团体讨论

Tuesday, June 19 & Thursday, June 21, 2018

This session features various discussion groups that are led by a moderator/s who ensures focused conversations around the key issues listed. Attendees choose to join a specific group and the small, informal setting facilitates sharing of ideas and active networking.

TUESDAY, JUNE 19 | beginning at 5:05 pm

ONCOLOGY & IMMUNO-ONCOLOGY

Preclinical Strategies for Combination Therapies

Mithun Khattar, PhD, Immuno-Oncology Lead, Cancer Pharmacology, Takeda Pharmaceuticals

  • Key factors to consider when selecting combination strategy
  • Immunomodulatory effects of small molecules
  • Translating preclinical models to the clinic - the expected and unexpected

VC Perspective on Immuno-Oncology

Leigh Zawel, PhD, Managing Director, MPM

Michael Gladstone, MD, Principal, Atlas Venture

  • Are more investments happening in the discovery of new IO targets or in strategies to enhance PD1/CTLA4 efficacy?
  • What critical data is coming in the next 12 months in the cancer vaccine and/or cell therapy space?
  • For your firm to consider an investment in an IO asset, what are the "must haves" in a data package?
  • Are there translational tools that are truly predictive of what kind of patients are most likely to respond to a given non-PD1 drug to predict (or at least narrow) populations to focus on in Ph1? Or are we going to have to continue to rely on empirical exploration large All-comer Ph1 studies?

Preclinical CAR T Cell Development Challenges in Solid Tumors

Matthew M. Hewitt, PhD, Principal Scientist, Tumor Biology & Director, Tumor Immunology/Microenvironment, Research & Development, Bellicum Pharmaceuticals, Inc.

  • Discuss the current assays available and their relevance
  • Translatability of in vitro and in vivo systems to predict efficacy/safety
  • Thoughts on how preclinical data translates to the clinic

Tumoroid/Microenvironment Interactions: Possibilities and Limitations

Anna L. Means, PhD, Associate Professor, Department of Surgery, Vanderbilt University Medical Center


SCREENING TOOLS & TECHNOLOGIES

Modeling Neurodegenerative Disorders for Drug Discovery and Development

Bilada Bilican, PhD, Investigator II, Neuroscience, Novartis Institutes for BioMedical Research(NIBR)

  • In vitro correlates of complex neurodegenerative diseases
  • How to model apparently sporadic neurodegenerative disorders
  • Advanced cellular models - how to address cell-autonomous vs. non-cell autonomous mechanisms of neurodegeneration
  • Phenotype- vs. target-based drug screening

Infrastructure and Strategy for iPS Cell-Based Drug Discovery

Asano Asami-Odaka, PhD, Associate Director, Ikeya Project, T-CiRA, Regenerative Medicine Unit, Takeda Pharmaceutical Company Limited

Chee Yeun Chung, PhD, Scientific Co-Founder and Associate Director, Discovery Biology, Yumanity Therapeutics

  • Technology advances
  • Phenotypic screenings applications
  • Target validation studies

MEDICINAL CHEMISTRY

Compound Collections for Phenotypic Screening and Chemical Biology

Ivan Cornella Taracido, PhD, Director, Chemical Biology, Merck Research Laboratories

  • How many and how large such collections should be?
  • Fit-for-phenotype and pathway sets, general MoA-annotated screening decks, diversity decks and the use of fragments and possibly other modalities (aptamers, etc.)
  • Screening modalities (fragments, small molecules, natural products, aptamers, peptides, proteins, RNAi and genome editing reagents)

Developments in Methodologies to Enable Target Identification

Ivan Cornella Taracido, PhD, Director, Chemical Biology, Merck Research Laboratories

  • What is new in chemical building blocks, reagents and reactions to enable omics and target ID?
  • Photo-affinity ligation and activity-based probe workflows
  • Advances in chemical genomics in mammalian haploid systems
  • In silico off-target prediction and other computational tools
  • Advances in analytical sciences (MS-based methods, Imaging, Bio-NMR, etc.)

