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Nadim Akhtar, PhD, Senior Principal Scientist, New Modalities, AstraZeneca
Nadim Akhtar, PhD, is a Senior Principal Scientist in the New Modalities and Parenteral Development Department in Pharmaceutical Technology and Development function at AstraZeneca. Nadim has been working for AstraZeneca since 2007. During this time Nadim has worked on both early- and late-stage portfolio supporting development of small molecules and new modalities i.e. oligonucleotides, peptides, polymeric nanoparticles, dendrimers, and mRNA products. Nadim is currently responsible for developing characterisation and control strategies for New Modalities.
Daniel Anderson, PhD, Professor, Chemical Engineering, Massachusetts Institute of Technology
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. 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 500 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 Therapeutics, VasoRx, and Souffle Therapeutics.
Oxana Beskrovnaya, PhD, CSO, Dyne Therapeutics
An accomplished R&D leader with a track record of discovering and developing first-in-class therapeutics for rare genetic diseases, Oxana has worked across multiple disease indications in both large biopharmaceutical and small biotech companies. Oxana is currently Chief Scientific Officer at Dyne Therapeutics where she leads preclinical research and development focused on receptor-mediated delivery of oligonucleotides for the treatment of muscle genetic diseases. Prior to joining Dyne, Oxana served as Head of Musculoskeletal and Renal Research in Sanofi. In that role, she was responsible for conducting research and advancing a pipeline of drug candidates using multiple therapeutic modalities including nucleic acids, proteins, and small molecules. Before that Oxana served in various capacities of increasing responsibility at Genzyme where she managed research in genetics, disease mechanisms, target discovery/validation, and translational biomarkers in rare renal, metabolic, skeletal, and neuromuscular diseases. Oxana authored numerous patents, invited reviews, editorials, book chapters, and original research articles in major scientific journals including Nature, Nature Medicine, Cell, and Molecular Cell. Oxana received her doctorate in genetics from Moscow Genetics Institute followed by postdoctoral fellowship training in neuromuscular diseases with HHMI at the University of Iowa.
Tracey Burr, PhD, Director, CMC Regulatory Affairs, Ionis Pharmaceuticals
Tracey Burr is Executive Director of CMC Regulatory Affairs at Ionis Pharmaceuticals 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 PhD in Materials Science and Engineering from MIT, M.Sc. in Chemistry from Caltech, and BA in Chemistry from Wellesley College.
Marvin Caruthers, PhD, Distinguished Professor, University of Colorado
Professor Marvin H. Caruthers research includes nucleic acids chemistry and biochemistry. Approximately 35 years ago, the methodologies that are used today for chemically synthesizing DNA and RNA were developed in his laboratory. His laboratory has also pioneered the synthesis of many new nucleic acid analogs that have found applications in the nucleic acid diagnostic and therapeutic areas. He is the recipient of the National Academy of Sciences Award for Chemistry in Service to Society, the Prelog Medal, the Economists Award in Biotechnology, and the US National Medal of Science. He is also the recipient of The National Academy of Science Award in the Chemical Sciences, The American Chemical Society Award for Creative Invention, The Frantisek Sorm Medal-The Academy of Sciences of the Czech Republic and The Life Time Achievement Award of the Oligonucleotide Therapeutic Society. Dr. Caruthers is an elected member of The US National Academy of Sciences, The American Academy of Arts & Sciences, The National Inventors Hall of Fame, The National Academy of Inventors, and a Corresponding Member of the German Academy of Science Gottingen. One of the co-founders of Amgen and Applied Biosystems, Dr. Caruthers remains active in the Biotechnology arena - most recently as a co-founder of Array BioPharma and miRagen Therapeutics.
David Corey, PhD, Professor, Department of Pharmacology, UT Southwestern
Dr. Corey graduated with a BA from Harvard in 1985 and a PhD from UC Berkeley under the direction of Dr. Peter Schultz in 1990. He has had his own laboratory at UT Southwestern since 1992 and is currently the Rusty Kelley Professor of Biomedical Science in the Department of Pharmacology.
