2025年 講演者
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Annemieke Aartsma-Rus, PhD, Professor of Translational Genetics, Leiden University Medical Center
Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. She played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy during her PhD research (2000-2004) at the Leiden University Medical Center (the Netherlands). As of December 2007 she became leader of the “DMD exon skip group”. Since 2013 she has a visiting professorship at the Institute of Genetic Medicine of Newcastle University (UK). She is President of the Oligonucleotide Therapeutics Society (2019-2020) and vice-chair of COST Action CA17103 (Delivery of antisense RNA therapies). In 2013 she was elected a member of the junior section of the Dutch Royal Academy of Sciences (DJA), which consists of what are considered the top 50 scientists in the Netherlands under 45. In 2018, she was one of the founding members of Young Academy Leiden (YAL), the local Leiden counterpart of the DJA. She has been selected as most influential scientist in Duchenne muscular dystrophy in the past 10 years by Expertscape based on contributions to the understanding and treatment of Duchenne muscular dystrophy four times in a row (2015-now).
Vikram Agarwal, PhD, Head of mRNA Platform Design Data Science, mRNA Center of Excellence, Sanofi
Vikram Agarwal completed his Ph.D. in Dr. David Bartel's lab at the Massachusetts Institute of Technology, where he utilized computational approaches to study the principles of microRNA targeting. He completed his post-doctoral research in Dr. Jay Shendure's laboratory at the University of Washington, where he applied deep learning methods and massively parallel reporter assays to investigate the mechanisms of transcriptional gene regulation. Transitioning into industry, Vikram worked at Calico Life Sciences LLC, an Alphabet company whose mission is to better understand the mechanisms underlying human aging. There he applied deep learning methods with Dr. David Kelley to better understand transcriptional and post-transcriptional gene regulation as well as its link to human genetics. He is currently the Head of mRNA Platform Design Data Science at the mRNA Center of Excellence at Sanofi, where his team is applying machine learning and deep learning methods towards the design of enhanced mRNA therapeutics.
Annabelle Biscans, PhD, Director, Oligonucleotides and Targeted Delivery, AstraZeneca
Annabelle Biscans is a Director in the Oligonucleotide Chemistry team at AstraZeneca R&D, Sweden. She has a robust expertise in designing and developing novel chemical modalities to improve oligonucleotide therapeutic potency, toxicity and delivery. Before joining AstraZeneca, she was a postdoctoral associate at the RNA Therapeutics Institute, USA in Anastasia Khvorova’s lab. There, she made major contributions to the development of novel chemical platforms for enhancing oligonucleotide extra-hepatic delivery. She was able to design and identify novel conjugated siRNAs that enable safe, sustainable and robust silencing in various extra-hepatic tissues including muscle, heart, and lung. This crucial discovery allows for the targeting of several new targets for therapeutic intervention and advance the oligonucleotide therapeutics field. In 2015, she received her PhD from the University of Montpellier, France, where her main research was focused on developing novel modified siRNA in siRNA prodrug-like approach.
George Bou-Assaf, PhD, Associate Director, Analytical Development, Biogen
George has a BS in chemistry and a PhD in Biochemistry. His graduate studies under Professor Alan Marshall at Florida State University focused on development and optimization of hydrogen/deuterium exchange mass spectrometry to study protein-protein interactions. Shortly after he graduated, he joined the technical development group at Biogen in 2011, where his job responsibilities focus on the biophysical characterization of proteins and gene therapy products. More recently, he manages a group of scientists focused on developing methods for the characterization of oligonucleotide products.
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.
Charles Chen, PhD, Senior Scientist, Advanced Drug Delivery, Pharmaceutical Sciences, AstraZeneca Pharmaceuticals, R&D
Dr. Charles Chen is a Senior Scientist at AstraZeneca working on designing and synthesizing novel lipids and focusing on non-viral nucleic acid delivery. Before joining AstraZeneca, Charles started his research journey when he was an undergraduate student and found his passion in drug development. Charles got his PhD in Chemistry at King’s College London under the supervision of Professor Martin Ulmschneider. In 2019, Charles moved to Boston in the US and did a 2-year postdoc training at the Massachusetts Institute of Technology and Massachusetts General Hospital, co-supervised by Professors Timothy Lu and Joanna Yeh. Charles’ combinational approaches have shown promising outcomes in antibiotics and cancer chemotherapy.
