Overview - mRNA Health Conference Speakers and Chairs
Keynote Speaker: Anthony S. Fauci
Anthony S. Fauci, M.D., is director of the National Institute of Allergy and Infectious Diseases (NIAID) at the U.S. National Institutes of Health, where he oversees an extensive research portfolio devoted to preventing, diagnosing, and treating infectious and immune-mediated diseases. Dr. Fauci has been a key advisor to six Presidents and their administrations on global HIV/AIDS issues, and on initiatives to bolster medical and public health preparedness against emerging infectious disease threats such as pandemic influenza and coronavirus disease 2019 (COVID-19). As an HIV/AIDS researcher he has been involved in the scientific effort since AIDS was recognized in 1981, conducting pivotal studies that underpin the current understanding of the disease and efforts to develop therapies and tools of prevention. Dr. Fauci was one of the principal architects of the President’s Emergency Plan for AIDS Relief (PEPFAR), which has helped save millions of lives throughout the developing world.
Dr. Fauci is a member of the US National Academy of Sciences, the US National Academy of Medicine and many other professional societies. He is the recipient of numerous awards for his scientific and global health accomplishments, including the Presidential Medal of Freedom (the highest honor given to a civilian by the President of the United States), the National Medal of Science, and the Mary Woodard Lasker Award for Public Service. He has been awarded 45 honorary doctoral degrees and is the author, coauthor, or editor of more than 1,300 scientific publications, including several major textbooks.
Keynote Speaker
Monday, 10:05 – 10:30 EST (recorded)
Daniel G. Anderson
Daniel G. Anderson is a Professor in the Department of Chemical Engineering, the Institute for Medical Engineering and Science, the Koch Institute for Integrative Cancer Research, and the Harvard-MIT Division of Health Science and Technology at MIT. The research done in Prof. Anderson’s laboratory is focused on developing new materials for medicine. He has pioneered the development of smart biomaterials, and his work has led to advances in a range of areas, including medical devices, cell therapy, drug delivery, gene therapy and material science. Prof. Anderson received a B.A. in mathematics and biology from the University of California at Santa Cruz and a Ph.D. in molecular genetics from the University of California at Davis. His work has resulted in the publication of over 400 papers, patents and patent applications. These advances have led products that have been commercialized or are in clinical development, as well as to the foundation of companies in the pharmaceutical, biotechnology, and consumer products space. Dr. Anderson is a founder of Living Proof, Olivo Labs, Crispr Therapeutics (CRSP), Sigilon Therapeutics, Verseau Therapeutics, Orna, and VasoRx.
Session II: Delivery
mRNA Delivery
Monday, 11:00 – 11:20 EST
Stéphane Bancel
Stéphane Bancel has served as Moderna’s Chief Executive Officer since October 2011 and as a member of Moderna’s board of directors since March 2011. Before joining the Company, Mr. Bancel served for five years as Chief Executive Officer of the French diagnostics company bioMérieux SA. From July 2000 to March 2006, he served in various roles at Eli Lilly and Company, including as Managing Director, Belgium and as Executive Director, Global Manufacturing Strategy and Supply Chain. Prior to Lilly, Mr. Bancel served as Asia-Pacific Sales and Marketing Director for bioMérieux.
Mr. Bancel currently serves on the board of directors of Qiagen N.V. and Indigo. He is currently a Venture Partner at Flagship Pioneering and a trustee of the Museum of Science in Boston. Mr. Bancel holds a Master of Engineering degree from École Centrale Paris (ECP), a Master of Science in chemical engineering from the University of Minnesota, and an M.B.A. from Harvard Business School.
Welcome and Logistics
Monday, 08:00 – 08:15 EST
Keynote Introduction
Monday, 09:55 – 10:00 EST
Patrick Baumhof
Patrick Baumhof, PhD
Vice President Formulation & Delivery
trained in Chemistry, at the University of Leipzig. His scientific expertise includes chemistry, pharmaceutical sciences and immunology. He joined CureVac in July 2007 when he was responsible for the development and preclinical testing of new formulations for mRNA vaccines and therapeutics. He is inventor of several patents and he co-authored several publications on mRNA technology. Currently he is heading the department for Product design an formulations and is Program manager for the RNAoptimizer Program.
Session IV: Manufacturing, Analytics and Commercialization
Chair
Monday, 15:10 – 16:30 EST
Norman W. Baylor
Dr. Norman W. Baylor is the President and CEO of Biologics Consulting Group, Inc. responsible for managing a team of consultants who provides national and international regulatory, clinical and product development advice on the commercial production of biological, pharmaceutical and device products. Prior to joining Biologics Consulting, Dr. Baylor was the Director of the Office of Vaccines Research and Review (OVRR) in the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research. During his 20-year tenure at FDA he evaluated and facilitated the development and licensure of numerous vaccines including acellular pertussis, varicella, pneumococcal conjugate, human papillomavirus (HPV), influenza, and Zoster. Dr. Baylor served as FDA’s liaison member of CDC’s Advisory Committee on Immunization Practices, the US Department of Health and Human Services National Vaccine Advisory Committee, the Advisory Commission on Childhood Vaccines, and a member of the National Institutes of Health NIAID Advisory Council. He also serves as an expert advisor to the World Health Organization on global vaccine initiatives.
Session VIII: Regulatory Considerations
The Regulatory Landscape for mRNA Vaccines: A U.S. Perspective
Tuesday, 14:45 – 15:00 EST
Session VIII: Regulatory Considerations
Round Table Discussion with Drs.Round Table Discussion with Drs.Baylor and Pfleiderer, and Regulatory Heads Charbel Haber from Moderna, John Talian from CureVac and Andreas Kuhn from BioNTech
Tuesday, 15:15 – 16:15 EST
Zwi N. Berneman
Zwi N. Berneman, MD PhD FRCP is Head of the Laboratory of Experimental Hematology at the University of Antwerp, Antwerp, Belgium and Head of the Division of Hematology at the Antwerp University Hospital. His basic and clinical research is focused on vaccination with immunogenic dendritic cells in cancer and with tolerogenic dendritic cells in multiple sclerosis. His laboratory has pioneered mRNA electroporation as a clinically safe gene transfer methodology and has applied it to the fields of dendritic cells and T-lymphocytes. He has been conducting clinical trials with cultured dendritic cells since 2005. For this, he helped establish the Center for Cell Therapy and Regenerative Medicine (CCRG), of which he is the Medical Director, at the Antwerp University Hospital and for which he obtained a Good Manufacturing Practices (GMP) Certification and Production License from the (Belgian) Federal Agency for Medicines and Health Products. He is a Fellow of the Royal College of Physicians, London, UK.