Robert Dream, PhD, Managing Director, HDR Co. LLC
Robert Dream is an industry leader with 30+ years of experience, including 20 years of executive leadership in the pharmaceutical and biotechnology industries. He has led projects, improved processes, helped move drug substance and drug products from R&D, clinical, regulatory licensing approvals through operational excellence strategies and leading edge technologies. He is business minded person and has an innovative knowledge and knowhow of manufacturing, logistics, supply chain, risk mitigation and management. He is experienced in therapeutic biotechnology and biological drug substance and drug products manufacturing, with extensive hands-on, senior leadership and executive experience at world-leading organizations. He wrote numerous articles for many journals and publications and lead and presented at many institutions, organizations, and conferences. He is a registered professional engineer and an active member of the ISPE and PDA. He is a member of the editorial advisory Board; Pharmaceutical Processing, Pharmaceutical Manufacturing, Pharmaceutical Technology, Pharmaceutical Engineering, and the INTERPHEX Advisory Council. He is a member and Process Chair of the PDA "Aging Facilities Modernization" Team. He is the Chair of the ISPE GUIDE: "Biopharmaceutical Process Development and Manufacturing"; published 2013, ISPE "Sustainability Handbook"; Published 2015 and numerous other relevant publication to list few.
Thomas Frischmuth, PhD, CEO, rnatics
As a serial entrepreneur, Thomas Frischmuth contributes more than 30 years of experience in the life science and medical device field as researcher, advisor, consultant, and in management positions. He brings his extensive expertise in the development and chemical synthesis (small- to large-scale) of oligonucleotides and carbohydrate ligands in the context of drug development to rnatics. To date, Thomas Frischmuth developed and evaluated 6 drugs preclinically and brought 4 of them into clinical evaluation studies Phase I and II. One drug is marketed for cancer treatment. In 2003, Thomas Frischmuth founded his own private venture fund company (Gradus Venture GmbH).
Paloma H. Giangrande, PhD, Vice President, Platform and Discovery Sciences Biology, Wave Life Sciences
Dr. Giangrande is the Vice President of Biology at Wave Life Sciences in Cambridge, MA and an Adjunct Professor of Internal Medicine at the University of Iowa. She obtained her Bachelor of Science degree in Biochemistry at Wheaton College, Norton, MA in 1994 and her PhD in Pharmacology and Cancer Biology at Duke University, Durham, NC in 1999. Her career has been dedicated to the investigation and development of RNA-based therapeutic approaches (including RNA aptamers, siRNAs, mRNA, RNA editing) for cancer, cardiovascular disease, rare diseases, and critical illness. As a consequence, Dr. Giangrande developed a deep understanding of these technologies, diseases, and associated disease mechanisms. She is an internationally recognized expert in oligonucleotide therapeutics and delivery. Towards this end, she was the first to demonstrate that RNA ligands (aptamers) can be used to deliver therapeutic siRNAs to target cells. Her 70+ publications and 9+ patents are a testament to this body of work and her commitment to the development of novel RNA-based therapies for many diseases.
Nagy Habib, ChM, FRCS, Head of R&D and CMO, MiNA Therapeutics Ltd.
Professor Habib is a leading academic hepatobiliary surgeon, Head of Surgery at the Hammersmith Hospital Campus, Imperial College London and Founder, Head of R&D and Chief Medical Officer for MiNA Therapeutics Ltd. His research programme evolved from research in oncogenes to tumour suppressor genes, epigenetic modification, gene therapy, stem cell therapy, RNA, and saRNA and RNA aptamers. Professor Habib is the first to trial a first-in-human and a first-in-class RNA oligonucleotide (MTL-CEBPA) in patients with liver cancer. Among his accomplishments, Professor Habib co-founded several biotech companies, including: EMcision (acquired by Boston Scientific), OmniCyte, MiNA Therapeutics, Apterna Therapeutics, Medeva (acquired by Celltech), and Bioenvision (acquired by Genzyme).
Xiao Shelley Hu, PhD, Vice President, Head of DMPK and Clinical Pharmacology, Wave Life Sciences
Dr. Xiao Shelley Hu, PhD, is a Vice President at Wave Life Sciences in Boston, currently the head of the DMPK and Clinical Pharmacology department. Her group is responsible for ADME, PK/PD, and Pharmacometric studies in preclinical and clinical stages in neuromuscular, CNS, and hepatic diseases. Prior to joining Wave Life Sciences, she was a Director at Akebia Therapeutics in Boston, leading the Clinical Pharmacology, Pharmacometrics and Bioanalytical function. Shelley joined Akebia from Biogen, where her responsibility evolved from pre-clinical DMPK to Clinical Pharmacology, and later to Pharmacometrics for both small molecules and large molecules from discovery to post-market in Neurology, Rheumatology, Oncology, and Immunology. Shelley received her PhD in Pharmaceutical Sciences from the Ohio State University, MS in Pharmaceutical Sciences from Ohio State University, MS in Environmental Chemistry from Chinese Academy of Sciences, and BS in Pharmaceutical Sciences from Peking University Health Science Center. She has fourteen publications in peer-reviewed journals and one book chapter and serves as reviewers for various journals.