Jeffery M. Coller, PhD, Bloomberg Distinguished Professor of RNA Biology and Therapeutics, Johns Hopkins University
Jeff Coller is the Bloomberg Distinguished Professor of RNA Biology and Therapeutics at Johns Hopkins University. His lab has made seminal discoveries in the area of messenger RNA stability and translation. He received his Ph.D. in Cellular and Molecular Biology from the University of Wisconsin and was a postdoctoral fellow in the Howard Hughes Medical Institute at the University of Arizona. Prior to moving to Johns Hopkins University, Dr. Coller served as Director of the RNA Center at Case Western Reserve University where he held the Henry Willson Payne Distinguished Professorship. He studies the very essence of life: translation of the genetic code. His work has led to fundamental shifts in the understanding of gene expression by demonstrating that the genetic code is a major determinant of mRNA fate. He is the Co-founder of Tevard Biosciences. In 2018, Tevard Biosciences was awarded Pfizer’s Golden Ticket award for promising neuroscience startups. His publications have been cited over 6000 times and he currently holds numerous patents for RNA-based therapeutic applications.
Hagen Cramer, PhD, CTO, QurAlis Corporation
Dr. Hagen Cramer is a renowned expert in all chemistry-related aspects of oligonucleotide therapeutic development. He has been working in the biotech industry for over 20 years spanning discovery, development, and manufacturing of oligonucleotide-based therapeutics. Early in his career, he worked with Gemini Technologies and Ridgway Biosystems on the discovery of 2-5A antisense for the treatment of a variety of cancers and viral diseases. In 2005, he joined Girindus, which was later acquired by Nitto Denko Avecia, where he headed the process development department, eventually becoming Director of Operations. More recently, he joined Wave Life Sciences where he was responsible for securing drug substance and drug product for its clinical pipeline of stereodefined oligonucleotide therapeutics. In 2020, he joined QurAlis as Chief Technology Officer supporting all programs targeting diseases affecting the CNS.
Rakesh Dixit, PhD, DABT, President & Founder, Bionavigen Oncology, LLC and Regio Biosciences
Rakesh Dixit is an accomplished executive, inventor, and scientist with over 35 years of success with top biotechnology and pharmaceutical companies, including Merck, Johnson & Johnson, and Medimmune - AstraZeneca. Currently, he is President and CSO of Regio Biosciences and Bionavigen, LLC. He is a Board Member of Regio Biosciences and a key member of multiple scientific advisory boards. Rakesh is also a chief adviser and consultant for more than 20 companies worldwide. His biopharmaceutical peers selected Rakesh as one of the 100 Most Inspiring People in the Pharmaceutical Industry by PharmaVOICE in 2015. Rakesh received the Most Prestigious Award of Long-Standing Contribution to ADCs by World ADC (Hanson-Wade), 2020. From 2006 to 2019, Rakesh was a Global Vice President of the Biologics R&D at Medimmune - AstraZeneca. Rakesh has unique expertise in developing biologics (e.g., monoclonal antibodies, bispecific biologics, antibody-drug conjugates, fusion proteins, peptides, gene and cell therapies, etc.) and small-molecule biopharmaceuticals. His areas of expertise include discovery, early and late preclinical development, safety assessment, DMPK, and translational sciences. Dr. Dixit conducted extensive graduate and post-graduate training in Pharmacology/Toxicology-Biochemistry with both Indian and USA Institutions (e.g., Case Western Reserve University, Medical College of Ohio, University of Nebraska) and is a Diplomate and Board Certified in Toxicology from the American Board of Toxicology, Inc. since 1992.
Austin Draycott, PhD, CEO, Cloverleaf Bio
I am the Co-Founder and CEO of Cloverleaf Bio. Cloverleaf is an early stage spin-out from Yale, developing therapeutics to inhibit the activity of RNA modifying enzymes and improve patient outcomes in oncology. Before starting cloverleaf, I completeed my PhD in Wendy Gilbert's lab at Yale. My PhD research focused on the development of new methods to find and quantify RNA modifications, and on understanding the mechanisms by which aberrant expression of the enzymes that install these modifications impacts human health. For the first part of my PhD, I developed D-seq, a method to find a specific RNA modification, dihydrouridine in high throughput using sequencing. Using D-seq, we revealed that dihydrouridine is present in a number of unexpected classes of RNA, including snoRNA and mRNA, where it can alter RNA structure and function. For the second part of my PhD, I studied an enzyme called dihydrouridine synthase 2 (DUS2) which is frequently overexpressed in lung cancer. I showed in human cells and mouse xenografts that DUS2 suppresses ferroptosis, a metal-dependent non-apoptotic form of cell death to which many lung cancers are unusually sensitive. Mechanistically, DUS2 is required to maintain specific tRNA expression and support translation of specific proteins including metallothioneins which serve as key regulators of both metal and redox homeostasis. My results reveal a tRNA-specific vulnerability in lung cancer cells and demonstrate the therapeutic potential of targeting RNA modifying enzymes in cancer. Before grad school, I worked in Alice Ting’s group, mapping the spatial localization of proteins in mammalian neurons. In my free time I like to paddle my kayak, and generally explore the outdoors.