Session III: Prophylactic and Therapeutic Vaccines
Therapeutic vaccination of cancer patients with WT1 mRNA-electroporated dendritic cells: Using mRNA as an active substance in the clinic since 2005
Monday, 14:10 – 14:25 EST
Horacio Cabral
Horacio Cabral is an Associate Professor in the Department of Bioengineering, Graduate School of Engineering, The University of Tokyo. He received his PhD in Materials Engineering from The University of Tokyo, in 2007 under the supervision of Prof. Kazunori Kataoka. Dr. Cabral was an Assistant Professor at the Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, The University of Tokyo until 2010, when he joined the Department of Bioengineering of The University of Tokyo as a Lecturer. In 2014, he was promoted to his current position. Dr. Cabral’s major research interests relate to the development of nanomedicines for diagnosis and therapy, particularly systems directed to intractable cancers. His work has provided basis for several nanomedicines aimed for tumor-targeted therapy and imaging, showing that the design parameters of nanomedicines greatly influence their activity. Some of his formulations loading anticancer agents have proceeded into human clinical studies, showing improved survival and reduced side effects.
Session II: Delivery
Supramolecular assemblies for enhanced mRNA delivery
Monday, 11:40 – 12:00 EST
Danilo Casimiro
Dr. Casimiro is Vice President and Head of Global Research and External Innovation at Sanofi Pasteur. He is responsible for vaccine research and search-and-evaluation function for external vaccine and vaccine technology opportunities. He has more than two decades of experience in research and development of vaccines and biologics in both pharmaceutical and non-profit sectors. Prior to joining Sanofi Pasteur on November 2017, he was the Chief Scientific Officer at Aeras, a non-profit organization dedicated to the development of new and more effective tuberculosis vaccines; he played a leading role in developing the long-term scientific strategy for the field and advancing promising candidates to the development stages. During his 19 years at the Merck Research Laboratories, Dr. Casimiro contributed to the licensure of Merck’s papillomavirus vaccines, and the development of novel vaccine candidates against several viral pathogens including herpesviruses, ebola, HIV-1, RSV, and dengue, and monoclonal antibodies against respiratory pathogens and oncology targets. As a prominent scientific leader, he has also served on numerous advisory committees to the Bill & Melinda Gates Foundation Global Health Program, International AIDS Vaccine Initiative, NIH Integrated Preclinical/Clinical AIDS Vaccine Development Program, U.S. Agency for International Development, HIV Vaccine Enterprise, and Human Vaccine Project. Dr. Casimiro received his B.S. and Ph.D. degrees in Chemistry from the University of the Philippines and the California Institute of Technology, respectively.
Session III: Prophylactic and Therapeutic Vaccines
Developing an mRNA Vaccine Platform Across Multiple Infectious Disease Targets
Monday, 14:40 – 14:55 EST
Frank DeRosa
Frank DeRosa serves as Chief Technology Officer at Translate Bio and joined the team in 2016. Prior to joining Translate Bio, Frank was Senior Director of Preclinical Research and Process Development for the mRNA therapeutics platform at Shire. He also worked at Enzon Pharmaceuticals developing polymer and lipid nanoparticle-based delivery systems for locked nucleic acid (LNA) antisense technology. Frank has previously held roles at Takasago International Corporation and the National Cancer Institute (NIH) focused on small molecule library synthesis and polymer-drug conjugate analogues, respectively. He is a co-inventor on over 50 issued patents and more than 75 additional pending patent applications. In addition, he is a co-author on eighteen scientific publications. Frank received his Ph.D. from the University of California, Santa Barbara.
Session III: Prophylactic and Therapeutic Vaccines
Developing an mRNA Vaccine Platform Across Multiple Infectious Disease Targets
Monday, 14:40 – 14:55 EST
Yusuf Erkul
Yusuf Erkul is the co-founder and CEO of Kernal Biologics, a Cambridge, MA-based biotech company developing messenger RNA 2.0 for cancer immunotherapy. As a member of MIT Startup Exchange and Y Combinator, Kernal received two awards from Amgen and a “Technology in Space” prize from CASIS/NASA and Boeing. Dr. Erkul co-invented Kernal’s proprietary precisely engineered stealth mRNA and onco-selective mRNA technologies. With his expertise in nucleic acid therapeutics, innate immunity and cancer biology, Dr. Erkul is currently leading Kernal’s drug discovery and development efforts in immuno-oncology. Before co-founding Kernal, he was a Director at Sentegen’s R&D division and prior to that he worked at Merck’s Oncology Department. His group at Merck provided exploratory pharmacology and biomarker support for various siRNA and small molecule therapeutic programs, including a PARP inhibitor (MK-4827) which later got FDA approval for ovarian cancer (niraparib, ZEJULA®). His group also established and validated state-of-the-art immunocompetent mouse models, which helped the company develop the largest immuno-oncology pipeline , which includes KEYTRUDA® (pembrolizumab). Dr. Erkul received Merck’s Award of Excellence, twice. He obtained his M.D. degree from Hacettepe University and his B.S. degree in Biology from MIT.
Yuta Fukushima
Ph.D., The University of Tokyo, School of Medicine (Neuroscience), September 2018.
Research theme: mRNA medicine for central nervous system diseases.