Radhakrishnan Iyer, PhD, CSO, RIGImmunce, Inc.
Radhakrishnan Iyer, (Kris) is the Chief Scientific Officer of RIGImmune, Inc., and has more than 30 years' experience in the Biopharmaceutical industry in drug discovery and development. Kris is considered a leading innovator in the fields of nucleic acid chemistry, and pharmaceutical sciences with over 100 publications and 250 issued as well as filed U.S. and international patents. Prior to RIGImmune, Kris was the co-founder and CSO of Spring Bank Pharmaceuticals, Inc., co-founder, and CSO of Origenix Technologies, Inc., and was the associate director of Antisense Discovery group at Hybridon, Inc. He has led multidisciplinary teams in the advancement of several small molecule and oligonucleotide drug candidates from discovery into clinical development in diverse therapeutic areas including antivirals, immuno-oncology, and inflammation.
Rachel Johns, Senior Director, Formulations, Avidity Biosciences LLC
Rachel Johns, PhD, 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 PhD 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 manages the Analytical Development, Formulation, and Quality Control efforts for Avidity's novel therapeutics.
Mark Kay, MD, PhD, Dennis Farrey Family Professor of Pediatrics and Genetics, Department of Pediatrics and Genetics, Stanford University
Mark A. Kay MD, PhD is the Dennis Farrey Family Professor of Pediatrics and Genetics at Stanford University and is Head of the Division of Human Gene Therapy in Pediatrics. Dr. Kay has made seminal contributions in the fields of Gene and Genome engineering based therapeutics, RNAi and non-coding RNA biology. His work spans basic research through clinical trials that includes the first systemic delivery of AAV into humans. Dr. Kay was a founding board member of the American Society for Cell and Gene Therapy and served as the President of the Society in 2006. He received the Outstanding Investigator Award in 2013. Dr. Kay served on the Board of Directors of the Oligonucleotide Therapeutics Society for 10 years. He has published >300 peer reviewed papers, and has served and continues to serves as an editor of several journals. He is an inventor on many patents including those related to key gene therapy vectors and the use of RNAi in whole mammals. He has co-founded 4 companies and serves on many biotech/pharmaceutical advisory boards. In 2020, he was elected to the National Academy of Inventors.
Kimmy Kolkey, Associate Director, Analytical Development and Quality Control, Ionis Pharmaceuticals
Kimmy Kolkey is currently an Associate Director of Analytical Development & Quality Control at Ionis Pharmaceuticals in Carlsbad, California. During 15+ years at Ionis, Kimmy's focus has been on the analysis of oligonucleotides in preclinical through commercial phases. Her work includes the management of drug substance and drug product stability studies, both in-house and at partner contract labs in the USA and Europe, authoring CMC sections of regulatory filings, and analytical method validations for late-stage compounds. Kimmy collaborates on multidisciplinary project teams facilitating the advancement of compounds at all stages of development. She also participates in routine regulatory audits, including Pre-Approval Inspections for three Ionis commercial products. Kimmy holds a San Diego State University BS in Biology and an MFS in Forensic Chemistry.
Sarah Lamore, PhD, Director, Toxicology, Pre-Clinical Development Sciences, WAVE Life Sciences
Sarah is currently Director of Toxicology and head of Investigative Toxicology at Wave Life Sciences. Prior to joining Wave, Sarah was a Toxicologist at Biogen where she worked on several modalities including small molecules and antisense oligonucleotides. She did her postdoctoral training at AstraZeneca and then joined the company as a Discovery Safety Scientist. She holds a PhD in Pharmacology and Toxicology from University of Arizona and is a Diplomate of the American Board of Toxicology.
Ekkehard Leberer, PhD, Senior Life Sciences Consultant, ELBIOCON; Advisor, Neuway Pharma
Dr. Leberer received his Ph.D. in Biology at the University of Konstanz, Germany (1986), and conducted post-doctoral training in molecular biology at the Banting and Best Institute of the University of Toronto, Canada. He then obtained the Habilitation for Professor of Biochemistry at the University of Konstanz, Germany (1992). From 1989-1998, he served as Senior Research Officer in genetics and genomics at the Biotechnology Research Institute, National Research Council of Canada, Montreal. He was also Adjunct Professor at McGill University, Montreal. Since joining the pharmaceutical industry in 1998, Dr. Leberer carried out various managing roles in Hoechst Marion Roussel, Aventis and Sanofi, including responsibilities in functional genomics, biological sciences, alliance management and external innovation for oligonucleotide-based therapeutics. In addition, from 2012-2018, he has been the Scientific Managing Director of the Innovative Medicines Initiative COMPACT Consortium on the delivery of biopharmaceuticals across biological barriers and cellular membranes, Brussels. Since March 2021, Dr. Leberer is Senior Life Sciences Consultant at ELBIOCON (www.elbiocon.com). He serves as a member in Scientific Advisory Boards of several biotechnology companies, and he is the Head of the Supervisory Board of BioM, Munich. His research has focused on the molecular mechanisms of signal transduction and the role of signalling molecules in human diseases. He is the co-discoverer of the p21 activated protein kinase (PAK) family of cell signalling proteins and of novel virulence-inducing genes in pathogenic fungi. He is co-author of more than 60 publications in prestigious peer-reviewed journals including Nature and Science.