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.
Arwa El Hagrasy, PhD, Director, Regulatory Affairs CMC, Alnylam Pharmaceuticals
Arwa El Hagrasy is Director, Regulatory Affairs (CMC) at Alnylam Pharmaceuticals, with focus on regulatory CMC strategy for siRNA product lifecycle management. Prior to Alnylam, Arwa worked at FDA as quality assessment lead for ~7.5 years and has reviewed a wide variety of drug product applications submitted to the Agency, including those implementing emerging technologies and advanced pharmaceutical manufacturing. Arwa has over 8 years of prior experience in industry and academia, with focus on the implementation of process analytical technology tools and continuous manufacturing in the pharmaceutical industry. Arwa received her B.S. in Pharmacy, Cairo University, Egypt, Ph.D. in Pharmaceutical Sciences from Duquesne University in Pittsburgh, and did a post-doctoral fellowship in the Chemical Engineering Department at Purdue University, IN.
Shanthi Ganesh, PhD Director, Pharmacology, Global Nucleic Acid Therapies, Novo Nordisk
Shanthi is the Director of Pharmacology at Dicerna, a Novo Nordisk company, with over 20 years of industry experience in nucleic acid therapeutics, drug delivery, cancer drug discovery, and gene therapy. At Dicerna, she was responsible for bringing the first two GalXC-Plus conjugates to clinical development for the treatment of cancer patients. Prior to that, she spent more than 10 years at NIBR and two other gene therapy companies developing RNAi therapeutics and oncolytic viruses for cancer treatment. Throughout her career, she has published extensively in the areas of RNAi, cancer therapeutics and gene therapy and holds several patents and awards for her work in this field. She has also been a member of AACR for several years and has attended and presented her work at several international conferences. She earned her PhD in RNAi therapeutics for cancer applications at Northeastern University.
Paloma Giangrande, PhD, CTO, Eleven Therapeutics
Dr. Giangrande was until recently 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.
Bas Groenendaal, PhD, Director CMC, Vico Therapeutics
Bas obtained his PhD in organic chemistry in the group of Prof. Romano Orru at the Vrije Universiteit Amsterdam, followed by a post-doc in the group of Prof. Nicholas Turner at the Manchester Institute of Biotechnology. He joined Prosensa (later BioMarin Nederland) as a scientist in the CMC department in 2013; first working in the lab on the in-house manufacturing of oligonucleotide batches, and later on becoming responsible of the supply of both internally and externally produced batches. He is currently Director CMC at Vico Therapeutics where he manages the complete CMC trajectory for VICO’s assets.
Iris Grossman, PhD, Chief Therapeutics Officer, R&D, Eleven Therapeutics US, Inc.
On the leading edge of Personalized Medicine, Big Data Analytics and BioPharmaceutical R&D for >20 years, Iris Grossman, PhD is the Chief Therapeutics Officer of Eleven Therapeutics, where she oversees the strategy and development of novel RNA-based therapeutics discovered by the company’s platform, which uniquely marries between combinatorial chemistry and A.I. to decipher the structure-activity relationship of durable, effective and safe respiratory and metabolic therapeutics. Prior to joining Eleven, Iris served in Chief Scientific Officer roles (CAMP4, Amide Technologies (acting)) and consulted for top VCs in the Boston area. Prior to that, Iris held leadership positions in, or consulted for Big Pharma (GSK, Eli Lily, Takeda and Teva - where she was VP, Head of Early-Stage Development). She specializes in leveraging multi-dimensional genomic insights and eHealth as engines of therapeutic discovery, development and lifecycle management across a wide range of modalities and therapeutic areas. Dr. Grossman serves as Chairman of the Pharma/Life Sciences SAB at BC Platforms, and as Strategic Founding Advisor at Modulight.bio, and CGRP Diagnostics. She was a pioneering Steering Committee member of Tipa (Maccabi biobank) and is an active member and strategic advisor to the 8400 Healthcare network. Iris has championed various multi-year, multi-million dollars partnerships and collaborations, as well as won multiple awards, throughout her career, including the prestigious Top 40 under 40 award (2013) by Globes - Israel’s business arena, and various recognition awards for contributions to R&D programs and cutting edge science. Dr. Grossman earned her B.Sc. in medicine at the Technion, and a Ph.D. in medicine, population genomics and pharmacogenetics from the Technion, co-mentored with the Weizmann Institute. Her post-doc fellowship in Personalized Medicine and Population Genomics was completed at Duke university. She recently completed a Harvard Business School Executive HealthTech program.
