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The Promise and Progress of
RNA Delivery & Gene Editing
March 20, 2024
and Progress of
RNA Delivery & Gene Editing
March 20, 2024
In Vivo Gene Editing
Biochemist and Nobel Prize-winning co-inventor of CRISPR technology Jennifer Doudna, PhD is a biochemist at the University of California, Berkeley. Her groundbreaking development of CRISPR-Cas9 — a genome engineering technology that allows researchers to edit DNA — with collaborator Emmanuelle Charpentier earned the two the 2020 Nobel Prize in Chemistry and forever changed the course of human and agricultural genomics research. She is also the Founder of the Innovative Genomics Institute, the Li Ka Shing chancellor’s chair in Biomedical and Health Sciences, and a member of the Howard Hughes Medical Institute, Lawrence Berkeley National Lab, Gladstone Institutes, the National Academy of Sciences, and the American Academy of Arts and Sciences. She is a leader in the global public debate on the responsible use of CRISPR and has co-founded and serves on the advisory panel of several companies that use the technology in unique ways. Doudna is the co-author of “A Crack in Creation,” a personal account of her research and the societal and ethical implications of gene editing. Learn more at .
Dr. Michael Snyder is the Stanford W. Ascherman Professor and Chair of the Genetics Department at Stanford Medicine. He was recruited by Stanford in 2009 to chair the Genetics Department and direct the Center for Genomics and Personalized Medicine. Under his leadership U.S. News & World Report has ranked Stanford University first in Genetics, Genomics, and Bioinformatics every year for the past decade. As the leading pioneer of 21st century healthcare, Dr. Snyder invented and significantly advanced many industry-standard approaches to personalized medicine. Most recently his research involving longitudinal baseline profiling and state-of-the-art “omic” technologies research has greatly accelerated the advancement of precision medicine. As an entrepreneur, Dr. Snyder’s co-founded companies have collectively raised $242 million in venture capital and are worth more than $6 billion in value. Dr. Snyder also serves on the board for a number of other companies.
Robert Langer is one of 8 Institute Professors at the Massachusetts Institute of Technology (MIT); being an Institute Professor is the highest honor that can be awarded to a faculty member. He has written over 1,500 articles, which have been cited over 412,000 times; his h-index of 319 is the highest of any engineer in history and the 3rd highest of any individual in any field. His patents have licensed or sublicensed to over 400 companies; he is a cofounder of a number of companies including Moderna. Dr Langer served as Chairman of the FDA’s Science Board (its highest advisory board) from 1999-2002. His over 220 awards include both the United States National Medal of Science and the United States National Medal of Technology and Innovation (he is one of 3 living individuals to have received both these honors), the Charles Stark Draper Prize (often called the Engineering Nobel Prize), Queen Elizabeth Prize for Engineering, Albany Medical Center Prize, Breakthrough Prize in Life Sciences, Kyoto Prize, Wolf Prize for Chemistry, Millennium Technology Prize, Priestley Medal (highest award of the American Chemical Society), Gairdner Prize, Hoover Medal, Dreyfus Prize in Chemical Sciences, BBVA Frontiers of Knowledge Award in Biomedicine, Balzan Prize, and the Dr. Paul Janssen Award. He holds 41 honorary doctorates, including Harvard, Yale, Columbia, and Northwestern, and has been elected to the National Academy of Medicine, the National Academy of Engineering, the National Academy of Sciences and the National Academy of Inventors.