Dong-Ki Lee, PhD, CEO, OliX Pharmaceuticals
Prof. Dong-ki Lee received B. S. in Chemistry from Korea Advanced Institute of Science and Technology (KAIST) in 1993, and Ph. D. in Biochemistry from Cornell University in 1999. After post-doctoral training in Pohang University of Science and Technology (POSTECH), Toolgen Inc., and KAIST, he joined POSTECH as an assistant professor in 2004. In 2008, He moved to Sungkyunkwan University and is a full professor of Chemistry since 2012. In 2008, Prof. Lee was selected as the principal investigator of the Global Research Laboratory program, funded by Korean government, to develop novel RNAi medicine. He is currently serving as the Asian editor of "Nucleic Acid Therapeutics" and a editorial board member of "Molecular Therapy: Nucleic Acids". His work on novel siRNA structures, nucleic acid aptamers, and eukaryotic gene regulation has been published as over 80 papers including prestigious journals such as Nature, Cell, PNAS, and Molecular Therapy. In 2010, he founded OliX Pharmaceuticals, a publicly traded RNAi therapeutics company, and serves as Chief Executive Officer.
Marc Lemaitre, PhD, Oligonucleotide Therapeutics CMC/Strategy Consultant, ML_Consult LLC
Marc Lemaitre holds a degree in Organic Chemistry and a PhD in Biochemistry from the University of Liege, Belgium. Since 1985, Marc's professional interests have been the study of the Nucleic Acids and therapeutics as well as diagnostic applications. Based in the USA since 2006 he worked for Glen Research, as CEO of Girindus America, a CMO with a focus on the cGMP manufacture of oligonucleotides for therapeutic applications. Marc is now working as consultant since 2013, working mostly on CMC with start-up and Pharma companies developing therapeutic oligonucleotides and peptides.
Arthur Levin, PhD, CSO, Avidity Biosciences
Arthur Levin is a key opinion leader in the RNA therapeutics field. He serves as the CSO at Avidity Biosciences. Previously, he held senior level positions at mRiragen, Santaris Pharma, and Ionis Pharmaceuticals. He played key roles in developing numerous of oligonucleotides including the first approved antisense drugs and the first microRNA-targeted therapeutic in clinical trials. He has three decades of experience in drug development from discovery through drug registration. Dr. Levin has published over 60 scientific articles and several of the most cited reviews in the field. He is on the scientific advisory boards of multiple institutions. Art received a doctorate in toxicology from the University of Rochester.
Li Li, PhD, Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical School
Li Li obtained his B.Sc. degree in Biology in 2007 from Fudan University in Shanghai, China. He moved to the University of Illinois at Urbana-Champaign and received a PhD in 2012. He then moved to Professor Jack Szostak's lab at Massachusetts General Hospital for postdoctoral studies, where he developed an efficient chemical method to copy RNA sequences. In 2020, Li started his independent group at the RNA Therapeutics Institute at UMass Medical School.
James Longden, PhD, Head, Discovery Biology, e therapeutics plc
James Longden is Head of Discovery Biology at e-therapeutics, a UK company integrating computational power and biological data to discover life-transforming RNAi medicines. At e-therapeutics, James and his team apply computational models to build protein-protein interaction networks driving disease and determine how these networks can best be perturbed in order to identify novel therapeutic strategies for GalNAc-siRNA drug discovery. Prior to joining e-therapeutics James spent over 20 years working in early drug discovery in both academia and industry, firstly in the pharmaceutical industry, at AstraZeneca and at Cenix BioScience, a pioneer in the field of RNAi, before moving to academia. His research at the University of Copenhagen was focused on 'big data' biology and the use of machine learning to predict network changes driving cancer cell metastasis and drug resistance. From there, he moved to the University of Edinburgh applying the same network methodologies to motor neuron disease and multiple sclerosis research. He has a PhD in biomedical science from the University of Nottingham.