Rachel Green
Rachel Green began her scientific career majoring in chemistry as an undergraduate at the University of Michigan. Her doctoral work was performed at Harvard in the laboratory of Jack Szostak where she studied RNA enzymes and developed methodologies for evolving RNAs in vitro. She came to the JHU School of Medicine in 1998 following post-doctoral work in Harry Noller’s lab at UC Santa Cruz where she began her work on ribosomes. Her laboratory is interested in deciphering the molecular mechanisms that are at the heart of protein synthesis and its regulation across biology. Most recently, her work has focused on ribosome-mediated quality control systems that are triggered on difficult-to-translate mRNA sequences deriving from genetic or environmental insults. She has found that such translational distress leads not only to mRNA-specific QC events, but also to the activation of cell-wide signaling and transcriptional responses, mediated by factors that specifically bind to colliding ribosomes. Her laboratory uses both biochemical and genomic approaches to get at these questions in bacterial and eukaryotic systems.
Session I: mRNA Biology and Translation
Colliding ribosomes function as a gauge for activation of cellular stress responses
Monday, 08:40 – 09:05 EST
Charbel Haber
Current Responsibilities: Charbel Haber joined Moderna in April 2020 as a Senior Vice-President of Regulatory Affairs. In this role, Charbel is responsible of building a global regulatory organization, comprising, Regulatory Operations, Regulatory CMC, and Regulatory Strategy, to support Moderna’s pipeline including the development and registration of Moderna COVID-19 Vaccine.
Prior Experience: Charbel worked most recently at Biogen, where he was a VP in Global Safety and Regulatory Sciences leading the Global Regulatory Strategy, Medical Writing, and the Clinical Trial Application team. Charbel has an extensive and diverse global regulatory background having worked at EMD Serono, Novartis (both in the US and in EU), PTC Therapeutics, MedImmune and Merck. His experiences include both large and small molecules across a number of modalities and therapeutic areas spanning gene therapy, vaccines, biologics, Anti-Sense Oligonucleotides, orphan, neurological, respiratory, cardiovascular, inflammatory, and auto-immune diseases. Of particular note, Charbel led the regulatory development and registration of the Fluvirin-H1N1 vaccine at Novartis during the H1N1 Flu pandemic in 2009.
Education: Charbel holds a PhD in Chemical and Biomolecular Engineering from the Johns Hopkins University, a Masters’ degree in Public Health from Thomas Jefferson University, and an MBA from Thunderbird School of Global Management.
Session VIII: Regulatory Considerations
Chair
Tuesday, 14:45 – 16:15 EST
Nicholas Jackson
Dr. Nicholas Jackson joined CEPI in 2019, as the Head of Programs and Technology for R&D. CEPI’s mission is to accelerate the development of vaccines against emerging infectious diseases and enable access to these vaccines for people during outbreaks.
Recently prior to CEPI, Dr. Jackson was VP, Head of Research for Sanofi Pasteur, responsible to lead the two premier Research facilities in Cambridge MA and Lyon, France. Sanofi Pasteur is the largest company in the world devoted entirely to vaccines.
Dr. Jackson has spent over 20 years in the prophylactic & therapeutic vaccine industry in research and development, working on a variety of global vaccine programs (from early research through to Phase 3 & licensure) in disease areas including bacterial, viral and neurological targets. He has lived and worked in the UK, Belgium, the United States and France.
Dr. Jackson was trained in the United Kingdom and holds a Bachelor of Science degree from Oxford Brookes University, a master of science from the London School of Hygiene & Tropical Medicine, and a doctorate from the University of Warwick in the field of viral immunology. He has served on, and continues to provide expertise for, several advisory groups in the field of vaccines.
Session VII: Pandemic Responses/COVID-19
mRNA platform advances and COVID-19 candidate vaccines
Tuesday, 11:30 – 11:50 EST
Andreas Kuhn
Andreas Kuhn has worked in the field of RNA biochemistry and molecular biology for more than thirty years. His work on RNA-based immunotherapies began in 2007 in the academic group of Ugur Sahin at the University Clinic Mainz, and Andreas joined BioNTech RNA Pharmaceuticals GmbH shortly after its founding in 2008. In his current role as Senior Vice President RNA Biochemistry & Manufacturing the main focus is expanding proprietary technologies to increase the efficacy of RNA-based therapies and to optimize GMP-compatible manufacturing processes for RNA. He has co-authored numerous publications and patents ranging from basic research on RNA to its application as a diagnostic and therapeutic agent.
Session VIII: Regulatory Considerations
Round Table Discussion with Drs.Round Table Discussion with Drs.Baylor and Pfleiderer, and Regulatory Heads Charbel Haber from Moderna, John Talian from CureVac and Andreas Kuhn from BioNTech
Tuesday, 15:15 – 16:15 EST
Kyuri Lee
Kyuri Lee is an Assistant Professor of College of Pharmacy and Research Institute of Pharmaceutical Sciences at Gyeongsang National University. She received her Ph.D. from KAIST under the supervision of Prof. Tae Gwan Park in 2011 and did her postdoctoral training in Prof. Victor C. M. Yang’s lab. at University of Michigan. Before joining Gyeongsang National University, she was a research professor of College of Pharmacy at Ewha Womans University. Her research has been focused on the development of novel nanomaterials for mRNA therapeutics.