Gaurav Sahay is Professor in the Department of Pharmaceutical Sciences and co-Director for the Center of Innovative Drug Delivery and Imaging (CIDDI), at the College of Pharmacy at Oregon State University. Dr. Sahay’s lab is developing novel nanotechnology-based platforms including lipid-based nanoparticles for effective delivery of messenger RNA therapeutics for treatment of cystic fibrosis, retinal degeneration and against SARS-CoV2. He has done pioneering work to dissect the intracellular transport essential for nucleic acid delivery to the cytosol and developed methods to overcome endosomal barriers. He has more than 60-peer-reviewed publications in top tier journals including Science Advances, Nature, Nature Communications, Nature Biotechnology, Nature Nanotechnology, Journal of Controlled Release, Nano Letters etc. He is the winner of a 2013 American Association of Pharmaceutical Scientists (AAPS) Postdoctoral Fellow Award, the 2015 Controlled Release Society (CRS) T. Nagai Award, a 2016 American Association of Colleges of Pharmacy (AACP) New Investigator Award, a 2019 Oregon Health & Sciences University (OHSU) Distinguished Faculty Senate Award for Collaboration, 2020 Phi Kappa Phi OSU Emerging Scholar Award and 2020 CMBE Young Innovator Award. He serves as the Principal Investigator on awards funded through the National Institutes of Health, Cystic Fibrosis Foundation and biotech companies. He serves as a consultant and scientific advisory board member to several biotech and venture capital firms. He is co-founder of Enterx Bio and RNAvax Bio. He was the Chair of the 2018 NanoMedicine and Drug Delivery Symposium (NanoDDS, Portland, OR) and is standing section member for Innovative in NanoSystems and Nanotechnology. Dr. Sahay completed his postdoctoral research with Prof. Robert Langer and Prof. Daniel Anderson at the Koch Institute for Integrative Cancer Research at MIT and received his Ph.D. from the University of Nebraska Medical Center under the mentorship of Prof. Alexander Kabanov.
Omar Abudayyeh is an Investigator at Brigham and Women’s Hospital and member of the faculty at Harvard Medical School where he directs a lab developing next-generation gene editing, gene delivery, and synthetic biology technologies and applies them towards new therapeutics and the study of aging. He previously was a McGovern Fellow at MIT where he directed his own research group and before that was at Harvard Medical School and MIT as a graduate student in Feng Zhang’s lab at the Broad Institute, where he earned a Ph.D. researching novel CRISPR enzymes for genome editing, therapeutics, and diagnostics. He is a pioneer in the gene editing space as an inventor on dozens of patents and patent applications relating to gene editing and diagnostic innovations, as well as over 30,000 citations on more than 40 peer-reviewed articles in journals like Nature, Science, and Cell. He is also co-founder of Sherlock Biosciences, Proof Diagnostics (acquired), and Tome Biosciences, which are commercializing CRISPR-based diagnostics for healthcare and at-home testing, as well as other stealth starts ups in the gene and RNA therapy space, which have collectively raised hundreds of millions. Dr. Abudayyeh has been recognized as Technology Review Innovators Under 35, Bloomberg New Economy Catalyst, Endpoints 20 under 40 Next Generation of Biotech Leaders, 2022 Termeer Scholar, 2018 Forbes 30 under 30, Business Insider 30 under 30, a 2018 TEDMED Hive honoree, and a 2013 Paul and Daisy Soros Fellow. Dr. Abudayyeh graduated from MIT in 2012 with a B.S. in mechanical engineering and biological engineering, where he was a Henry Ford II Scholar and a Barry M. Goldwater Scholar. He also spent two years studying towards an MD at Harvard Medical School.
Jonathan Gootenberg is a member of the faculty at Harvard Medical School and an investigator at Beth Israel Deaconess Medical Center. Dr. Gootenberg draws from fundamental biological insights to engineer new molecular tools, with a focus on methods that leverage programmability across biological scales. These technologies, including CRISPR-mediated genome and transcriptome perturbation and non-destructive cellular sensing, allow for unprecedented manipulation and profiling of cellular states in the body, and have myriad applications in basic science, diagnostics, and therapeutics. These technologies have been adopted by multiple biotechnology companies, several of which Dr. Gootenberg has co-founded. Previously, Dr. Gootenberg led an independent research group at MIT as a McGovern Fellow. He received his PhD in Systems Biology from Harvard University, conducting research with Aviv Regev and Feng Zhang at the McGovern Institute and Broad Institute of MIT and Harvard, and earned his bachelor’s degree in mathematics and biological engineering at MIT. He has been honored with awards such as Forbes 30 Under 30, Technology Review 35 Under 35, Endpoints 20 Under 40, and as a scholar of the Termeer Foundation.