Robert Mabry, PhD, CSO, Orna Therapeutics
Robert Mabry is currently the CSO at Orna Therapeutics. Previously, he served as Global Head of Biologics at Takeda Pharmaceuticals where he led the discovery and optimization of biologic/cell therapy candidates across four therapeutic areas and provided strategic support in overseeing their scientific operations. Prior to Takeda, Dr. Mabry served as Vice President, Protein Sciences for Cogen Therapeutics, now Repertoire Immune Medicines. During his time as head of department, he was responsible for protein engineering, antibody technology, protein production and analytics. Dr. Mabry has also held positions at Jounce Therapeutics, Adimab, ZymoGenetics, and ImClone Systems. He received his Ph.D. in Biochemistry from the University of Texas at Austin and a B.A. in Biology and Biochemistry from Baylor University.
Mano Manoharan, PhD, Distinguished Scientist & Senior Vice President, Innovation Chemistry, Alnylam Pharmaceuticals
Muthiah Manoharan, PhD. Dr. Muthiah (Mano) Manoharan serves as a Senior Vice President of Drug Innovation, a Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. In 2003, he was the first chemist hired at Alnylam. He and his team pioneered the discovery and development of the chemical modifications, GalNAc conjugation chemistry, and LNP delivery platform that make RNA interference-based human therapeutics possible. This work led to the approval of four RNAi therapeutics: ONPATTRO® (patisiran, 2018), GIVLAARI® (givosiran, 2019), OXLUMO® (lumasiran, 2020) and Lequio® (inclisiran, 2020). Dr. Manoharan has had a distinguished career as a world-leading chemist in the field of oligonucleotides. He is an author of more than 225 publications (nearly 50,000 Google Scholar citations with an h-index of 101 and an i10-index of 399) and over 400 abstracts, as well as an inventor of over 250 issued U.S. patents. Prior to Alnylam, Dr. Manoharan worked in the field of antisense oligonucleotide therapeutics at Ionis (formerly Isis) Pharmaceuticals and LifeCodes Corporation (1988-1990) . Dr. Manoharan received his B.Sc. and M.Sc. degrees in chemistry at the American College, Madurai, India. He earned his Ph.D. in chemistry (with Professor Ernest L. Eliel) at the University of North Carolina, Chapel Hill and carried out post-doctoral research (with Professor John A.Gerlt) in the field of oligonucleotide chemistry at Yale University and at the University of Maryland. Dr. Manoharan is the winner of the Lifetime Achievement Award of the Oligonucleotide Therapeutics Society (2019), the M. L. Wolfrom Award from the American Chemical Society (2007) and D. Horton Industrial Carbohydrate Chemistry Award from the American Chemical Society (2021).
Thomas McCauley, PhD, CSO, Omega Therapeutics, Inc.
Dr. Thomas McCauley joined Omega Therapeutics as Chief Scientific Officer in August 2019. Prior to Omega, he served as CSO at Zikani Therapeutics (formerly Macrolide Pharmaceuticals) and before that at Translate Bio (formerly RaNA Therapeutics). In these roles, Dr. McCauley was responsible for overall scientific vision, strategic direction and leadership of research and development. He provided scientific, technical and operational oversight for advancement of the company's oligo, mRNA and novel macrocyclic technology platforms and management of portfolio therapeutic programs in pulmonary, metabolic, genetic and infectious disease. Previously, Dr. McCauley served as Vice President and Head of Global Nonclinical Development at Shire Pharmaceuticals, where he made significant contributions to the development and global approvals of many of Shire's products, including Replagal® for Fabry disease, Vpriv® for Gaucher disease, Elaprase® for Hunter syndrome, Firazyr® for hereditary angioedema and Xiidra® for dry eye disease. Dr. McCauley has more than 20 years of experience in the biotechnology and pharmaceutical industries and has authored more than 40 scientific publications, book chapters and patents. He holds undergraduate and graduate degrees from Cornell University and received his Ph.D. from the University of Alabama at Birmingham.
Lubomir Nechev, PhD, Senior Vice President, CMC Development, Alnylam Pharmaceuticals
Dr. Nechev leads the CMC Development Department at Alnylam Pharmaceuticals. He joined Alnylam in March 2004 and in the last 18 years, he has led the development and implementation of the CMC strategy for siRNA therapeutics, which is now used in five approved products - ONPATTRO, GIVLAARI, OXLUMO, Leqvio(Novartis), and AMVUTTRA. ONPATTRO is the first approved siRNA therapeutic and the first approved lipid nanoparticle (LNP) formulated oligonucleotide. GIVLAARI is the first approved GalNAc-conjugated siRNA. Before Alnylam, Dr. Nechev worked at Ribozyme Pharmaceuticals (RPI) and the Nucleic Acids Synthesis unit of Transgenomic, Inc. Dr. Nechev received his PhD degree in Organic Chemistry from St. Kl. Ohridski University, Sofia, Bulgaria and completed his postdoctoral training at Vanderbilt University, Nashville, Tennessee.
