellowships at the University of Rochester, NY, and NINDS at NIH.

Sorcha McCrohan is a Senior Scientific Project Manager for Global Science 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 franchise. Sorcha holds a BA in Sociology from Mount Holyoke College and an MSc in Global Health, Disease Prevention & Control from Georgetown University.

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. Jen Sisler is a board certified toxicologist and a Director of Toxicology at Eli Lilly. As toxicology project lead on numerous ONT programs, she directs nonclinical safety strategy in close collaboration with cross-functional teams, ensuring that program approaches keep pace with the scientific and regulatory complexity of this emerging therapeutic class. Dr. Sisler currently serves as co-chair of the IQ DruSafe ONT Working Group, which focuses on industry standards for ONT drug development, and as the PhRMA alternate topic lead to ICH S13 Expert Working Group. She is also an active member of the American College of toxicology, where she has served on committees for the past six years and currently holds an elected position on the Award

Dr. Xing Jing is an expert in oligonucleotide-based therapeutics. He possesses a broad spectrum of experiences from biology to clinical trials. Currently, Dr. Jing is a reviewer of cell and gene therapies in CBER Office of Clinical Evaluation at the FDA. Prior to that, Dr. Jing was a clinical pharmacology reviewer in CDER Office of Clinical Pharmacology at the FDA since 2020. Dr. Jing's regulatory review experience covers a wide range of therapeutic modalities and disease areas. Before FDA, he was trained as a biologist.

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 drugs Farydak® and Kisqali®.

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.

Ron is a Principal Consultant at Aclairo, which he joined in 2025 after a 20-year career in the Office of New Drugs (OND) in CDER at the FDA. He served as a Pharm-Tox reviewer, a Pharm-Tox supervisor and an Associate Director for Pharm-Tox. In this latter role, he was a member of the leadership team for the Pharm-Tox discipline in OND and provided expert scientific and regulatory policy support for all Pharm-Tox and Clinical Review Divisions. He has played key roles in the development of multiple FDA guidances related to the nonclinical development of drugs, including oligonucleotide-based therapeutics, rare diseases, developmental and reproductive toxicity, immunotoxicity, and the use of nonhuman primates.

Susanne Brendler-Schwaab studied biology and earned her Ph.D. in the field of genotoxicity. In 1990, she joined Bayer Healthcare as study director in the toxicology division. Since 2004, she has worked as senior toxicologist, regulatory affairs expert and project manager, heading several units at the Federal Institute for Drugs and Medical Devices (BfArM) in Bonn. From 2012, she has served as the German mem-ber of the former “Safety Working Party (SWP)” at EMA, becoming its vice-chair in 2019 and chair in 2021. In 2022, she was elected as chair of the newly established Nonclinical Working Party (NcWP) at EMA, a position she continues to hold today. In April 2024, she was appointed rapporteur of the new ICH S13 Expert Working Group.

Bryan Laffitte is the Head of Genetic Medicine and leads the Neuromuscular portfolio for the Neuroscience Disease Area at Novartis. He is currently the Head of Research Integration for Avidity Biosciences and previously was Site Head for Kate Therapeutics. Bryan rejoined Novartis in 2023 through the acquisition of DTx Pharma where he was the Chief Scientific Officer. DTx Pharma developed delivery technologies for RNA therapeutics and Bryan helped to define their strategy, shape their portfolio, and advance the platform leading to the acquisition by Novartis. Prior to DTx, he was the Vice President of Biology at Inception Therapeutics, part of Versant Ventures, and helped to start multiple biotechnology companies.

Dr. Wayne Doyle earned his PhD at UT Southwestern and completed his postdoctoral training at UC San Diego. He previously worked at Active Motif, where he led the bioinformatics team, supported biopharma partners, and developed analytical strategies for emerging technologies. Dr. Doyle joined Eclipsebio in 2022 and now leads the development of the company’s platform for RNA therapeutics development support, including AI-powered design and end-to-end mRNA characterization solutions.

