Unlocking the Potential of RNA: New Paths for Targeting RNA
This webinar is Day 3 of the "Illuminating New Frontiers-Cracking the Undruggable Code"
This session delves into the innovative realm of RNA as a key therapeutic target in modern drug discovery.
Illuminating New Frontiers-Cracking the Undruggable Code
“Illuminating New Frontiers: Cracking the Undruggable Code" invites you on a groundbreaking journey across the evolving landscape of drug discovery. This three-day virtual event unites trailblazers in science and industry to explore how innovative approaches are reshaping our ability to tackle 'undruggable' targets. From the complexities of the RAS pathway and the intricacies of protein-protein interactions to the pioneering fields of targeted protein degradation/induced proximity and RNA targeting, each day illuminates a different frontier of therapeutic potential.
We stand at the cusp of a new era, where challenges become opportunities for breakthroughs that extend beyond conventional boundaries. This event is a testament to the power of collaboration and innovation in unveiling new therapeutic strategies, offering hope for untreatable conditions. Join us as we navigate the cutting-edge of science, inspiring a future where every target is within our reach, and no patient is left without options.
Day 3 - Unlocking the Potential of RNA: New Paths for Targeting RNA
In the realm of drug discovery, certain targets have long been deemed "undruggable" due to their complex nature and the technical challenges associated with modulating them therapeutically. Among these, the RAS pathway stands out as an important element in cell signaling implicated in numerous cancers and diseases. Historically deemed undruggable due to its complex biochemistry and lack of suitable binding sites, RAS has recently witnessed a paradigm shift. Recent innovations in molecular biology—coupled with breakthroughs in drug design and technology—have challenged the notion that the RAS pathway is undruggable, leading to the development and market approval of RAS-targeting therapeutics. Similarly, protein-protein interactions (PPIs)—key elements of RAS signaling—represent untapped therapeutic potential, offering avenues to intervene in disease processes in ways previously deemed infeasible.
In this session, we will explore the latest research targeting the RAS/MEK/ERK signaling pathway and learn about novel approaches being developed to manipulate and measure PPIs within RAS and other signaling networks.
View other sessions of the series
Day 1: Rethinking Undruggable Targets: New Insights into RAS and Protein Interactions
Day 2: Next-Generation Therapeutics: The Power of Induced Proximity and Targeted Protein Degradation
Speakers
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Matt Disney, PhD
Institute Professor and Chair of the Department of Chemistry
The Herbert Wertheim UF Scripps Institute for Biomedical Innovation and Technology
Presentation Title: Sequence-Based Design of Small Molecules Targeting RNA Structure
Matthew Disney is a native of Baltimore, Maryland. He received his early schooling in the Baltimore Catholic School System, his B.S. from the University of Maryland, and his Ph.D. from the University of Rochester in Physical Chemistry
Matt is an Institute Professor and Chair of the Department of Chemistry at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation and Technology (formerly Scripps Florida).
His laboratory has pioneered the development of small molecules targeting RNA, particularly by answering fundamental questions surrounding the molecular recognition of RNA folds by small molecules. The resulting data have informed the design of chemical probes to study problems of biomedical importance and to advance a gene-to-RNA-to precision medicine paradigm. Applications include development of sequence-based design of small molecules from genome sequence, using a disease-affected cell to manufacture its own medicine on-site, understanding the biology of coding and non-coding RNAs, and interfacing disease-causing RNAs with quality control machinery to edit or eliminate them from cells.
The lab’s research has garnered various awards including the ACS Nobel Laureate Signature Award for Graduate Education in Chemistry (with Alicia Angelbello), the Scripps Florida Mentor of the Year, the Sackler Prize in the Physical Sciences, Barry Cohen Award in Medicinal Chemistry, NIH Director’s Pioneer Award, the Tetrahedron Young Investigator Award, the Eli Lily Award in Biological Chemistry, the David W. Robertson Award in Medicinal Chemistry, among others.