Emily Noonan Place, PhD, Senior Consultant, Biologics Consulting
Emily is currently a Senior Consultant at Biologics Consulting. She recently made a transition from working as a pharmacology/toxicology reviewer at the FDA in CDER's Office of Oncologic Diseases for over 8 years. At FDA, she worked on a team that supported a division reviewing applications in benign heme and hematological malignancies, and also on a team in a division reviewing applications in solid tumors. During her time at the FDA co-chaired a Pharmacology/Toxicology Subcommittee for Oligonucleotide Therapeutics on which she performed consults, provided education, developed internal practice, and oversaw the safety and activity of drug applications for Oligonucleotide Therapeutics in all of the therapeutic divisions across the Office of New Drugs. Emily received a bachelor's in science in Biology from State University of New York at Binghamton, her PhD in Cell Biology from University of Connecticut, and an MPH in Epidemiology at the University of California at Berkeley. Following her PhD, she was an Associate Investigator at San Francisco VA Medical Center from 2007-2009 where her research focused on miRNA dysregulation in prostate cancer. She completed a postdoctoral fellowship at Stanford School of Medicine testing chemotherapeutics in a translational model of 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. Her research has culminated in several publications in peer-reviewed journals and book chapters. She is also currently Faculty at the University of California at Berkeley, School of Public Health, in the Division of Biostatistics and teaches Biostatistics in the master's in public program.
Steve Pascolo, PhD, Senior Scientist, University Hospital of Zurich; Founder & CEO, Miescher Pharma GmbH
Trained as an immunologist at the Pasteur Institute (Paris, France), Dr. Steve Pascolo used mouse models to test and develop mRNA-based vaccines (direct injection of mRNA) during his post-doc in Tuebingen, Germany from 1998 till 2000. In 2000, he co-founded CureVac with Dr. Hoerr and Dr. von der Mulbe. Dr. Pascolo was Chief Scientific Officer (CSO) of the company from 2000 till 2006, developing the technology, implementing the worldwide first GMP production of mRNA and in 2003 performing the worldwide first mRNA vaccine clinical studies where humans (including himself) got injections of in vitro transcribed mRNA. In 2006, he joined the oncology department of the University Hospital of Zurich, Switzerland and continued the development of immunotherapies based on RNA. In 2008, he founded Miescher Pharma to support this work. In 2017, Dr. Pascolo implemented in Zurich an academic mRNA platform https://www.cancer.uzh.ch/en/Research/mRNA-Platform.html. In collaboration with several research and clinical departments in Zurich he optimizes, tests and implements mRNA based vaccines and therapies.
Leo Ziqing Qian, PhD, Co-Founder & Vice President, Discovery Research, Entrada Therapeutics
Ziqing "Leo" Qian, Ph.D. is co-founder and Vice President, Discovery Research at Entrada Therapeutics, a Boston-based biotechnology company dedicated to transforming the treatment of devastating diseases using intracellular biologics. Leo co-invented Entrada's Endosomal Escape Vehicle (EEV) technology, which is applied to the design and development of intracellular delivery of otherwise impermeable biological cargos, including oligonucleotides, peptides, and proteins. Leo obtained his PhD in organic chemistry from Ohio State University. He has co-authored over 30 peer-reviewed publications and two book chapters, as well as co-inventor on over 10 patents.
Dmitry Samarsky, PhD, CTO, Sirnaomics
Dmitry Samarsky, PhD has been at the inception of RNAi technology and drug development - starting in 2001 as Director of Technology Development at Sequitur (acquired by Invitrogen) and (in 2005) as Director of Technology Development at Dharmacon (now part of GE). He then served as VP of Technology Development at RXi Pharmaceuticals, USA (2007-2011), SVP of Technology and International Business Development at RiboBio, China (2011-2016) and, most recently, as Chief Scientific Officer at Silence Therapeutics, Germany/UK (2016-2018). Dr. Samarsky has authored more than 40 scientific papers, articles, book chapters, patents and patent applications. He has been an invited speaker at more than 100 international conferences, and currently serves on the Scientific Advisory Boards for the OTS (Oligonucleotide Therapeutics Society). Dr. Samarsky received his doctorate in biochemistry and molecular biology from University of Massachusetts, Amherst (1998), followed by a postdoctoral position as an H. Arthur Smith Fellow for Cancer Research in Michael Green's lab at University of Massachusetts Medical School (1998-2001).