Jenny has worked for Ionis Pharmaceuticals, Inc. for 19 years and for the past nine has been responsible for CMC Regulatory Affairs encompassing Ionis’ early development programs through marketing authorization and commercial lifecycle management. Prior to that, she was responsible for Quality Assurance oversight of oligonucleotide drug substance manufacturing and testing activities, and associated functions. She earned her B.S in Biology and French from the University of Wisconsin.

VP, Global Head of CMC & Regulatory at Hongene Biotech, where Dr. Wu leads global CMC and regulatory strategy and oversees the transformation of the company's quality systems to support GMP manufacturing. With 25+ years of experience in the global biotechnology and pharmaceutical industry, prior to joining Hongene, she served as VP of CMC and then Head of R&D at Sperogenix Therapeutics. Earlier in her career, Yansheng held roles in oligonucleotide PD/AD, QC and external quality at Bayer, Archemix, and Baxter. She explored a wider variety of modalities from small molecule drugs, oligonucleotide and mRNA, biosimilars, biologics to gene therapies through R&D to commercial stage products at Baxter, Baxalta, Shire, and Alexion.

Julian oversees the portfolio that is designed to ensure the quality and access of products to the UK market - this includes scientific advice, licensing assessment, marketing authorisations for all innovative and established medicines. A committed professional with over 15 years of Director/Board level experience with a broad demonstrated history at Medicines Healthcare Products Regulatory Agency, Novartis, Pfizer and GSK in pharmaceuticals. Flexibly skilled in regulatory, compliance, quality, health and safety, operations with passion for people development with results and improvement focus. Quickly adaptable with strong links to industry and regulatory bodies with country, scientific, commercial and business focuses.

Teresa Buracchio, MD, is Director of the Office of Neuroscience in the Center for Drug Evaluation and Research, Food and Drug Administration (FDA). She oversees the review of new drug programs for neurologic and psychiatric diseases. Dr. Buracchio joined FDA in 2013. Dr. Buracchio completed her medical degree and neurology residency at Rush University Medical Center in Chicago, Illinois. Dr. Buracchio completed fellowship training in geriatric neurology at Oregon Health & Science University and Veterans Affairs Medical Center in Portland, Oregon.

Mike Dombeck joined EnFuego as CEO and Chairman in August 2025. A proven biotech growth leader, Mike’s accomplishments span nearly $10b in partnering value; two IPOs raising $274m; and successful management of the growth of companies and advancement of programs from discovery into late clinical development. His most recent role was as Chief Operating Officer (COO) for Arcellx, Inc., a CAR-T company acquired by Gilead in April 2026 for $8b. Prior to Arcellx, he led Corporate Development for Precision BioSciences, Inc., securing over $2b in partnerships and being recognized by the Licensing Executives Society with their 2016 Deal of Distinction award. Mike holds a BS in Biology, a Masters of Public Policy, and an MBA from Duke University.

Dr. YanLing He is Global TM Profiling Head, CVM, at Novartis Biomedical Research in Boston, MA. Previously, she was a visiting assistant professor at MGH, Harvard Medical School. Her clinical research focuses on clinical pharmacology and translational medicine in cardiovascular and metabolic diseases. She has published more than 70 peer-reviewed articles and led IND filings for eight novel or first-in-class new molecular entities, as well as first-in-human and proof-of-concept clinical trial designs and publications. She also plays a leadership role in clinical pharmacology studies, advancing mechanistic understanding and supporting drug labeling. She is passionate about delivering innovative medicines for patients in need.

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.

RR is Director of Toxicology at Denali Therapeutics, where she leads nonclinical safety assessment for oligonucleotide therapeutics utilizing Denali’s Transport Vehicle (TV) platform for targeted delivery across the blood–brain barrier. She has extensive experience across siRNA therapeutics, small molecules, and biologics, advancing drug candidates from discovery through clinical development. Prior to Denali, RR held leadership roles at ADARx Pharmaceuticals and Sorrento Therapeutics, where she led toxicology strategies for programs spanning CNS diseases, pain, oncology, cardiovascular, and rare diseases. RR earned a Ph.D. in Neuroscience from the University of Copenhagen, Denmark.