Mahender Gurram, PhD, Senior Director, Entrada Therapeutics
Trained synthetic organic chemist with >20 years of experience in synthesis of drug substances, intermediates in CROs and in innovative Biotechs. OVer 12 years of experience in clinical and late phase product development, and CMC. Currently overseeing development and manufacturing of PMOs and related drug substances at Entrada therapeutics.
Sushma Gurumurthy, PhD, Director, Oncology Research, Moderna, Inc.
Sushma Gurumurthy, PhD is a distinguished scientist with over 15 years of experience in oncology research and mRNA therapeutics. Since joining Moderna in 2015, Sushma has led the preclinical discovery and development of mRNA therapeutics for application in oncology. Prior to Moderna, she led different oncology early-stage discovery programs at the Belfer Institute in Dana Farber Cancer Institute. She received her PhD from the University of Kentucky and completed her postdoctoral training at the Massachusetts General Hospital Cancer Center and Harvard Medical School.
Nagy Habib, ChM, FRCS, Professor of Surgery, Imperial College London
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).
Ian Harding, PhD, Senior Scientist I, Wave Life Sciences
Completed my Bachelor's degree in Biomedical Engineering at Bucknell University after which I attended Northeastern University to receive my PhD in Bioengineering. Following my PhD, I began working at Wave Life Sciences in the Platform Biology group and have now been here for almost 4 years.
Michael Hellerstein, Head of Operations, Vaxxinity
Michael Hellerstein is Head of Operations at Vaxxinity, a company developing novel immunotherapeutic products against neurodegenerative disorders and other chronic diseases. At Vaxxinity, Mr. Hellerstein is responsible for multiple functional areas including program management, manufacturing, process and analytical development, and quality control. Vaxxinity’s platform technology utilizes both oligonucleotides and peptides, and Mr. Hellerstein is responsible for the scale-up, manufacture, and testing of these molecules. Initially trained as a molecular biologist, Mr. Hellerstein worked at several companies including Pfizer, Cell Signaling Technology, and GeoVax before he joined Vaxxinity. Mr. Hellerstein has a broad background in product development encompassing research and development, manufacturing, quality control and assurance, regulatory affairs, and program and portfolio management.
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.
Edward Kaye, MD, CEO and Director, Stoke Therapeutics
Ed Kaye, a pediatric neurologist, is the chief executive officer and a director of Stoke Therapeutics. Ed previously served as CEO of Sarepta Therapeutics, where he led the successful push to win FDA approval of Exondys 51, a pioneering drug for Duchenne muscular dystrophy. Before taking on the CEO role at Sarepta, he served as the company’s chief medical officer for six years, leading the drive to develop Exondys 51. He also served on Sarepta’s board of directors. Earlier in his career, Ed spent 10 years at Genzyme Corporation, most recently as group vice president of clinical development, where he supervised clinical research in programs focused on lysosomal storage disease and genetic neurological disorders. At Genzyme, Ed also held various roles, including vice president of medical affairs for lysosomal storage diseases, vice president of clinical research and interim head of PGH global medical affairs. Ed currently serves as a member of the boards of directors at Cytokinetics, Inc. and the Massachusetts Biotechnology Council. Prior to entering the biotech industry, Ed trained in pediatrics, pediatric neurology and biochemical genetics. He was on the research staff at Massachusetts General Hospital and Tufts University Medical Center and was the chief of biochemical genetics at the Children’s Hospital of Philadelphia. He remains on the pediatric neurology staff at Boston Children’s Hospital. Ed earned his B.S. in biology from Loyola University and earned his M.D. at the Loyola University Stritch School of Medicine.
Jaspreet Khurana, PhD, Senior Director, mRNA Programming, Strand Therapeutics, Inc.
Dr. Jaspreet Khurana is an RNA molecular biologist by training who is passionate about transforming innovative platforms into meaningful therapies for patients. Following a PhD in Biomedical Sciences from UMass medical school, Worcester, he joined Princeton and Columbia University for a postdoc where he utilized functional genomics to study genomic rearrangements. With 13+ years of post-PhD expertise in RNA molecular biology, he has proven experience leading cross-functional teams across early stages of drug development. Currently he is focused on mRNA platform development for immunotherapy programs at Strand Therapeutics.