Dr. Jinjun Shi is an Associate Professor at Harvard Medical School and a faculty member in the Center for Nanomedicine and Department of Anesthesiology, Perioperative and Pain Medicine at Brigham and Women’s Hospital. His laboratory has a broad interest in nanomedicine, drug delivery, RNA therapy, and immunotherapy for transformative biomedical applications. Notably, the immuno-nanotherapeutic technology developed by him and colleagues has led to several clinical trials by Selecta Biosciences. His research is currently focusing on i) RNAi delivery for gene silencing; ii) mRNA delivery for protein restoration; iii) immuno-nanotherapy; and iv) development of stimuli-responsive biomaterials. He is an elected Fellow of the American Institute of Medical and Biological Engineering (AIMBE) and a Clarivate’s Highly Cited Researcher.Dr. Jinjun Shi is an Associate Professor at Harvard Medical School and a faculty member in the Center for Nanomedicine and Department of Anesthesiology, Perioperative and Pain Medicine at Brigham and Women’s Hospital. His laboratory has a broad interest in nanomedicine, drug delivery, RNA therapy, and immunotherapy for transformative biomedical applications. Notably, the immuno-nanotherapeutic technology developed by him and colleagues has led to several clinical trials by Selecta Biosciences. His research is currently focusing on i) RNAi delivery for gene silencing; ii) mRNA delivery for protein restoration; iii) immuno-nanotherapy; and iv) development of stimuli-responsive biomaterials. He is an elected Fellow of the American Institute of Medical and Biological Engineering (AIMBE) and a Clarivate’s Highly Cited Researcher.
Dr. Afonin obtained his M.S. in Chemistry from Saint Petersburg State University, followed by a Ph.D. in Photochemistry from Bowling Green State University, Ohio. Additionally, he acquired a Graduate Certificate in Bioinformatics, Proteomics/Genomics. Over the subsequent three years, he conducted his Postdoctoral Fellowship in Biochemistry at the University of California Santa Barbara. In 2011, he was invited as a Research Fellow at the National Cancer Institute, NIH, where he played a pivotal role in establishing and overseeing an experimental branch within the Computational RNA Structure Group. He started his tenure-track appointment at UNC Charlotte in 2015 and achieved promotion with permanent tenure to the rank of Associate Professor in 2019 and then was further promoted to Full Professor in 2021. Dr. Afonin currently serves as a founding council member and vice-president of International Society of RNA Nanotechnology and Nanomedicine (). He is an author of over 100 research publications and his accolades include two NIH Fellows Awards for Research Excellence (FARE), a prestigious NIH Maximizing Investigators’ Research Award (MIRA R35), the First Citizens Scholars Medal, and several substantial grants from the NIH and NSF.
Jin Billy Li, Ph.D., is a Professor of Genetics at Stanford University. He earned his bachelor’s and master’s degrees at Tsinghua University in Beijing, China, and completed his Ph.D. at Washington University in St. Louis. Following postdoctoral training at Harvard Medical School, he established his laboratory at Stanford. Dr. Li’s research focuses on the study of RNA editing mediated by ADAR enzymes. Together with collaborators, his team unveiled a significant biological role of RNA editing in evading dsRNA-mediated autoimmunity. This discovery has paved the way for innovative approaches to cancer and autoimmune disease treatment. Additionally, they played a key role in co-developing site-directed RNA base editing, utilizing endogenous ADAR enzymes. This breakthrough overcomes challenges associated with CRISPR/Cas-based DNA editing, holding immense promise for addressing both rare and common diseases.