Session I: mRNA Biology and Translation
Development of RNA structures for cancer immunotherapy
Monday, 09:05 – 09:30 EST
David R. Liu
David R. Liu is Director of the Merkin Institute and Vice-Chair of the Faculty at the Broad Institute; Professor of Chemistry and Chemical Biology at Harvard University; and Howard Hughes Medical Institute Investigator. Liu graduated first in his class at Harvard in 1994. He performed research on sterol biosynthesis under Professor E. J. Corey’s guidance as an undergraduate. During his Ph.D. research with Professor Peter Schultz at U. C. Berkeley, Liu initiated the first general effort to expand the genetic code in living cells. He earned his Ph.D. in 1999 and became Assistant Professor at Harvard in the same year. Liu was promoted to Full Professor and became a Howard Hughes Medical Institute Investigator in 2005. Liu has published >190 papers and is the inventor of >70 issued U.S. patents. His research and teaching accomplishments have earned distinctions including the NIH Marshall Nirenberg Lecturer, Ronald Breslow Award for Biomimetic Chemistry, the American Chemical Society David Perlman Award, ACS Chemical Biology Award, the American Chemical Society Pure Chemistry Award, the Arthur Cope Young Scholar Award, and awards from the Sloan Foundation, Beckman Foundation, NSF CAREER Program, and Searle Scholars Program. In 2016 he was named one of the Top 20 Translational Researchers in the world by Nature Biotechnology, and in 2017 was named to the Nature’s 10 researchers in world. Professor Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. Prime editing, base editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. He is the scientific founder or co-founder of seven biotechnology and therapeutics companies, including Editas Medicine, Pairwise Plants, Exo Therapeutics, Beam Therapeutics, and Prime Medicine.
Session V: Gene and Base Editing of/with mRNA
Base Editing and Prime Editing: Chemistry on the Genome Editing Without Double-Strand Breaks
Tuesday, 08:25 – 08:50 EST
Nadia Luheshi
As an Associate Director in the Oncology R&D Unit at AstraZeneca, Nadia Luheshi leads research to discover and develop novel immuno-oncology therapies, including IL-12 mRNA (MEDI1191), which is now being evaluated in a phase I clinical trial. Her research group at AZ also has a particular focus on understanding the role of the myeloid cells in the tumour microenvironment in regulating anti-tumour immunity. Before she joined AstraZeneca 9 years ago, her PhD and post-doctoral research at the University of Manchester explored the innate immune response to tissue injury.
Katherine Mallory
Katherine Mallory received her Master’s in 2013 from Virginia Commonwealth University in Microbiology and Immunology. Her thesis research was focused on characterizing the cyclic di-GMP secondary messenging pathway of Borrelia burgdorferi (Lyme disease) and B. hermsii (relapsing fever). Since joining the team at the Walter Reed Army Institute of Research, her focus has shifted to another vector borne disease, Plasmodium falciparum. As part of the Malaria Biologics Branch, she engages in research to determine options for potent malaria prophylaxis.
Session III: Prophylactic and Therapeutic VaccinesImmunodominant Malaria Antigen, pfcsp Induces Functional, Protective Immune Responses in Mice using mRNA Platform
Monday, 14:25 – 14:40 EST
Pierre Meulien
Pierre Meulien is executive director of the Innovative Medicines Initiative (IMI), a €5 billion public-private partnership between the European Union and the European pharmaceutical industry. At IMI, he is responsible for the overall management of the program, which works to improve and accelerate the drug development process by facilitating collaboration between the key players involved in health research. Previously, Dr Meulien was president and CEO of Genome Canada, where he raised money and oversaw the launch of novel projects and networks in the field of genomics-based technologies. Prior to that, he was chief scientific officer for Genome British Columbia and was the founding CEO of the Dublin Molecular Medicine Center. Dr Meulien also worked with the French biotechnology company Transgene and with Aventis Pasteur (now Sanofi Pasteur). He has a Ph.D. in molecular biology from the University of Edinburgh and carried out a postdoctoral fellowship at the Institut Pasteur in Paris.
Session VII: Pandemic Responses/COVID-19
The Innovative Medicines Initiative: Responding to COVID-19 through a public-private partnership
Tuesday, 14:05 – 14:25 EST
Jacqueline Miller
Jacqueline Miller is a pediatrician who graduated from Northwestern University’s Feinberg School of Medicine, and trained at the Children’s Hospital of Philadelphia. For the past 20 years, she has worked in the field of Vaccine Development, contributing to the licensure of 5 vaccines targeting populations ranging from infants to older adults. She joined Moderna in 2020 as the Therapeutic Area Head for Infectious Diseases, and currently leads the medical teams developing Moderna’s vaccines and monoclonal antibodies directed against infectious pathogens. Prior to joining Moderna, she was most recently Head, Clinical R&D, US R&D Center at GlaxoSmithKline. She is pleased to take this opportunity to provide an update with respect to Moderna’s mRNA-1273 program.
Session VII: Pandemic Responses/COVID-19
The Moderna mRNA Vaccine Platform to Address SARS-CoV-2
Tuesday, 11:50 – 12:15 EST
Melissa J. Moore
Melissa J. Moore, PhD
Chief Scientific Officer, Research Platform, Moderna Therapeutics
In her role as Chief Scientific Officer of Moderna’s research platform, Dr. Melissa Moore is responsible for leading all mRNA biology and delivery science research at Moderna. She joined Moderna from the University of Massachusetts Medical School (UMMS), where she served as Professor of Biochemistry & Molecular Pharmacology, Eleanor Eustis Farrington Chair in Cancer Research and Investigator at the Howard Hughes Medical Institute (HHMI). Dr. Moore was also a founding Co-Director of the RNA Therapeutics Institute (RTI) at UMMS, and was instrumental in the creation of the Massachusetts Therapeutic and Entrepreneurship Realization initiative (MassTERi), a faculty-led program intended to facilitate the translation of UMMS discoveries into drugs, products, technologies and companies. She is a member of the National Academy of Sciences as well as American Academy of Arts & Sciences.
Dr. Moore’s research at UMMS and HHMI encompassed a broad array of topics related to the role of RNA and RNA-protein (RNP) complexes in gene expression and touched on many human diseases including cancer, neurodegeneration and preeclampsia. Prior to UMMS, she spent 13 years as a professor at Brandeis University, where she also received her Ph.D. in Biological Chemistry. Dr. Moore holds a B.S. in Chemistry and Biology from the College of William and Mary.
Upon joining Moderna, Dr. Moore has retained her academic affiliation with UMMS as a part-time faculty member.