Anastasia Khvorova, PhD, Professor, RNA Therapeutic Institute, University of Massachusetts Medical School
Anastasia Khvorova, PhD, is The Remondi Family Chair in Biomedical Research and Professor in the RNA Therapeutics Institute (RTI) and Program in Molecular Medicine at the University of Massachusetts Medical School (UMMS). She has more than twenty years of experience developing oligonucleotide technology and therapeutics. Her lab brings together hardcore organic and oligonucleotide chemists, RNA biologists, and pharmacologists to develop novel approaches and solutions to understanding natural and therapeutic RNA trafficking and delivery. She established the RTI’s Nucleic Acid Chemistry Center, which provides expertise in RNA chemistry to labs within and outside UMMS, and is the only non-profit center in North America capable of synthesizing complex RNAs at scales necessary to support both in vitro and in vivo studies. Dr Khvorova joined UMMS after several years in industry, during which she served as Chief Scientific Officer at lead biotech companies (Dharmacon, ThermoFisher; RXi Pharmaceuticals) and co-founded several startups. She is At-Large Director and Scientific & Research Council Chair of the American Society of Gene & Cell Therapy and served for several years as Director of the Oligonucleotide Therapeutics Society. Dr Khvorova is named as inventor on more than 150 patents and 200 patent applications, and she has authored more than 80 peer-reviewed publications, including seminal articles in Cell, Nature, and Nature Biotechnology (citation index exceeding 2000 per article) defining the field of RNAi drug design and development. Dr Khvorova is principal investigator on four major National Institutes of Health grants.
William Kiesman, PhD, Chief Technology Officer, Alltrna
WILLIAM KIESMAN, Chief Technology Officer of Alltrna, Cambridge, MA, USA, has more than two decades of experience in the design, development, and manufacturing of small molecule and oligonucleotide therapeutics. Prior to joining Alltrna, he served as the Vice President and Head of Oligonucleotide and Small Molecule Development at Biogen. During his 24 years with Biogen, he was responsible for a wide range of activities from medicinal chemistry research and automated parallel synthesis to building both the chemical development and the end-to-end oligonucleotide development and manufacturing teams. Will and his teams have supported dozens of small molecule and oligonucleotide clinical programs and played pivotal roles in the successful worldwide regulatory CMC approvals of Tecfidera® (dimethyl fumarate) in multiple sclerosis, Spinraza® (nusinersen) for spinal muscular atrophy and Qalsody® (tofersen) for ALS. Will earned his BS and PhD in Chemistry from the University of Connecticut, Storrs-Mansfield, CT, USA and pursued his postdoctoral research at Duke University, Durham, NC, USA. He is co-author on more than 40 publications and patents and has contributed to five book chapters.
Edo Kon, PhD, Director of Business Development, RiboX Therapeutics
Edo Kon is the Director of Business Development at RiboX Therapeutics, where he leverages his extensive background in biotechnology and strategic planning to identify emerging trends and novel therapeutic opportunities. Before this, he served as the Principal Scientist of Lipid Nanoparticle (LNP) Science at RiboX Therapeutics. He founded and led the LNP research teams to develop novel formulations and strategies for circular RNA delivery. His past experience includes leading projects to develop mRNA-LNPs as cancer nanomedicines and innovative vaccines against lethal antibiotic-resistant bacteria. He is the author of multiple high-impact manuscripts that focus on RNA delivery. He holds a PhD and an MBA from Tel Aviv University.
Arthur Krieg, MD, Founder, President and Acting CEO/CSO, Zola Therapeutics
Art Krieg, MD, is currently an adjunct Professor in the UMass Chan Medical School RNA Therapeutics Institute and serves on the scientific advisory boards of several companies developing oligonucleotide therapeutics. Art founded Zola Therapeutics in 2023, and serves as CEO with the goal of developing a new generation of TLR7/8/9 oligonucleotide agonists for cancer immunotherapy, and small molecule antagonists for the treatment of SLE and other autoimmune diseases. Previously, Art founded Checkmate Pharmaceuticals, until its acquisition by Regeneron in 2022. Previously, Art was CSO at Sarepta until July 2014; co-founder and CEO at RaNA Therapeutics from 2011 to 2013; CSO of Pfizer’s Oligonucleotide Therapeutics Unit from 2008 to 2011; co-founder, CSO of Coley Pharmaceutical Group from 1997 to 2008, and Professor, University of Iowa College of Medicine Division of Rheumatology from 1991 to 2001. Art co-founded the first antisense journal, Nucleic Acid Therapeutics, which he edited for 16 years, and the Oligonucleotide Therapeutics Society, for which he recently served as President. He has published more than 250 scientific papers, and is an inventor on >50 issued US patents covering oligonucleotide technologies.
