TUESDAY, JUNE 18
6:40 - 7:30 PM
3D Cellular Models
Human 3D Organ Models for Target Discovery and Drug Screening
Moderators: Hansjoerg Keller, PhD, Senior Investigator I, Musculoskeletal, Novartis Institutes for BioMedical Research
John Lowman, Biomedical Engineer and Business Developer, Mimetas
- 50 Ways to leave your lover 2D cell culture, just start functional 3D cell models
- I can see clearly now; visualization and imaging challenges of 3D microtissues
- ‘It’s the economy and throughput, stupid!’ - what hinders 3D models in drug screening
Developing Organs-on-a-chip/Microphysiological Systems for Drug Discovery
Moderator: Kambez H. Benam, PhD, Assistant Professor, Division of Pulmonary Sciences and Critical Care Medicine, Departments of Medicine & Bioengineering, University of Colorado
- How can we scale?
- Moving towards ‘Human Body-on-a-Chip’
- Building additional organ-level micro-complexities? Hoe fare should we go and what are key determinants
Preclinical Strategies, Models & Tools in Oncology
Imaging in Drug Development
Charles Glaus, PhD, Director, Translational Research & Early Clinical, Takeda
- Emerging technologies for tissue analysis
- Advanced preclinical models for IO research
- Leveraging blood samples vs tissue biopsies
- Shortcomings of preclinical models for CAR-T drug development
Chemical Biology and Target Validation
Recent Advances in Proteomics to Enable Target Discovery and Mechanism of Action Studies
Moderator: An Chi, PhD, Director, Chemical Biology Proteomics, Merck
- Recent development/trends of chemoproteomics strategies for target ID to follow up on hits from phenotypic screens
- Effective strategies to prioritize target hypothesis
- Omics use for MOA/hit prioritization in phenotypic screening campaigns today
- Best practice in assessing translatability of complex disease models
Chemical Probes as Tools for Target Validation
Moderator: Milka Kostic, PhD, Program Director, Chemical Biology, Department of Cancer Biology, Dana-Farber Cancer Institute
- What are the common strategies for validating chemical probes?
- How can we, as a community, most effectively share best practices for chemical probe use?
- What are different mechanisms for unrestricted sharing of high-quality chemical probes?
- How can we talk about the importance of chemical probes to the bottom-line?
CNS Targets and Translational Strategies
Lost in Translation? Asking the Right Questions in the Right Language
Moderator: Dario Doller, PhD, Sage Therapeutics
- CNS disease etiology understanding: Who owns it?
- What are the major disconnects between lab experimentation and clinical research?
- Are differences in physiology between preclinical species and human affecting the translational gap?
- Patient segmentation: Strategies and outcomes
- Correlation or causation? Importance of genetic links for different CNS diseases
- What are the attributes that have real impact minimizing risk during novel target selection?
- Human as a model for human – What is the future of experimental medicine in CNS drug discovery?
Dynamic Collaborations between Industry and Patient Advocacy Organizations
Moderator: Steven Roberds, PhD, CSO, Tuberous Sclerosis Alliance
- Examples of mutual beneficial collaborations
- Patient-focused drug development
- Accelerating clinical trials through early and frequent engagement
Optimizing Drug Metabolism & Pharmacokinetics
Key Issues Related to Drug Transporters in a Pharma R&D Setting
Moderators: Li Di, PhD, Research Fellow, Pharmacokinetics, Dynamics and Metabolism, Pfizer, Inc.
Manthena Varma, PhD, Associate Research Fellow, PDM, Medicine Design, Worldwide R&D, Pfizer, Inc.
- How can we best generate reliable in vitro transport kinetics and inhibition data?
- Many transporters do have overlapping substrate-specificity, how can we assess and quantify an individual transporters' contribution?
- Regulation of transporters (induction, disease, epigenetics) can significantly modulate overall transport capacity. How can this be integrated into current in vitro - in vivo extrapolations?
Use of Modeling Tools and Strategies for Predicting ADME-Tox Properties
Moderator to be announced
- Machine-learning or structure-based approaches for ADME-Tox prediction and optimization?
- Should the modeling tools for toxicity predicting be specific for xenobiotics and drugs?
- Quantum-mechanics methods for drug metabolism prediction
Lead Optimization for Biologics and Combination Therapies
Moderators: Alison Betts, PhD, Associate Research Fellow, Translational Modeling & Simulation, Biomedicine Design, Pfizer Worldwide R&D
Renu Singh Dhanikula, PhD, Senior Research Investigator, Metabolism and Pharmacokinetics, Bristol-Myers
- When are relevant assays and models for PKPD testing and/or safety pharmacology studies for biologics?