Welcome and Logistics
Monday, 08:00 – 08:15 EST
Session I: mRNA Biology and Translation
Chair
Monday, 08:15 – 09:45 EST
Session V: Gene and Base Editing of/with mRNA
Chair
Tuesday, 08:00 – 09:15 EST
William L. Murphy
William L. Murphy, PhD, is the Harvey D. Spangler Professor of Biomedical Engineering, Professor of Orthopedics & Rehabilitation, and Director of the Forward BIO Institute at the University of Wisconsin. He has invented and developed multiple new classes of biomimetic materials inspired by the materials found in nature. Murphy’s research group has used new biomaterials to manufacture medical devices, human cells, and human tissues. These products are now being applied to emerging applications in tissue engineering. He has published more than 180 scientific manuscripts, 10 book chapters, and 2 books, filed over 50 patents, and co-founded multiple start-up companies. He has received awards that include the National Science Foundation Career Award, the Wisconsin Vilas Associate Award, the H.I. Romnes Faculty Fellowship, induction into the National Academy of Inventors and induction as a fellow in the American Institute for Medical and Biological Engineering.
Session II: Delivery
Biomaterials strategies to enhance and prolong the effects of therapeutic mRNA
Monday, 11:20 – 11:40 EST
Lidia Oostvogels
Lidia Oostvogels is a MD with over 25 years of global clinical development expertise, of which 17 in development of Prophylactic Vaccines. As Therapeutic Area Head Infectious Diseases, she leads the clinical development/medical team and overviews the strategy for the Area of Infectious Diseases in Curevac, which she joined in 2018. Prior to moving to CureVac, she was leading vaccine clinical teams in the Belgian Clinical R&D center at GSK, including the Shingrix clinical team. At the mRNA conference, she will present most recent data, including clinical data, from CureVacs Prophylactic Vaccine development programs.
Session VII: Pandemic Responses/COVID-19
Development of SARS-CoV-2 vaccine based on CureVac’s mRNA technology
Tuesday, 12:40 – 13:05 EST
Norbert Pardi
Dr. Norbert Pardi holds a Ph.D. in biochemistry and genetics. He has been working at the University of Pennsylvania since 2011. His research interest is the development of mRNA-based therapeutics with particular focus on new generation vaccines. He explored the development of a novel protein delivery platform using nucleoside-modified mRNA in lipid nanoparticles (LNPs) and used it to generate highly effective mRNA-LNP vaccines targeting various pathogens.
Session III: Prophylactic and Therapeutic Vaccines
Development of broadly protective influenza virus vaccines using nucleoside-modified mRNA
Monday, 13:10 – 13:30 EST
Michael Pfleiderer
Michael Pfleiderer is a biologist holding a Ph.D. in molecular virology. After his university career he firstly joined the molecular biology laboratories at former IMMUNO AG, Vienna, Austria (now BAXALTA), where he worked on various aspects related to the development and production of recombinant medicinal products including vaccines.
Since 1998, Dr. Pfleiderer had been the Head of the Human Viral Vaccines Section at the Paul-Ehrlich-Institut (PEI), German Federal Institute for Vaccines and Biomedicines.
He was responsible for all issues related to vaccine licensing and regulation, for batch testing and release of vaccines as well as inspection-related aspects. His team has had a leading function as Rapporteur, Co-Rapporteur or Peer Reviewer for a significant proportion of the Marketing Authorization Applications (MAAs) for vaccines submitted to the European Medicines Agency (EMA).
The team also acted on behalf of Germany as the Reference Member State (RMS) for the European Union (EU) in the Mutual Recognition (MRP) or Decentralised Procedure (DP) for a broad range of vaccines, most notably tri- and tetravalent seasonal influenza vaccines as well as pandemic influenza vaccines.
Dr. Pfleiderer has thus in-depth experience as an ex-regulator as he had guided through the decision making process on the benefit-risk ratio of vaccines and also decided on regulatory, legal and administrative issues related to vaccine applications. He has performed review of all kind of life cycle applications of vaccines related to all parts of the CTD, i.e. CMC, pre-clinical and clinical modules.
Moreover, numerous scientific advice procedures related to vaccines on the national level and at the level of EMA were performed and steered by him.
Session VIII: Regulatory Considerations
Regulatory Considerations Specific to mRNA-based SARS-CoV-2 Vaccines
Tuesday, 15:00 – 15:15 EST
Chantal Pichon
Dr. Chantal Pichon is Full Professor at the University of Orléans (France). She is performing her research activities at the Center for Molecular Biophysics of Scientific Research National Center (CNRS, Orléans). She is leading the department of Cell Biology, Pharmacological targets and Innovative therapies. Her research is mainly focused on nucleic acid therapeutics delivery with non-viral systems. She has a record of accomplishment of 160 peer-reviewed articles and 12 filled patents.
Shu-Bing Qian
Shu-Bing Qian is a biochemist at Cornell University where he is the James Jamison Professor of Molecular Nutrition. He received his PhD degree in biochemistry from Shanghai Jiaotong University School of Medicine in China and conducted postdoctoral studies at National Institute of Allergy and Infectious Disease and the University of North Carolina at Chapel Hill. At Cornell, Dr. Qian discovered stress-induced ribosome pausing and his laboratory was one of the first to profile initiating ribosomes on a transcriptome-wide scale from cells in culture to tissues in vivo. In addition to translational control of gene expression, Dr. Qian’s research interests include RNA modification, degradation, and quality control in both cytosol and nucleus. Dr. Qian was a recipient of 2009 NIH Director’s New Innovator Award, is a faculty scholar at Howard Hughes Medical Institute, and a recipient of 2020 NIH Director’s Pioneer Award.
Session I: mRNA Biology and Translation
Decoding mRNA translatability and stability from the 5’UTR
Monday, 09:30 – 09:45 EST
Syed Reza
Syed Reza serves as a drug delivery consultant to NOF Corporation since 2017. In this role, Syed has been responsible for managing industry partnerships for NOF’s COATSOME technology and advises on strategic partnerships and licensing. Previously, Syed was Head of US Business Development for Octoplus N.V. and managed industry alliances and partnerships for Octoplus’ protein nanoparticle technology platform. Prior to that, Syed served as Director of Business Development for Hoffman LaRoche’s peptide technologies group in Colorado. He is a graduate of the New Jersey Medical School with an MD and Ph.D. in Molecular Biology.