Importantly, the laboratory’s work has spurred small to large pharmaceutical companies to invest in small molecule targeting of RNA. Matt has founded several biotech companies including Expansion Therapeutics (scientific headquarters in Jupiter, FL), which raised >$150M, and a new company that is currently in stealth mode.
Email: mdisney@ufl.edu
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Amanda Garner, PhD
Professor in the Department of Medicinal Chemistry and Director of the Interdepartmental Program in Medicinal Chemistry
University of Michigan
Presentation Title: Enabling Technologies for Revealing Druggable Paths in RNA Biology
Amanda L. Garner is a Professor in the Department of Medicinal Chemistry and Director of the Interdepartmental Program in Medicinal Chemistry at the University of Michigan. She received her Ph.D. in Chemistry from the University of Pittsburgh and completed NIH-funded postdoctoral studies at The Scripps Research Institute. Dr. Garner began her independent career in 2013 at the University of Michigan and her research integrates chemical biology, medicinal chemistry, and molecular and cellular biology approaches for early-stage drug discovery efforts with a primary focus on validating new therapeutic targets in RNA biology. For her research accomplishments, she was awarded Catalyst Awards from the Dr. Ralph and Marian Falk Medical Research Trust in 2016 and 2022, named a Rogel Scholar from the Rogel Cancer Center in 2022, awarded the David W. Robertson Award from the ACS Division of Medicinal Chemistry in 2022 and Ono Pharma Breakthrough Science Initiative Award in 2023.
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John S. Schneekloth, Jr. (Jay), PhD
Senior Investigator and head of the Chemical Genetics
National Cancer Institute, Frederick, MD
Presentation Title: Combining Structure and Design to Improve the Properties of RNA-Binding Small Molecules
Jay Schneekloth is a Senior Investigator and head of the Chemical Genetics Section in the Chemical Biology Laboratory at the National Cancer Institute in Frederick, MD. He earned his A.B. from Dartmouth College in 2001 where he worked for Gordon W. Gribble on the total synthesis of bioaccumulating halogenated natural products. He then moved to Yale University where received his Ph.D. under the direction of Prof. Craig M. Crews, where he designed the first cell-permeable protac molecules. He then pursued an NIH postdoctoral fellowship with Erik Sorensen at Princeton University, where he designed cascade reactions applied to the total synthesis of analgesic alkaloid natural products. In 2011, Dr. Schneekloth was recruited to the National Cancer Institute. At the NCI, his research focuses on understanding nucleic acids as targets for small molecules, particularly in the area of cancer therapeutics. His work encompasses development of high throughput screening techniques, chemical biology probe design development, biophysical characterization of target-ligand interactions, target validation, and studying the structure and conformational dynamics of nucleic acids. Dr. Schneekloth is currently the Chair of NCI’s Medicinal Chemistry Accelerator, a program aimed toward translational development of novel biologically active small molecules discovered within the NCI.
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Brad Swanson, PhD
Strategic Collaborations Manager
Promega Corporation
Presentation Title: Suite of Assays to Enable RNA-Targeted Drug Discovery
Brad Swanson is a Strategic Collaborations Manager at Promega Corporation, bringing with him over 30 years of experience in the fields of Cellular and Molecular Biology, Stem Cell Biology, and Immunology. Brad works with the R&D and business teams to connect scientists with Promega products, technologies, and services with an emphasis on Promega’s cellular Drug Discovery products for large and small molecule drug discovery.
Prior to joining Promega, Brad was Vice President, Life Science R&D and Strategic R&D Programs at Cellular Dynamics International (CDI), where he led the life science R&D operations from start-up through IPO and eventual acquisition by Fujifilm. Before CDI, Brad was a Senior Scientist at both Roche-Nimblegen and at Promega.
Brad performed his postdoctoral research in the laboratory of Phillipa Marrack at the National Jewish Medical and Research Center as a Howard Hughes Medical Institute postdoctoral research fellow. This work focused on understanding the molecular events regulating memory T cell homing and differentiation. Brad received his PhD in Cellular and Molecular Biology from the University of Wisconsin-Madison in 1998 and graduated with a BS degree from Iowa State University in 1991.