- What are some emerging safety concerns for biologics and what are potential strategies to de-risk them?
- Where are there gaps in understanding preclinical to clinical safety translation for biologics?
Immuno-Oncology Biomarkers 1: Predictive Biomarkers and Companion Diagnostics
Clinical and Translational Biomarkers
Digital Health: Pharmaceutical Executive Summit
Immuno-Oncology Clinical Trials Strategy and Execution
Immuno-Oncology Drug Development
Moderator: Jonathan Cheng, MD, Vice President & Oncology Therapeutic Area Head, Merck
- How to move quickly to register a drug
- How to utilize biomarkers in clinical trials
- How to choose which novel combinations to pursue
Optimizing for Value in the Age of Precision Medicine: Complications and Opportunities
Moderator: Nikolas T. Weissmueller, PhD, Associate Director, Strategic Options & Assessment, Business Insights & Analytics, R&D, Bristol-Myers Squibb
- Value-based IO development
- Probability of success assessment
- Use of computational methods to inform trial design choices
Benefit-Risk Evaluation of Immunotherapies Today: A Discussion of EMA/FDA Guidance for Industry
Moderator: Céline Adessi, PhD, Senior Group Director, Product Development, Safety Science Oncology, Licensing and Early Development, F. Hoffmann-La Roche
- Modernizing the reporting, collection, assessment of adverse events
- Patient reporting outcome
- Benefit-risk balance evaluation
Combination Cancer Immunotherapy
THURSDAY, JUNE 20
7:15 - 8:10 AM
iPS Cells for Disease Modeling and Drug Discovery
iPSC-Based Disease Modeling
Moderator: Colin Choi, PhD, Scientist, Drug Safety Research and Evaluation, Takeda
- Utilizing patient-derived iPSCs to study disease pathogenesis and develop therapeutics in relevant genetic and cellular context
- Can human iPSCs systems replace animal models? How close are we (in lab and regulatory agencies)?
- What are the current challenges or weaknesses for adopting iPSCs-derived in vitro systems in discovery and toxicology?
Infrastructure and Strategy for iPS Cell Based Drug Discovery
Moderator: Gabriele Proetzel, PhD, Director, Regenerative Medicine, Takeda Pharmaceuticals, Inc.
- Technology advances
- Phenotypic screenings applications
- Target validation studies
Patient-Derived Tumor Organoids
Deploying Bioengineered 3D Tumor Models/Organoids into Clinical Practice And The Pharmaceutical Pipeline
Moderator: Aleksander Skardal, PhD, Assistant Professor, Wake Forest Institute for Regenerative Medicine
- Where is the clinical enterprise and/or pharma in terms of seeing value in these model systems?
- How do we convince clinicians and more traditional scientists that these new technologies are important and effective?
- Complexity (more cell types or multiple organoids per model system) versus scale-up/high throughput
Developing Patient-Derived Tumoroids
Moderator: Alejandro Amador, PhD, GSK, Scientific Leader, Platform Biology Automation, GSK
- Evaluating tumor organoid protocols
- Characterizing growth characteristics
- Improving establishing success rates
Target Identification & Phenotypic Screening
Exploiting CRISPR for In vivo Screening: What You Need to Know Before and After
Moderators: Danilo Maddalo, PhD, Lab Head, ONC Pharmacology, Novartis Institutes for BioMedical Research, Novartis Pharma AG
- Overview of current strategies for in vivo screening for target identification
- Optimization and validation of current models
- Understanding inherent limitations and need for using complementary techniques
- Analyzing and interpreting the data
Exploring Artificial Intelligence/Machine Learning for Improving Drug Discovery
Moderators: Arvind Rao, PhD, Associate Professor, Department of Computational Medicine and Bioinformatics, University of Michigan
Deepak K. Rajpal, Senior Scientific Director, Computational Biology, GSK R&D
Johannes M. Freudenberg, Senior
Scientific Investigator, Computational Biology, GSK R&D
- In silico modeling of complex cellular phenotypes and disease models: paradigms to bridge the computational-experimental gap
- Deconvolution of preclinical data and translating into clinical space: integrative modeling, generalizability, transportability and data harmonization
- Understanding limitations and where the opportunities lie: the power of scale and the complexity of medicine
Image-Based Approaches to Single-Cell 'Omics