Deputy Director for CDER/OPQ's Office of Policy for Pharmaceutical Quality. He joined the Agency in November 2009 after graduating with his PharmD from the University of Maryland and his MBA from the University of Baltimore. Prior to FDA, he had experience in the generic pharmaceuticals industry. He continues to practice pharmacy in the community setting.

Susann Rosmus is „Head of Quality Assurance“ at BioSpring GmbH. Susann did her PhD in Biochemistry and joined BioSpring in 2000 for research and development in the field of gene synthesis. In 2005 Susann became head of QC, where she developed and established analytical methods for oligonucleotides. In 2007 Susann started directing quality assurance and regulatory affairs and is now responsible for compliance and improvements in this field.

Christine (Kris) Siezen is a senior non-clinical assessor at the Medicines Evaluation Board (MEB) of the Netherlands with a research PhD focusing on cancer pathways. Her experience as an assessor, and application of ICH safety guidelines, goes back 19 years with specific focus on carcinogenicity studies and their value in risk assessment of human medicines. Another focus area is safety evaluation of oligonucleotide-based therapeutics. In addition she is a long-standing member of EMA’s Non-Clinical Working party and of various Operational Expert Groups. Since the kick-off in 2024, she is the European topic lead in the Expert Working Group of the ICH S13 guideline on oligonucleotide therapeutics.

Dr Thornton is a physician scientist and specialist studying neuromuscular disease at the University of Rochester. His focus is on mechanisms and therapeutic development for myotonic dystrophy. He serves as co-director of the University of Florida/ University of Rochester Wellstone Muscular Dystrophy Research Center, one of 6 NIH-designated Centers of Excellence for research on muscle diseases.

Erin has worked at Moderna in Analytical Development for the past four and half years, focusing on analytical strategy and clinical and commercial regulatory filings for mRNA products. Prior to joining Moderna, she spent approximately 20 years in the biotech industry, with roles in Quality Control, Analytical Development, and Manufacturing Sciences, primarily supporting biologics.

Dr. Benson received a Bachelor of Science B.S. from Massachusetts Institute of Technology (M.I.T.) before attending Indiana University, where he received a M.D. and a Ph.D., the latter in Physiology and Biophysics. He then completed a residency in Internal Medicine at the Scripps Clinic, La Jolla, California. He is currently a vice-president at Eli Lilly and company where he has spent over 26 years in early phase clinical drug development, including Clinical Pharmacology. He is internationally recognized for his leadership in applying quantitative approaches to early drug development, particularly his pioneering work in using concentration-response relationships for QT interval measurement.

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.

Katie Duncan is a Director of CMC Policy and Advocacy with Global Regulatory Affairs/CMC Excellence. Prior to joining GSK, Katie was a senior pharmaceutical quality assessor with the Office of Pharmaceutical Quality at the US Food and Drug Administration. She previously worked in small molecule drug discovery at a biotechnology company in San Diego, CA. She received her Ph.D. in organic chemistry from the Scripps Research Institute in La Jolla, CA and B.A. from Amherst College in Amherst, Massachusetts.

Ronen Eavri, is the CEO and Co-founder of Barcode-Nanotech which develops nano-based delivery vehicles for next-gen therapeutics. Prior to his current role, Ronen served as a R&D manager at J&J for over 5 years, leading drug and device development from early to advanced stages. Ronen holds a Ph.D. in Biochemistry from the Hebrew University, followed by a Post doc at MIT, and an MBA from the Technion.

Toby is a neurologist, scientist, and drug development leader with deep expertise in neuroscience and oligonucleotide therapeutics. He currently serves as Senior Vice President and Neuroscience Therapeutic Area Head at Alnylam. Prior to joining Alnylam in 2026, Toby spent a decade at Biogen, where he led teams focused on neuromuscular and movement disorders and helped advance the tofersen program for SOD1 ALS from research through approval. He later served as Chief Medical Officer at Voyager Therapeutics, building the clinical development organization and advancing multiple gene therapy and antibody programs for neurological diseases into clinical development.