Venkat Krishnamurthy, PhD, Senior Vice President & Head of Platform, Korro Bio, Inc.
Venkat Krishnamurthy, Ph.D., is senior vice president and Head of Platform at Korro Bio, with 12+ years of experience in RNA based therapeutics, oligonucleotide chemistry and non-viral delivery technologies. Before arriving at Korro, Dr. Krishnamurthy held an executive director role at Eli Lilly & Company, where he was responsible for strategic direction for Lilly’s genetic medicine portfolio. Prior to this, Dr. Krishnamurthy was a team lead at AstraZeneca, where he built a new modalities group at the Boston site to support the company’s mRNA and genome editing projects from discovery to clinic. He began his industry career at Dicerna Pharmaceuticals and was one of the early chemists to advance the company’s siRNA platform. He also made critical contributions to the advancement of Nedosiran, Dicerna’s first GalNAc-siRNA program currently in Phase 3. Ahead of his experience in industry, Dr. Krishnamurthy was a faculty member at Harvard Medical School, where he led a lab focused on non-viral delivery for cell therapy and tissue engineering. He has contributed to over 30 publications and patents, and some of his work laid the foundation for projects funded by the National Institutes of Health. Dr. Krishnamurthy earned his doctorate in organic chemistry from the University of Illinois, Chicago, and completed postdoctoral training at Princeton University. He also holds bachelor’s and master’s degrees in chemistry from Loyola College and the Indian Institute of Technology, Chennai, respectively.
Sarah Lamore, PhD, DABT, Senior Director, Toxicology, PepGen
Sarah was until recently 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, Professor of Biochemistry, Technical University of Munich; Senior 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.
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.
Mark Lowenthal, PhD, Research Chemist, Analytical Chemistry, National Institute of Standards and Technology (NIST)
Mark Lowenthal received a PhD in chemistry from the University of Maryland in 2002 and completed post-docs at the National Cancer Institute in 2005 and at the University of Colorado Health Sciences Center in 2007. He has been a research chemist in the Biomolecular Measurement Division at NIST for the last 17 years, developing high-impact Standard Reference Materials and designing novel measurements to support the clinical chemistry and biomanufacturing communities focusing primarily on mass spectrometry applications.
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.
Ajit Magadum, PhD, Assitant Professor, Department of Cardiovascular Diseases + ACDC, Lewis Katz School of Medicine, Temple University
Dr. Ajit Magadum, Ph.D., is a Distinguished Research Fellow at Department of Cardiovascular Sciences at Temple University in Philadelphia, USA. He received his Ph.D. from the Max Planck Institute for Heart and Lung Research, Germany. During his postdoctoral tenure at Mount Sinai in New York, Ajit delved into the innovative realm of modified mRNA (modRNA) as a potent gene therapy tool for combating cardiovascular and metabolic diseases (CVMD). He made significant strides in designing modRNA delivery systems tailored for the cardiovascular system, employing various carriers, to ensure robust and enduring modRNA expression within cardiac tissues. Ajit's groundbreaking work unveiled a treasure trove of novel genes (6 targets), delivered as modRNA to the heart, inducing cardiomyocyte proliferation and cardiac regeneration, inhibiting cardiac hypertrophy and fibrosis offering promising avenues for treating CVMD and fibrotic conditions. Ajit is celebrated for pioneering the development of cell-specific mRNA delivery platforms known as SMARTs (Specific Modified mRNA Translation System) in the context of CVD in 2016. This innovation allowed for the precise targeting of modRNA expression exclusively in cardiomyocytes or non-cardiomyocytes within the heart, opening up new horizons for cell-specific mRNA therapeutics in the realm of CVMD. He has published over 20 papers, along with the successful filing of 3 patents, which have been licensed and sublicensed to leading biotechnology companies. He won the Outstanding Research Innovation Award from Mount Sinai Hospital, New York, in 2017 for his contributions to mRNA therapeutics development for CVMD. Ajit received the esteemed ISHR-NAS Young Investigator Award (YICA, runner-up) in 2022 and the prestigious Melvin L. Marcus Early Career Investigator Award from the American Heart Association (AHA) in 2022. At present, Ajit's research endeavors continue to revolve around leveraging modRNA and cell-specific modRNA as innovative therapeutic modalities to target CVMD.
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).