Session II: Delivery
COATSOME SS-Lipids as novel, biodegradable, ionizable Lipids for mRNA Therapeutics and Vaccines
Monday, 12:40 – 12:55 EST
Justin M. Richner
Dr. Justin M. Richner is currently an Assistant Professor in the Department of Microbiology & Immunology at the University of Illinois Chicago College of Medicine. He earned his graduate degree from the University of California Berkeley studying RNA turnover during a herpesvirus infection in the lab of Dr. Britt Glaunsinger. Dr. Richner joined the lab of Dr. Michael Diamond at Washington University in St. Louis for his postdoctoral research where he investigated viral immunology and vaccine development against flaviviruses. He started an independent lab in 2018 where he now leads a research team focused on viral immunology, immune senescence, and novel vaccination strategies.
Session III: Prophylactic and Therapeutic Vaccines
A mRNA-LNP vaccine against Dengue Virus elicits robust, serotype-1 specific immunity
Monday, 13:30 – 13:50 EST
Joshua Rosenthal
Joshua Rosenthal is a Senior Scientist at the Marine Biological Laboratory (MBL) in Woods Hole, Massachusetts. He received his Ph.D. in Biology from Stanford University and completed his postdoctoral training in biophysics and physiology at UCLA. Before coming to the Marine Biological Laboratory, he rose from Assistant to Full Professor at the University of Puerto Rico’s Medical Sciences Campus. Dr. Rosenthal’s research focuses on the process of RNA editing from a variety of angles. His group has shown that mRNA recoding is unusually active in cephalopods. They are interested in how cephalopod RNA editing can respond to environmental cues and how the underlying machinery for RNA editing in this taxon is able to drive such extensive recoding. The Rosenthal lab also pioneered systems to use RNA editing as a therapeutic in order to correct genetic mutations and engineer protein function. Dr. Rosenthal is a founder of Korro Bio, a biotech in Cambridge MA that is developing RNA editing platforms to treat human disease.
Session V: Gene and Base Editing of/with mRNA
Spatial and environmental tuning of genetic information within the cephalopod nervous system by RNA editing
Tuesday, 08:00 – 08:25 EST
Gaurav Sahay
Gaurav Sahay is an Assistant Professor in the Department of Pharmaceutical Sciences, College of Pharmacy at Oregon State University. Dr. Sahay completed his postdoctoral research with Prof. Robert Langer and Prof. Daniel Anderson at the Koch Institute for Integrative Cancer Research at MIT and received his Ph.D. from the University of Nebraska Medical Center under the mentorship of Prof. Alexander Kabanov. Sahay Lab is developing novel nanotechnology-based platforms for effective delivery of messenger RNA therapeutics for treatment of cystic fibrosis and retinal degeneration. He has done pioneering work to dissect the intracellular transport essential for nucleic acid delivery to the cytosol and developed methods to overcome endosomal barriers. Dr. Sahay has more than 43 peer-reviewed publications in top tier journals including Nature, Nature Communication, Nature Biotechnology, Nature Nanotechnology, Journal of Controlled Release, Nano Letters etc. He is the winner of a 2013 American Association of Pharmaceutical Scientists (AAPS) Postdoctoral Fellow Award, the 2015 Controlled Release Society (CRS) T. Nagai Award, a 2016 American Association of Colleges of Pharmacy (AACP) New Investigator Award, and a 2019 Oregon Health & Sciences University (OHSU) Distinguished Faculty Senate Award for Collaboration. He serves as the Principal Investigator on awards funded through the National Institutes of Health, Cystic Fibrosis Foundation and industry. He is a consultant to several biotech and venture capital firms. He was the Chair of the 2018 NanoMedicine and Drug Delivery Symposium and currently Chairs the CRS’s Gene Delivery and Editing Focus Group.
Session II: Delivery
Boosting intracellular delivery of messenger RNA
Monday, 12:00 – 12:20 EST
Ugur Sahin
Prof. Ugur Sahin, MD, co-founded BioNTech in 2008 and has served as the Chief Executive Officer of BioNTech since that time. Prof. Sahin also served as the head of the Scientific Advisory Board and Chief Medical Officer of Ganymed Pharmaceuticals AG from 2008 until the company was acquired by Astellas Pharma Inc., or Astellas, in 2016. In 2010, Prof. Sahin co-founded TRON, and served as a Managing Director from 2010 until 2019. Prof. Sahin has also been a professor (W3) at the Mainz University Medical Center since 2014. Prof. Sahin co-founded the Ci3, the German Cluster Initiative of Individualized ImmunIntervention (Ci3), a non-profit organization. Prof. Sahin earned an MD in 1990 from the University of Cologne.
He has made groundbreaking scientific and technological contributions in the field of personalized cancer immunotherapy and is a co-inventor on more than 100 patents. His daily work is motivated by his experience as a researcher and cancer physician aiming to exploit scientific insights and drive technological progress to develop commercially viable products that could help individual patients, an attitude and culture that has become the DNA of BioNTech.
Session VII: Pandemic Responses/COVID-19
BioNTech’s SARS-CoV-2 mRNA vaccines
Tuesday, 12:15 – 12:40 EST
Robin Shattock
Robin Shattock is a Professor of Mucosal Infection and Immunity at Imperial College, London. The main focus of his research is the investigation of the mechanisms of mucosal infection and development of novel preventative strategies. His group has a strong interest in mucosal immune responses to infection and vaccination with active programs on COVID-19, HIV, Ebola, Lassa, Marburg and Chlamydia. Prof Shattock has secured funding from the MRC-UK, EPSRC, CEPI and European Commission. He is the scientific director of the European AIDS Vaccine Initiative (EAVI2020) that coordinates a program of EU funded vaccine research across 22 institutions. He directs the Future Vaccines Manufacturing Research Hub and Imperial College. His research team is leading a range of clinical trials of candidate vaccines. Prof. Shattock Chairs the Research and Advisory Steering Committee of the International Partnership for Microbicides. He is the Founder of VacEquity Global Health and VaxEquity Ltd. His is an elected fellow of the Academy of Medical Sciences.