Prof. Kazunori Kataoka is a Professor Emeritus at the University of Tokyo and the Founding Center Director of the Innovation Center of NanoMedicine (iCONM), Kawasaki Institute of Industrial Promotion. Over the past 40 years, Prof. Kataoka has made significant contributions to the fields of biomaterials, particularly in drug delivery/drug targeting, non-viral gene delivery, and nanomedicine. He has published over 600 peer-reviewed articles with over 100,000 citations (h-index of 165). He has been certified as a Highly Cited Researcher (Clarivate Analytics) for the past eight years since 2016. He also has over 600 issued patents and founded five start-ups. Awards include the Clemson Award (the Society for Biomaterials) (2004), the Founder’s Award (Controlled Release Society) (2006), the NIMS Award (National Insitute of Materials Science, Japan) (2009), the Humboldt Research Award (2012), the Leo Esaki Prize (2012), Gutenberg Research Award (2015), Princess Takamatsu Cancer Research Fund Prize (2017), Biomaterials Global Impact Award (2023), and Clarivate Citation Laureate in Chemistry (2023). He has been elected as a Member of the Engineering Academy of Japan (2011), an International Member of the US National Academy of Engineering (2017), and a Fellow of the US National Academy of Inventors (2017).
Daniel G. Anderson is the Joseph R. Mares (1924) Professor of Chemical Engineering, a core member of the Institute for Medical Engineering and Science, and an intramural member of the Koch Institute for Integrative Cancer Research. He is a leading researcher in the fields of nanotherapeutics and biomaterials. His work has led to advances in a range of areas, including medical devices, cell therapy, drug delivery, gene therapy and material science, and has resulted in the publication of more than 500 papers, patents, and patent applications. He has founded several companies, including Living Proof, Olivo Labs, Crispr Therapeutics (CRSP), Sigilon Therapeutics, Verseau Therapeutics, oRNA, VasoRx, and Souffle Therapeutics. He is a member of National Academy of Medicine, the Harvard-MIT Division of Health Science and Technology, a fellow of the National Academy of Inventors, and is an affiliate of the Broad and Ragon Institutes.
Dr. Le Cong is an Assistant Professor in the Pathology and Genetics departments at Stanford University School of Medicine, and member of Bio-X, Wu-Tsai Neuroscience, and Cancer Institutes. His research group is developing technology for large-scale genome editing and single-cell perturbation-profiling, integrating advance from metagenomics, computational biology, and machine learning. His past work has led to one of the first FDA-approved clinical trials using CRISPR for in vivo gene therapy. More recently, his group invented tools for cleavage-free large gene insertion with novel recombination proteins, and developed single-cell tracking approach for studying cancer immunology, enabled by deep learning. Dr. Cong is a recipient of the NHGRI Genomic Innovator Award, Baxter Foundation Faculty Scholar, Genetic Engineering and Biotechnology News (GEN) Top 10 Under 40, Clinical OMICs Pioneers Under 40, and Clarivate Web of Science Highly Cited Researcher.
Mark A. Kay, MD, PhD, is the Dennis Farrey Family Professor in the Departments of Pediatrics and Genetics, and Head of the Division of Human Gene Therapy in Pediatrics at the Stanford University School of Medicine. Professor Kay received his MD-PhD at Case Western Reserve University and completed a residency in pediatrics, fellowship in medical genetics and inborn errors of metabolism, and post-doctoral research at Baylor College of Medicine. Dr. Kay was an assistant/associate professor at the University of Washington in the Department of Medicine from 1993-1998 before moving to Stanford. Dr. Kay’s group has published over 275 papers in leading journals. Dr. Kay is most well-known for his contributions in the field of gene-based therapeutics and non-coding RNA biology specifically related to microRNA biogenesis and tRNA derived small RNAs. Dr. Kay is one of the founders of the American Society of Gene and Cell Therapy and served as the President in 2005-2006 and received the society’s outstanding investigator award in 2013. In 2021, he was elected to the National Academy of Inventors. He spends much of his spare time doing landscape and nature photography.
The Promise and Progress of RNA Delivery & Gene Editing
March 20, 2024 – Online Conference
and Progress of
& Gene Editing
March 20, 2024
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Reserve Your Spot
- Engaging presentations from over ten distinguished experts in the fields of RNA delivery and in vivo gene editing
- An exclusive fireside chat with Robert Langer, co-founder of Moderna, and Jennifer Doudna, Nobel Prize-winning co-inventor of CRISPR technology
- Poster presentations from emerging postdoctoral scholars
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Snyder Lab Research Studies