Rubina Parmar, PhD, Vice President, Chemistry & Delivery Sciences, Intellia Therapeutics
Rubina Parmar, PhD, is the vice president at Intellia Therapeutics, a leading clinical-stage genome editing company developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. At Intellia, Rubina oversees the Company's chemistry & delivery sciences group and co-lead platform development. Prior to Intellia, Rubina worked in the medicinal chemistry group at Alnylam Pharmaceuticals as well as the RNAi therapeutics group at Merck. Over those years, she was involved in several research projects to enhance the potency and delivery of nucleic acids to specific organs. Rubina received her PhD in organic chemistry from Punjabi University, India.
Dan Peer, PhD, Professor & Vice President, Research & Development, Tel Aviv University
Dan Peer is a Professor and the Director of the Laboratory of Precision NanoMedicine at Tel Aviv University (TAU). He is also the Vice President for Research and Development at Tel Aviv University. From 2017 - Present, he is the Founding and Managing Director of the SPARK program of Translational Medicine at TAU. Prof. Peer’s work was among the first to demonstrate systemic delivery of RNA molecules using targeted nanocarriers to the immune system and he pioneered the use of RNA interference (RNAi) in immune cells. His lab was the first to show systemic, cell specific delivery of modified mRNA in an animal to induce therapeutic gene expression of desired proteins. This has enormous implications in cancer, inflammation and infection diseases (e.g. COVID 19 mRNA vaccines). In addition, his lab was the first to show high efficiency, systemic, cell specific therapeutic genome editing in cancer. Prof. Peer has more than 130 pending and granted patents. Some of them have been licensed to several pharmaceutical companies and one is currently under registration (as a new biological drug in Inflammatory Bowel Disease). In addition, based on his work, five spin-off companies were generated aiming to bring innovative personalized medicine into clinical practice. Prof. Peer received more than 30 awards and honors and he serves on the scientific advisory board and as Board Member of more than 15 companies, and on the editorial board of more than 20 journals. In 2014, he was elected to the Israel Young Academy and in 2023 he was elected to the US National Academy of Engineers (NAE).
Dmitry Samarsky, PhD, Former 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).
Kuldeep Singh, PhD, Senior Director & Head Pathology, Wave Life Sciences
Kuldeep Singh is currently the Head of Pathology at Wave Life Sciences. He obtained his BVSc (Bachelor of Veterinary Sciences) from G.B. Pant University of Agriculture and Technology in India, MS from Utrecht University, The Netherlands, and PhD and residency in anatomic pathology from Oklahoma State University. He joined the American College of Veterinary Pathologists as a diplomate in 2009. Prior to joining Wave Life Sciences, he worked as an Investigative Pathologist at Sanofi and as a Director of Toxicologic Pathology at WuXiApptech. He served the University of Illinois as Head of Anatomic Pathology and Clinical Associate Professor in the College of Veterinary Medicine, Urbana-Champaign. His professional interests include oligonucleotides, toxicology, biomarker identification, safety assessment, animal model characterization, and investigative and digital pathology. Kuldeep has authored or co-authored more than 55 peer-reviewed articles, book chapters, and more than 75 abstracts and posters. He is currently an ad hoc reviewer for 14 journals. Kuldeep has presented at multiple national and international conferences as an invited speaker. He is a primary or co-investigator on 2 patents (application filed) based on oligonucleotides.
Eric Swayze, PhD, Executive Vice President, Research, Ionis Pharmaceuticals
Dr. Swayze is Executive Vice President of Research at Ionis Pharmaceuticals. He is responsible for leading preclinical antisense drug discovery and antisense technology research. Previously, Dr. Swayze was vice president of Chemistry and Neuroscience Drug Discovery at Ionis, overseeing the advancement of multiple programs to clinical development. He joined Ionis in 1994 and has contributed to key technology advancements, including Ionis' Generation 2.5 chemistry and Ligand-Conjugated Antisense (LICA) technology. He is an author on more than 150 scientific manuscripts, several reviews and book chapters, and is an inventor of more than 140 issued US patents. He received his PhD in Organic Chemistry at the University of Michigan under the guidance of Professor Leroy B. Townsend.
David Tabatadze, PhD, President, ZATA Pharmaceuticals, Inc.