Session VII: Pandemic Responses/COVID-19
Development of a self-amplifying RNA SARS-CoV-2 vaccine candidate
Tuesday, 13:15 – 13:40 EST
Daniel J. Siegwart
Dr. Daniel J. Siegwart is an Associate Professor in the Department of Biochemistry and Simmons Comprehensive Cancer Center at UT Southwestern Medical Center. 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). The central goal of the Siegwart Lab is to use materials chemistry to solve challenges in disease therapy and diagnosis. An array of coding and non-coding RNAs can now be used as therapeutics (siRNA, miRNA, tRNA, mRNA, CRISPR RNAs) because they are able to manipulate and edit expression of the essential genes that drive disease development and progression. Although great advances have been made in the delivery of short RNAs, the ideal chemical and formulation composition is poorly understood for longer RNA cargo. The Siegwart Lab aims to discover and define the critical physical and chemical properties of synthetic carriers required for therapeutic delivery of small (e.g. ~22 base pair miRNA) to large (e.g. ~5,000 nucleotide mRNA) RNAs. They have designed synthetic carriers for CRISPR/Cas and applied these technologies for correction of genetic diseases. Their research is grounded in chemical design and takes advantage of the unique opportunities for collaborative research at UT Southwestern.
Session II: Delivery
Chair
Monday, 11:00 – 12:55 EST
John Talian
John Talian, is Senior Vice President, Regulatory Affairs at Curevac. He is a senior regulatory affairs leader with over 25 years’ experience in the development, registration and life cycle management of compounds in the global marketplace. He has interacted extensively with FDA, EMA, and other health authorities worldwide.
Before joining Curevac, he led the Regulatory Affairs group at Hurley Consulting, was Vice President, US Head of Regulatory Affairs, Global Regulatory Affairs at Bayer Healthcare Pharmaceuticals where he led a staff of 80 professionals with both local and global responsibilities for development projects and marketed products, as well as compliance activities. He managed critical US regulatory activities for the Bayer portfolio of products. He previously worked for Hoffmann-LaRoche Pharmaceuticals, Inc., in various regulatory positions in the US and Swiss Headquarters. Dr. Talian holds both a bachelor’s degree in cell and molecular biology and a doctoral degree in cell biology.
Session VIII: Regulatory Considerations
Round Table Discussion with Drs.Round Table Discussion with Drs.Baylor and Pfleiderer, and Regulatory Heads Charbel Haber from Moderna, John Talian from CureVac and Andreas Kuhn from BioNTech
Tuesday, 15:15 – 16:15 EST
Lior Zangi
Lior Zangi, PhD, is an assistant professor at the Icahn School of Medicine at Mount Sinai, New York. He completed his education and training at the Weizmann Institute of Science and at Harvard University. He has established mRNA based approach for transiently and specifically changes gene expression of heart cells post injury. Currently, Dr. Zangi’s laboratory investigates cellular and organ level regenerative mechanisms that can contribute to new therapeutic approaches to repair, protect and regenerate the heart following myocardial infarction.
Xiaowei Zhuang
Xiaowei Zhuang is the David B. Arnold Professor of Science at Harvard University and an investigator of Howard Hughes Medical Institute. She pioneered the development of single-molecule, super-resolution, and genomic-scale imaging methods, and has applied these methods to investigate an array of biological questions on molecular structures in cells, chromatin organization in the nucleus, regulation of gene expression, and cell organization in tissues.
Zhuang received her Ph.D. in physics under the supervision of Prof. Y. R. Shen from University of California at Berkeley, and her postdoctoral training in biophysics from Prof. Steven Chu at Stanford University. She joined the faculty of Harvard University in 2001 and became a Howard Hughes Medical Institute investigator in 2005. She is a co-founder of Vizgen, Inc. Zhuang is a member of the National Academy of Sciences and the American Academy of Arts and Sciences, a member of the American Philosophical Society, and a foreign associate of the Chinese Academy of Sciences and the European Molecular Biology Organization. She received honorary doctorate degrees from the Stockholm University and the Delft University of Technology. She has received a number of awards, including Vilcek Prize in Biomedical Science, Breakthrough Prize in Life Sciences, Pearl Meister Greengard Prize, National Academy of Sciences Award for Scientific Discovery, Heineken Prize for Biochemistry and Biophysics, Raymond and Beverly Sackler International Prize in Biophysics, Max Delbruck Prize in Biological Physics, MacArthur Fellowship, etc.
Session I: mRNA Biology and Translation
Single-cell transcriptome imaging and cell atlas of complex tissues
Monday, 08:15 – 08:40 EST
Speakers - Company Talks
Company talk Translate Bio: John Androsavich
John Androsavich, PhD, is the Senior Director of Pulmonary & Liver Biology at Translate Bio. John previously held research positions at Regulus Therapeutics and RaNA Therapeutics, where he discovered and developed candidate drug molecules targeting non-coding RNAs. He has focused his career on the full spectrum of nucleic acid therapeutics, working on both oligo and mRNA drug discovery, over the last decade after receiving his PhD in Chemical Biology from the University of Michigan. At Translate Bio, he currently leads the biology team in efforts focused on rare genetic diseases; and is the program lead for Translate Bio’s next-generation CFTR program.
