Agenda
The Quest for Innovative Molecular Treatment Modalities for Intractable Disease Targets
November 17-18, 2022 | 11:00am-4:00pm EST
Virtual Workshop
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AGENDA
DAY 1: TARGETS
November 17, 2022 - 11:00 AM – 4:00 PM EST
11:00-11:15 AM EST Welcome and Workshop Overview
11:00 AM: Workshop Welcome
Joni Rutter, PhD, Acting Director, National Center for Advancing Translational Sciences, National Institutes of Health
11:05 AM: Workshop Overview
Karlie Sharma, PhD, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health
11:15-12:00 PM EST Introduction to Day 1
Overcoming Long-Standing Therapeutic Challenges Using Emerging Technologies: The Development of Covalent Inhibitors of KRAS (G12C)
Brian Lanman, PhD, Director of Research, Amgen
12:00-1:15 PM EST Session 1: RNA
12:00 PM: Introduction to Session
12:05 – 12:45 PM: Ten Minute Talks
Design of Small Molecules Targeting RNA Structure
Chair: Matthew Disney, PhD, Chair, Department of Chemistry, University of Florida Scripps Biomedical Research
RNAs as Drug Targets: The Uniqueness of the Ribosome
Adegboyega Oyelere, PhD, Associate Professor, Georgia Institute of Technology
piRNAs as Regulators of Trypanosoma Cruzi Pathogenesis
Pius Nde, PhD, Assistant Professor, Meharry Medical College
Targeting Pre-mRNA Splicing to Discover and Develop Novel Small Molecule Therapeutics
Anu Bhattacharyya, PhD, Executive Director Biology, PTC Therapeutics
12:45 PM: Discussion
1:15 – 1:30 PM EST Break
1:30 – 2:45 PM EST Session 2: Protein Stability
1:30 PM: Introduction to Session
1:35 – 2:15 PM: Ten Minute Talks
How Prions Escape Drug Therapy
Chair: Surachai Supattapone, MD, PhD, Professor, Geisel School of Medicine, Dartmouth College
Molecular Structures Reveal Synergistic Rescue of Δ508 CFTR by Trikafta Modulators
Jue Chen, PhD, Professor, The Rockefeller University
Making the Undruggable Druggable: Molecular Simulation as Computational Microscopy for Target Detection in Disordered and Liquid-Like Assemblies
Gül Zerze, PhD, Assistant Professor, University of Houston
The Quest for Disease Modifying Treatments for Neurodegenerative Disorders
M. Maral Mouradian, MD, Director, RWJMS Institute for Neurological Therapeutics, Rutgers University
2:15 PM: Discussion
2:45 – 4:00 PM EST Session 3: Other Novel Targets
2:45 PM: Introduction to Session
2:50 – 3:30 PM: Ten Minute Talks
Pivotal Role of Cholesterol Rich Lipid Rafts in Organizing the Dynamics of Membrane Signaling Proteins
Chair: Tony Yaksh, PhD, Professor, Department of Anesthesiology, University of California San Diego
Targeting Golgi Defects for Treatment of Alzheimer’s Disease
Yanzhuang Wang, PhD, Professor, University of Michigan
Drug Screening for Keratinopathies and Porphyrias
Bishr Omary, MD, PhD, Professor of Medicine, Rutgers University
Patient-Specific Stem Cell Models for Intractable Mitochondrial Disorders
Shilpa iYer, PhD, Associate Professor, University of Arkansas
3:30 PM: Discussion
4:00 PM EST Day 1 Workshop Wrap-Up and Adjourn
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DAY 2: MODALITIES
November 18, 2022 - 11:00 AM – 4:00 PM EST
11:00 – 11:15 AM EST Welcome to Day 2
Karlie Sharma, PhD, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health
11:15 – 12:00 PM EST Introduction to Day 2
Site-Directed Discovery and Covalent Leads for Difficult Targets
Michelle Arkin, PhD, Department Chair and Professor, University of California San Francisco
12:00 – 1:15 PM EST Session 4: Repurposing Modalities
12:00 PM: Introduction to Session
12:05 – 12:45 PM: Ten Minute Talks
The New World of Induced Proximity
Chair: Derek Lowe, PhD, Director in Chemical Biology and Therapeutics, Novartis
The Next Frontiers for PROTAC® Protein Degraders
Ian Taylor, PhD, Chief Scientific Officer, Arvinas
Expansion of the Druggable Genome Through Molecular Glue
Mary Matyskiela, PhD, Vice President, Molecular Science at Neomorph
Deubiquitinase-Targeting Chimeras for Targeted Protein Stabilization
Lydia Boike, PhD, Scientist, Vicinitas Therapeutics
12:45 PM: Discussion
1:15 – 1:30 PM EST Break
1:30 – 2:45 PM EST Session 5: Novel Modalities
1:30 PM: Introduction to Session
1:35 – 12:15 PM: Ten Minute Talks
Novel Modalities: Hope and Hurdles
Chair: Melissa Junttila, PhD, Vice President, ORIC Pharmaceuticals
Rewriting Cellular Post-Translational Modifications for Challenging Targets
Christina Woo, PhD, Associate Professor, Harvard University
Cracking the Code of Macrocycle Drug Discovery
Christian Cunningham, PhD, Director and Principal Scientist, Genentech
Cystine-Dense-Peptides (CDP) as Therapeutic and Diagnostic Modalities
James Olson, MD, PhD, Professor, Fred Hutchinson Cancer Research Center
2:15 PM: Discussion
2:45 – 4:00 PM EST Session 6: Novel Drug Delivery
2:45 PM: Introduction to Session
2:50 – 3:30 PM: Ten Minute Talks
Novel Therapeutics and Delivery Mechanisms
Chair: Xandra Breakefield, PhD, Professor, Massachusetts General Hospital, Harvard Medical School
Novel Bioengineered Microbiome-Based Therapeutic Delivery for Neurodegenerative Diseases
Anu Kanthasamy, PhD, Director, Center for Brain Science and Neurodegenerative Disorders, University of Georgia
Non-Viral RNA and Genome Editing Delivery
Daniel Anderson, PhD, Professor, Koch Institute at MIT
Amplifying Targetable Surface Areas Through New Delivery Approaches
Juliane Nguyen, PhD, Associate Professor, University of North Carolina
3:30 PM: Discussion
4:00 PM EST Workshop Wrap-Up and Adjourn