Dr. Kelle Franklin is a board-certified toxicologist and Director of Toxicology at Eli Lilly and Company. Kelle holds undergraduate degrees from Johns Hopkins and a Master's and Ph.D. in Addictions Neuroscience from Purdue University. She completed postdoctoral training in Behavioral Neuroscience at Indiana University School of Medicine, before joining Labcorp as a Toxicology Study Director. Now at Lilly, she leads nonclinical safety strategy for novel CNS-shuttling platforms and is the company's subject matter expert in nonclinical abuse liability. Kelle is a member of the American College of Toxicology and the Cross-Company Abuse Liability Council, and co-chairs the IQ DruSafe CNS Delivery working group.

Dr. Steve Hughes is the Chief Medical Officer of Atrium Therapeutics, with more than 25 years of experience in the biopharmaceutical industry. He has contributed to more than 50 clinical trials and multiple product filings and launches across cardiovascular, neurology and rare disease therapeutic areas at Avidity Biosciences, Ionis Pharmaceuticals, Biogen, CSL Behring and Sanofi. His previous positions include the Chief Medical Officer at Avidity, Arcturus and Organovo, Chief Clinical Development Officer at Ionis Pharmaceuticals and clinical leadership positions at Biogen, CSL Behring and Sanofi.

Ditte Jæhger, PhD, is CEO and co-founder of Hazel Therapeutics, a biotechnology company developing locally delivered RNA therapeutics for osteoarthritis and other chronic diseases. She has more than 10 years of experience in immunology, biomaterials, and translational drug development across academia and industry. Prior to founding Hazel, she led translational research programs at the Technical University of Denmark (DTU), with a particular focus on cell and gene therapies. In this role, she designed and led preclinical development programs for several US-based biotechnology companies, including Repertoire Immune Medicines, Tidal Therapeutics, and Ceptur. Her current work focuses on advancing localized oligonucleotide therapies.

Dr. Sally Jiao joined the Synthetic Molecule Design and Development department at Eli Lilly in Feb 2024 and works on upstream process development, PAT, and modeling for oligonucleotide assets, focusing on the fluidized bed reactor (FBR) technology. Sally holds a PhD in chemical engineering from the University of California, Santa Barbara, and a BS in chemical engineering from Princeton University. Sally has received Lilly Innovator Awards for her work on enzymatic ligation and PAT.

Carolyn is an Executive Director in the Analytical Development and Quality Control department at Ionis Pharmaceuticals. She is responsible for the analytical activities for oligonucleotide therapeutics in all phases of development. Her expertise includes method development, validation, stability testing, establishing specifications, authoring and defending regulatory submissions, and supporting commercial supply chains. Prior to joining Ionis, she worked at Dart NeuroScience, Gilead Sciences, and Vertex Pharmaceuticals. She received a B.S. in Chemistry from the University of North Carolina at Chapel Hill and Ph.D. in Chemistry from the University of Texas at Austin.

Klaus Romero MD, MS, FCP is a clinical pharmacologist and epidemiologist by training, with 15 years combined experience in academic clinical research. Dr. Romero has been with C-Path since December of 2007, and during his tenure, he has helped lead clinical pharmacology, pharmacoepidemiology and modeling and simulation projects in Alzheimer’s disease, polycystic kidney disease, tuberculosis, type 1 diabetes, Parkinson’s disease, Duchenne muscular dystrophy, kidney transplantation and Huntington’s disease, achieving major milestones such as the first regulatory endorsement by FDA and EMA of a clinical trial simulation tool for mild and moderate Alzheimer’s Disease and the qualification of an imaging prognostic biomarker for PKD.

Pooja Dua is Senior Director of Alliance Management at OliX Pharmaceuticals and brings in over 15 years of experience in RNA therapeutics and translational science. She holds a PhD in Cancer Biology and completed postdoctoral research in RNAi variants and aptamers for oncology. Previously, as Principal Scientist and Head of R&D at OliX US, she led development of asymmetric GalNAc siRNAs and spearheaded company’s dual-targeting siRNA platform. Leveraging her R&D expertise, in her current role she manages strategic partnerships with leading biotech and pharmaceutical companies.