Jeske Smink, Head of Drug Substance, Manufacturing, Silence Therapeutics
Jeske is a biologist with over 18 years of experience in the biotech industry and academic research. She has expertise in CMC aspects in different areas, including cell therapies, biologics, and siRNAs. After 7 years in academic research focusing on molecular mechanisms in bone development and diseases, she moved to co.don AG as Head of Scientific Affairs in 2012. Here her work focused on preclinical development and quality aspects of cartilage cell transplants. Following, she joined ProBioGen as CMC project leader, where she was overseeing customer projects, focusing on process development, scale-up and large scale GMP manufacturing of biologics. In 2020, Jeske joined Silence Therapeutics as Head of Drug Substance overseeing CMC development, including process and analytical development, scale-up and clinical product manufacturing of siRNA compounds.
Katarina Stenklo, Enterprise Solutions Commercial Activation Leader, Bioprocess, Cytiva
Katarina Stenklo holds a licentiate degree in biochemistry with more than 20 years of biopharma industry experience. She currently works for Cytiva's Enterprise Solutions business unit where she lends her expertise to developing end-to-end manufacturing solutions, including process workflows, process equipment, automation, consumables, and facilities. In the last few years, she has been developing and designing solutions for the manufacture of new molecules such as viral vectors, plasmids, and mRNA-based therapies.
Chandra Vargeese, PhD, CTO & Head, Platform Discovery Sciences, WAVE Life Sciences
Dr. Vargeese is one of the industry's leading oligonucleotide chemists with approximately 30 years of oligonucleotide research experience, authoring numerous scientific publications and patents in the field. Dr. Vargeese joined Wave Life Sciences in 2014 and was named Chief Technology Officer in 2020. She is responsible for the advancement of Wave's PRISMTM platform, which is foundational to all of Wave's drug discovery and development efforts. Before joining Wave, Dr. Vargeese served as Executive Director of Chemistry and Delivery, RNA Therapeutics Division at Novartis. She led siRNA delivery as Senior Director in the RNA Therapeutics division at Merck. She joined Merck through its $1.1 billion acquisition of Sirna Therapeutics, where she was Vice President of Chemistry. Dr. Vargeese served as Associate Director of Chemistry at NeXstar Pharmaceuticals and is the co-inventor of Macugen (pegaptanib), an approved therapy for wet AMD. She earned a PhD in organic chemistry at the Indian Institute of Science, Bangalore, India, and completed post-doctoral work at the University of Rhode Island.
Xiao Wang, PhD, Assistant Professor, Department of Chemistry, Broad Institute of MIT and Harvard
Xiao Wang is a core institute member of the Broad Institute of MIT and Harvard and an assistant professor in the Department of Chemistry at MIT. She started her lab in 2019 to develop and apply new chemical, biophysical, and genomic tools to better understand and tune tissue function at the molecular level. Wang joined the MIT and Broad after conducting postdoctoral research at Stanford University. She received her B.S. in chemistry and molecular engineering from Peking University in 2010 and her Ph.D. in chemistry from the University of Chicago in 2015.
Jonathan Watts, PhD, Associate Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical School
Jonathan K. Watts is Associate Professor at the RNA Therapeutics Institute of UMass Chan Medical School (Worcester, MA, USA). He completed his training at Dalhousie University (BSc), McGill University (PhD) and UT Southwestern Medical Center (Postdoctoral). After establishing his independent laboratory at the University of Southampton, UK, he moved to the RNA Therapeutics Institute in 2015. He has won over 20 awards including the 2013 OTS Young Investigator Award, the 2015 Vice Chancellor's award for teaching, and the 2018 Angel Award for ALS research. Current work in the Watts lab is focused on the optimization of ASO chemistry for use in the CNS and lung, and on the chemistry of guides and donors for genome editing. The Watts group works on both platform technology and disease applications and has contributed to the development of two drugs that have reached patients on a compassionate use basis. Jon is on the Board of Directors of the Oligonucleotide Therapeutics Society since 2015.