Moderator: Jeffrey Moffitt, PhD, Assistant Professor, Program in Cellular and Molecular Medicine, Boston Children's Hospital, Department of Microbiology, Harvard Medical School
- State-of-the-art methods for massively multiplexed protein and RNA imaging
- Construction of spatially resolved cellular atlases and their potential in basic and clinical science
- Image-based approaches to high-throughput screening of pooled libraries of genetic perturbations
Project Human BBB Penetration from Preclinical Studies
Moderator: Guangqing Xiao, PhD, Associate Director, DMPK, Takeda
- In vitro and in vivo models to assess brain penetration
- Study design
Image-Guided Opening of the Blood Brain Barrier and Intra-Arterial Drug Delivery
Moderator: Piotr Walczak, MD, PhD, Associate Professor, Radiology, Johns Hopkins University
- Techniques for blood brain barrier opening (osmotic vs. FUS)
- Advantages of Intraarterial route for targeting the brain
- Need for image-guidance of BBB manipulation and drug targeting
Brain Regional Drug Disposition in CNS Diseases and Drug Development
Moderator: Irena Loryan, PhD, Researcher, Department of Pharmaceutical Biosciences, Translational PKPD, Uppsala University
- Brain regional drug disposition
- State of regional blood-brain barrier transport in Alzheimer’s disease
- How to address brain regional drug disposition in CNS drug development?
Predicting Drug Toxicity
Gaps in Translating Preclinical Findings to the Clinic
Moderators: William Proctor, PhD, DABT, Associate Director/Senior Scientist, Investigative Toxicology, Department of Safety Assessment, Genentech Inc.
Terry Van Vleet, PhD, DABT, Head of Molecular and Computational Toxicology, Department of Preclinical
- Model/assay sensitivity – what level (or magnitude) of change can we detect?
- Do we have appropriate study design, data analysis, statistical analysis, and statistical power?
- How predictive are our preclinical findings/models to the clinic?
- What is it we are missing? Are there other useful models? Are their gaps in our translation knowledge?
Using iPSC for Drug Safety Screening
Moderators: Gary Gintant, PhD, Senior Research Fellow, AbbVie
Paul W. Burridge, PhD, Assistant Professor, Department of Pharmacology, Center for Pharmacogenomics, Northwestern University Feinberg School of Medicine
- What are concerns for using human induced pluripotent stem cell (iPSC)-derived cells to assess drug safety screening?
- How to consider standardizing assays and comparing results? Assay reproducibility?
- Generation of patient derived hiPSC-derived cardiomyocytes: present and future roles.
- The role of newer co-cultures and 3D structures in present and future efficacy and safety studies.
- How to best adapt hiPSCs for high-throughput phenotypic screening.
Use of 3D and Microphysiological Systems for Drug Safety Assessments
Moderators: Volker Lauschke, PhD, MBA, Associate Professor, Group Leader in Personalized Medicine and Drug Development, Department of Physiology and Pharmacology, Karolinska Institutet
Bo Yeon Lee, PhD, Scientific Program Manager, Office of the Director,
Tissue Chip for Drug Screening Program, National Center for Advancing Translational Sciences, National Institutes of Health
Sophie Lelievre, DVM, PhD, LLM, Professor, Cancer Pharmacology, Purdue University College of Veterinary Medicine
- Update on benchmarking studies covering emerging cell models and 3D platforms
- How to best integrate on-a-chip devices with other toxicity studies
- Speed, accuracy and reliability of tissue-on-a-chip systems
- How to utilize these systems for predicting toxicity, efficacy and designing clinical trials
Immuno-Oncology Biomarkers 2: Immune Profiling and Immune Monitoring
Digital Biomarkers: Biosensors, Wearables, and mHealth
Small Molecules for Immuno-Oncology Therapeutics
Why is STING so IntereSTING as an IO Target?
Moderator: Gottfried Schroeder, PhD, Senior Scientist, Department of Pharmacology, Merck Research Labs Boston
- Distinct structural features of STING to design modulators against
- Biologics v. small molecule approaches for modulating STING
- MOA and physiological considerations, possible side effects, etc.
Targeting the Tumor Microenvironment
Moderator: Earp, PhD, Director, University of North Carolina Lineberger Cancer Center and Co-Founder, Meryx Pharmaceuticals
- Types of Tumor-associated Macrophages (TAMs) in the TME
- Promising intracellular kinase targets in TAMs
- Other TAM targets