In 2009, Dr. David Tabatadze cofounded ZATA in partnership with Dr. Paul Zamecnik (1912-2009) where he has served as the President and CEO. Dr. Tabatadze has led the development of novel nucleic acid platform technology enabling the synthesis of next generation oligonucleotides (ON) with custom-designed properties based on the incorporation of various branched chemical groups on the internucleoside phosphates. ZATA’s ONs (ZONs) are applicable to all currently known oligotherapy-based approaches including antisense and CRISPR. Dr. Tabatadze has significant experiencing in consulting medical device companies in the development and FDA clearance of commercially available light-based medical devices. His thirty-plus years of R&D experience includes developing novel drug candidates and diagnostic probes, business development and management, execution of IP portfolio and regulatory submission. Dr. Tabatadze possesses a MS in organic chemistry, a PhD in bioorganic chemistry, and has received post-doctoral training in nucleic acid chemistry at the Worcester Foundation for BioMedical Research (Shrewsbury, MA). He has also co-authored over three dozen peer-reviewed papers, patent applications, and patents. At ZATA, Dr. Tabatadze has established business and collaborative relationships with several biotech and academic institutions locally and internationally.
Lori Troup, Director, Analytical Development, Novo Nordisk
Lori is the Director of Analytical Development for Dicerna Pharmaceuticals, a Novo Nordisk company, where her team is responsible for method development and validation activities for custom starting materials, drug substance and drug product, as well as drug substance characterization, drug substance and drug product release and stability studies, and CMC regulatory authoring. Prior to joining Dicerna in 2018, Lori spent 12 years at Agilent Technologies in a number of different roles within Quality Control, Analytical Development, and Analytical Services. Lori holds a B.S. in Chemistry from Abilene Christian University.
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.
Jonathan Watts, PhD, Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical School
Jonathan K. Watts is 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.
Timothy Weeden, Vice President, Head of Platform, Dyne Therapeutics
Tim Weeden is Head of Platform Development at Dyne and was one of the first company hires, leading the design and implementation of the FORCE platform. He has over 20 years’ experience in discovery and development of small molecules, peptides and antibody therapeutics across areas of immunology and rare disease.
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/
Jim Weterings, PhD, Vice President Research, RNA Therapeutics & Delivery, Sirnaomics
Jimmy Weterings, PhD, is Senior Director, Head of Technology Development at Sirnaomics, USA. Jimmy has had a tight bond with oligonucleotides since 2002, performing research on PNA (conjugates) in the van Boom group at Leiden University, the Netherlands, and targeted SSO conjugates at Prosensa. During his PhD at Leiden University he synthesized and studied CpG oligonucleotide and other TLR ligand-containing conjugates. At Cenix BioScience, he developed targeted siRNA conjugates for CNS delivery. And at Cristal Therapeutics he led various projects on the (targeted) nanomedicine delivery of oligonucleotides in oncology. In 2019 he joined AstraZeneca in Sweden, to further the advancement of (targeted) oligonucleotide therapeutics. In December, 2022, Jimmy joined Sirnaomics in the USA as Senior Director, designing novel oligonucleotide chemistries and targeted versions thereof. He now leads RNA therapeutics & delivery efforts as Vice President Research. Jimmy (co-)authored 13 articles and holds several patents for drug delivery-enabling technologies, including the most recent one on TMTHSI, ClicR exclusively licensed to Synaffix and commercially available for R&D purposes.
Renee Williams, Founder and Managing Partner, Williams Biotech Consulting; Independent Board Director, AGS Therapeutics
Renee Williams is a chief executive, business strategist, operator, and change agent with diversified experience working with large global, mid-market, and early-stage life science companies. She has a foundation in the science and business of RNA for large matrixed companies and entrepreneurial organizations, and direct experience in drug discovery, business operations, and external strategy for a broad range of modalities. Navigating complexities, delivering innovative solutions, and creating strategic partnerships within the pharmaceutical industry are hallmarks of her career. Building on a decade of experience in pharma, Dr. Williams recently founded and is managing partner of Williams Biotech Consulting which is a boutique firm specializing in business development liaison services with expertise in RNA therapeutics, genetic medicines, and non-viral delivery.
Steven Wolk, PhD, Vice President, Analytical Chemistry, Editas Medicine
Steve is currently the Vice President of Chemistry & Boulder Site Head for Editas Medicine, where he leads the Process Chemistry and Analytical Sciences/Structural Biology teams. His scientific teams are responsible for gRNA and mRNA manufacture and characterization, conjugation chemistries, as well as analytical method development, and biophysical and structure/function characterization. He received his bachelor’s degree in chemistry from UC San Diego, where he received the Harold Urey Award. He completed his Ph.D. work in Biophysical Chemistry at UC Berkeley under Dr. Ignacio Tinoco, where he used 2D-NMR and other spectroscopic techniques to characterize nonstandard DNA structures. He has also led analytical groups at a polymer company (Rohm & Hass) as well as numerous other biotechnology companies (NexStar, Invenux, Amgen & SomaLogic), and has a broad background in analytical techniques used for the characterization of both small and large molecules.