Session II: Delivery
Advancing pulmonary delivery of mRNA therapeutics for the treatment of genetic diseases
Monday, 12:20 – 12:40 EST
Company talk Acuitas Therapeutics: Thomas D. Madden
Dr. Thomas M. Madden obtained his BSc. and Ph.D. in Biochemistry from the University of London, U.K. He has held several senior academic and industry positions including Assistant Professor in Pharmacology at the University of British Columbia and Senior Director, Technology Development and Licensing at Tekmira Pharmaceuticals (formerly INEX Pharmaceuticals). At Tekmira Pharmaceuticals Dr. Madden was responsible for product development of several liposomal anticancer agents including Marqibo™ (liposomal vincristine), Alocrest™ (liposomal vinorelbine) and Brakiva™ (liposomal vinorelbine). All of these products were subsequently licensed to Talon Therapeutics. Dr. Madden founded Acuitas Therapeutics in February 2009 and through his role as President and CEO and has guided the company to a leadership position in the application of lipid nanoparticle (LNP) technology for delivery of nucleic acid therapeutics. This includes the development of the cationic lipid MC3 and the LNP carrier used by Alnylam Pharmaceuticals for Onpattro™. Onpattro™ was approved in 2018 in the US and Europe for the treatment of transthyretin amyloidosis. Dr. Madden has over 60 publications in peer-reviewed journals relating to LNP technology and the development of pharmaceutical products, including recent publications in Nature.
Session IV: Manufacturing, Analytics and Commercialization
Characterizing the therapeutic application of mRNA-lipid nanoparticles
Monday, 15:50 – 16:10
Company talk TriLink BioTechnologies: Anton McCaffrey
Dr. Anton McCaffrey is the Senior Director of Emerging Science and Innovation at TriLink BioTechnologies and an adjunct Assistant Professor of Internal Medicine at the University of Iowa School of Medicine. Dr. McCaffrey has over 30 years of experience designing nucleic acid-based therapeutics for antisense, RNA interference, aptamer and genome editing applications. Dr. McCaffrey established the mRNA contract manufacturing program at TriLink BioTechnologies and led a research group that developed CleanCap® co-transcriptional capping reagents. Dr. McCaffrey’s research interests include the role of the innate immune system in recognition of nucleic acid drugs.
Session IV: Manufacturing, Analytics and Commercialization
Strategic CDMO partnering for accelerated mRNA vaccine development: A COVID-19 case study
Monday, 15:30 – 15:50 EST
Company talk New England Biolabs: John Pelletier
John Pelletier is the Associate Director of Regulated Market Development at New England Biolabs. He received a BA in biology from Colby College and a MS in biochemistry from the University of Massachusetts. Following that, he started his 32-year career at NEB in enzyme cloning, and, as a purification scientist and product manager, co-developed several key products. He is the co-author of a number of publications that focus on the relationship of enzyme structure and function. John initiated and developed NEB’s custom product business which he led for over 20 years. In that time, John helped drive forward the establishment of NEB’s formal quality systems and its ISO9001 and ISO13485 certifications, and, more recently, NEB’s GMP-grade manufacturing capabilities. Doing what he’s most passionate about, John now develops and manages key customer relationships, addressing their needs for GMP-grade reagents used in the manufacture of novel therapeutics, particularly nucleic acid therapies.
Session IV: Manufacturing, Analytics and Commercialization
Meeting the quality and scale requirements for IVT enzymes in the production of therapeutic mRNAs
Monday, 15:10 – 15:30 EST
Company talk Arcturus Therapeutics: Sean Sullivan
Sean Sullivan is the Executive Director, Process Development at Arcturus. He has more than 20 years of biotech experience developing nucleic acid based therapeutics, including antisense DNA, ribozymes and plasmid DNA.
Most recently, Sean was the Senior Executive Director of Pharmaceutical and Process Development at Vical, Inc. While there, he led a multidisciplinary team within drug substance, drug product and process development. Prior to Vical, Sean was a tenured Associate Professor at the University of Florida in the Department of Pharmaceutics, where he developed a cancer research program focused on inhibiting tumor angiogenesis for the treatment of brain cancer. Prior to that, Sean held various positions at Valentis, Cell Genesys, Ribozyme Pharmaceuticals, Vestar and was a post doctoral fellow at the California Institute of Technology. In prior roles, Sean held the position of Principal Investigator for NIH funded projects developing a genital herpes simplex virus vaccine and enzymatically synthesized linear DNA expression cassettes.
Sean holds a Ph.D. and Master of Science degree in Biochemistry from the University of Tennessee, and Bachelors of Science degree in Chemistry from Marysville College.
Session VII: Pandemic Responses/COVID-19
LUNAR®-COV19: The Arcturus Therapeutics SARS-CoV-2 mRNA Vaccine Candidate
Tuesday, 13:40 – 14:05 EST
Company talk Thermo Fisher Scientific: Tadas Trunca
As a Senior Manager of Product Management at Thermo Fisher Scientific, Tadas Trunca leads Nucleic Acid Therapeutics TheraPureTM raw materials product portfolio from development to commercial scale supply stages. His global Product Management group in particular focuses on enabling mRNA platform developers and commercializers by supplying starting and ancillary Nucleic Acid synthesis raw materials (enzymes, nucleotides, modified nucleotides products, Cap analogs). For the past 6 years at Thermo Fisher Scientific, Tadas played key role in enabling and partnering with multiple mRNA medicines developers by positioning Thermo Fisher Scientific as key strategic supplier and partner. He obtained his BSc in Biology from Vilnius University as well as Master of Business Administration from ISM University of Management and Economics.
Session IV: Manufacturing, Analytics and Commercialization
Enabling mRNA Platform Commercialization and Setting Up for Future Success
Monday, 16:10 – 16:30 EST
Company talk eTheRNA Immunotherapies NV: Wim Tiest
Wim Tiest, Director Infectious Disease Programme at eTheRNA holds an MSc in Biotechnological Engineering and Artificial Intelligence. After a long professional career in immunology, oncology and vaccine R&D. he is now increasingly involved in projects that bring together life sciences and smart data. His current positions include roles for eTheRNA immunotherapies, Innoser Laboratories and the Board of Flanders Bio.
Session III: Prophylactic and Therapeutic Vaccines
Intranasal mRNA Vaccines: COVID and far beyond
Monday, 13:50 – 14:10 EST