Debra is an accomplished biotechnology executive with more than 25 years of industry experience. She currently serves as Chief Regulatory Affairs Officer at Dyne Therapeutics. Prior to Dyne, Debra served as Vice President of Regulatory Affairs at Sage Therapeutics, where she played a key leadership role in the filing and approval of Zulresso® (brexanolone), the first FDA-approved treatment for postpartum depression. Earlier in her career, she held regulatory leadership positions at Medivector, FoldRx Pharmaceuticals (a Pfizer company), AMAG Pharmaceuticals, and EPIX Pharmaceuticals. She holds a Master of Public Health from the Boston University School of Public Health and a B.A. in Economics from the University of Massachusetts Amherst.

Dr. Kerr is the Chief Medical Officer at Dyne Therapeutics. He is a neuroscientist and neurologist by training with a ten-year career as a faculty member at the Johns Hopkins School of Medicine before a 20-year career at present in the Boston area creating and serving in a series of biotechnology companies in the genetic disease and neurology/neuromuscular space.

Alfica is the CSO of Judo Bio working on delivering oligos to kidney for therapeutic intervention. Over the years she has held multiple leadership positions at CAMP4 therapeutics and Alnylam Pharmaceuticals, spanning from early discovery, new target search, platform biology, improvising delivery and leading programs to the clinic. Her experience spans rare liver diseases like Alpha-1 antitrypsin deficiency (Belcesiran), Bleeding disorders (Fitusiran); UCD; kidney disease, neurodegenerative and neurodevelopment disorders. Alfica graduated from TIFR, Mumbai; received her post-doctoral training at Johns Hopkins and Yale University, studying lipid uptake, cholesterol and oxygen homeostasis in parasites, mammalian cells and

Mark Stewart, PhD, is Vice President of Science Policy at Friends of Cancer Research, a Washington, DC-based organization that brings together stakeholders to advance science, policy, and regulation in cancer care. He leads the organization’s research and policy agenda, overseeing initiatives that inform regulatory decision-making and accelerate access to safe and effective cancer therapies. His work has helped advance the use of real-world evidence, improve clinical trial design, support biomarker development, and promote regulatory innovation to accelerate access to next generation therapies.

Cecilia Tami is currently the Head of US Technical Regulatory Policy at Genentech-Roche. In her role, she leads and executes US CMC regulatory policy strategies and advocacy plans and external outreach in technical, regulatory and legislative CMC related topics. Before joining Genentech-Roche, Cecilia served for over 12 years in the Office of Biotechnology Products (OBP) in CDER, US FDA, where she held positions of increasing responsibilities performing and then overseeing CMC/product quality assessments of regulatory submissions at all phases of development. Cecilia is active in several expert groups in PhRMA and BIO trade associations and is part of the ICH Expert Working Group revising the specifications guideline.

Sandor Batkai, MD, PhD, is a physician-scientist, biotechnology company builder, and strategic clinical development leader. As Medical Director at Resalis Therapeutics, he combines company-level strategy with hands-on leadership of oligonucleotide programs in metabolic disease. He has more than 20 years of experience across cardiovascular and metabolic research, translational medicine, biomarkers, regulatory strategy, and early clinical development. He co-founded Cardior Pharmaceuticals and helped advance its lead non-coding RNA therapeutic from discovery into clinical development before its acquisition by Novo Nordisk in 2024. He previously held leadership roles at the NIH and Hannover Medical School and is also a co-founder of GenKardia.

Dr. Daniel Butnaru is an AI/ML scientist focused on oligonucleotide design. He spent three years as the RNA Data Science Lead in the RNAHub at Roche, building end-to-end computational tools for oligonucleotide design — spanning sequence selection, chemical modification, and safety assessment. Over eight years at Roche, he has driven the application of machine learning and AI to RNA therapeutics across research and platform roles. Daniel holds a PhD in Computational Science and Engineering from the Technical University of Munich.

Takasumi Shimomoto is Associate Senior Scientist for Toxicology in PMDA. He has 30 years of experience in pharmaceutical industry and regulatory authority, focusing on toxicology and toxicologic pathology. Takasumi holds veterinary license and PhD in Medicine. His current work includes pharmaceutical product consultations and reviews. In his free time, he enjoys strolling around the suburbs and exploring delicious food with my wife.