Mike Webb, PhD, Founder and CEO, Mike Webb Pharma; Former Vice President, API Chemistry & Analysis, GSK
Mike received his PhD from imperial college in London. He spent most of his career at GSK and its legacy companies primarily as an analytical scientist and then becoming the Vice President of Development Chemistry and Analysis for GSK in the UK. He was heavily involved in establishing initial GSK's oligonucleotide CMC development efforts. Mike has edited 3 books on the analysis of pharmaceuticals, including one on the analysis of oligonucleotides. Since leaving GSK in 2016, he has been consulting with Big Pharma and Biotechnology companies in the development, analysis and manufacture of therapeutic oligonucleotides from pre-clinical to marketing submission.
Ron Weiss, PhD, Professor, Biological Engineering, Massachusetts Institute of Technology
Ron Weiss is Professor in the Department of Biological Engineering and in the Department of Electrical Engineering and Computer Science at the Massachusetts Institute of Technology, and is the Director of the Synthetic Biology Center at MIT. Professor Weiss is one of the pioneers of synthetic biology. He has been engaged in synthetic biology research since 1996 when he was a graduate student at MIT and where he helped set up a wet-lab in the Electrical Engineering and Computer Science Department. After completion of his PhD, Weiss joined the faculty at Princeton University, and then returned to MIT in 2009 to take on a faculty position in the Department of Biological Engineering and the Department of Electrical Engineering and Computer Science. The research pursued by Weiss since those early days has placed him in a position of leadership in the field, as evidenced both by publications from his lab as well as a variety of awards and other forms of recognition. He pursued several aspects of synthetic biology, including synthesis of gene networks engineered to perform in vivo analog and digital logic computation. The Weiss lab also published seminal papers in synthetic biology focused on programming cell aggregates to perform coordinated tasks using engineered cell-cell communication with chemical diffusion mechanisms such as quorum sensing. Several of these manuscripts were featured in a recent Nature special collection of a select number of synthetic biology papers reflecting on the first 10 years of synthetic biology. While work in the Weiss lab began mostly with prokaryotes, during the last 5 years a majority of the research in the lab shifted to mammalian synthetic biology. The lab focuses both on foundational research, e.g., creating general methods to improve our ability to engineering biological systems, as well as pursuing specific health related applications where synthetic biology provides unique capabilities.http://groups.csail.mit.edu/synbio/
Drew Weissman, MD, PhD, Roberts Family Professor in Vaccine Research, Department of Infectious Diseases; Director of the Penn Institute for RNA Innovation and Professor, Department of Medicine, University of Pennsylvania
Drew Weissman, M.D., Ph.D. is a professor of Medicine at the Perelman School of Medicine, University of Pennsylvania. He received his graduate degrees from Boston University School of Medicine. Dr. Weissman, in collaboration with Dr. Katalin Kariko, discovered the ability of modified nucleosides in RNA to suppress activation of innate immune sensors and increase the translation of mRNA containing certain modified nucleosides. The nucleoside-modified mRNA-lipid nanoparticle vaccine platform Dr. Weissman's lab created is used in the first 2 approved COVID-19 vaccines by Pfizer/BioNTech and Moderna. They continue to develop other vaccines that induce potent antibody and T cell responses with mRNA-based vaccines. Dr. Weissman's lab also develops methods to replace genetically deficient proteins, edit the genome, and specifically target cells and organs with mRNA-LNPs, including lung, heart, brain, CD4+ cells, all T cells, and bone marrow stem cells.
Lior Zangi, PhD, Associate Professor, Department of Medicine, Cardiology and Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai
Lior Zangi, PhD, is an Associate Professor with Tenure at the Icahn School of Medicine at Mount Sinai, New York. He completed his education and training at the Weizmann Institute of Science, and Harvard University. Studies methods for delivering modified mRNA (modRNA) to the heart to induce cardiac regeneration, protection, or cardiovascular regeneration post-ischemic injury. He has established a new method, modRNA-based, for minimal invasive gene delivery into specific cell types and organs. His early work showed the first successful delivery of modRNA into cardiac and skeletal muscle, and pioneered using modRNA to treat ischemic disease. His invention led to the first successful Phase 2 cardiac modRNA therapeutic clinical trial using VEGFA modRNA. In the last few years, these modRNA technology have been used for COVID-19 vaccinations and promoting cardiovascular regeneration in ischemic heart disease. Currently, Prof. Zangi's laboratory investigates mRNA delivery method into healthy or unhealthy specific cell types and organs, to fight different diseases such as heart failure and cancer.
Xiaolei Zhuang, PhD, Scientific Liaison, Global Biologics, USP
Xiaolei Zhuang is a senior scientist II in Global Biologics at USP, who focuses on reference standard development. Before joining USP, she had been working on analytical and preclinical/clinical studies to support biological drug development in biopharmaceutical companies.
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