Developing Anticancer Therapies with Dual Specific Kinases

Lijun Sun, PhD, Director, Center for Drug Discovery and Translational Research, Beth Israel Deaconess Medical Center, Harvard Medical School

  • Dual specific kinases and their benefits in treating cancers
  • Other therapeutic areas of interest?
  • Potential uses of the DYRK

Safety Considerations of Kinase Inhibitors

Samit, Bhattacharya, PhD, Senior Principal Scientist, World Wide Medicinal

  • Chronic safety concerns for kinase inhibitors for you in non-oncology indications
  • Advancing chemotypes
  • Using orthogonal profiles to assess safety

CNS

The Suitability of Animal Models for Preclinical Studies

Caghan Kizil, PhD, Helmholtz Young Investigator Group Leader, German Center for Neurodegenerative Diseases (DZNE) within Helmholtz Association

  • What type of animal/experimental models are used for neurodegenerative disease models?
  • What limitations are there for experimental models?
  • How well can we recapitulate the diseases in experimental animal models?
  • How can humanized models be improved?
  • What is the future of research for CNS disease models?

Identifying Cerebrovascular Links to Neurodevelopmental Disorders

Baptiste Lacoste, PhD, Assistant Professor, Cellular and Molecular Medicine/Neuroscience Program, The University of Ottawa/Ottawa Hospital Research Institute

  • What are the biggest challenges in identifying cerebrovascular links to neurodevelopmental disorders?
  • What are the most promising directions for this research?
  • How can this information be used to further development of drugs that pass the BBB?

DRUG METABOLISM & TOXICITY

Key Issues Related to Drug Transporters in a Pharma R&D Setting

Li Di, PhD, Research Fellow, Pharmacokinetics, Dynamics and Metabolism, Pfizer

  • 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?

VESICLES & NANOPARTICLES

Using Big Data to Help Nanotechnology

Cory Sago, PhD Student, James Dahlman Lab, Georgia Institute of Technoloy / Emory Medical School

  • DNA barcodes can be used to track more than 100 nanoparticles in a single mouse.
  • Big data analytics can be applied to understand how nanoparticle traits affect delivery in vivo.
  • The biology of drug delivery can be studied using DNA barcodes.

Nanoparticle Fabrication

Ghazal Hariri, PhD, Senior Scientist, Pfizer Global Research and Development

  • Challenges in nanoparticle fabrication and scale up for pharmaceutical production
  • Opportunities and applications for nanoparticle technology in the pharmaceutical industry
  • Impact of nanoparticle fabrication on drug development

THURSDAY, JUNE 21 | beginning at 9:40 am

ONCOLOGY & IMMUNO-ONCOLOGY

Emerging Targets in Immuno-Oncology

Quamrul Hassan, PhD, Group Leader, Molecular and Cellular Pharmacology, EMD Serono

  • Advancement in technology to validate targets in immuno-oncology
  • Chemo-genomic approach in target validation
  • Modality of inhibition in selecting targets: small molecules vs Biologics
  • Phenotypic Screen to target generation and validation

Modeling Innate and Adaptive Immunity in Murine Models

Marcus Bosenberg, MD, PhD, Associate Professor of Dermatology and Pathology, Yale University, Co-Leader, Genomics, Genetics and Epigenetics Program, Yale Cancer Center

  • Can animal models of cancer immunology predict human responses?
  • How should preclinical models be used to guide drug development in immunology?
  • How can existing models be improved?

Emergence of Microfluidic Models for Preclinical Assessment of Efficacy of Immune Checkpoint Inhibitors

Jeff Borenstein, PhD, Laboratory Technical Staff, Biomedical Microsystems, Draper

  • Immune checkpoint inhibitors exhibit variable patient response rates for reasons that are not completely clear. What attributes of an in vitro model are seen as most critical for establishing it as a tool to study mechanisms of responder/non-responder behavior?
  • Microfluidic model systems may find use as tools for drug development and ultimately for precision medicine applications, where patient response could be predicted before administering therapy. Where are the most significant opportunities for these systems, and would the attributes models for each respective application be different?
  • May existing in vitro cancer models are cell culture systems, where cancer cells are cultured in a 3D microenvironment. Emerging systems are focusing on tumor spheroids and on unmodified tumor fragments obtained from patient (or animal) biopsy. What are the principal advantages and disadvantages of each type of model?

SCREENING TOOLS & TECHNOLOGIES

The Use of 3D Cellular Models, Organ-on-a-Chip Technologies, or Microphysiological Systems in Preclinical Evaluation of Pharmaceuticals

William Daly, PhD, Managing Director & Faculty Scientist, Orthopedics and Rehabilitation, University of Wisconsin - Madison

  • What are the key barriers to adoption of 3D cellular models in preclinical workflows?
  • What are pharmaceutical wish lists (biomarkers, function, histology, etc.) for validation of 3D cellular models (groups to split into organ of choice)?
  • Where is the ideal fit for 3D cellular models in the drug development pipeline/pharmaceutical testing?
  • What are the missing models/unmet needs for preclinical evaluation and efficacy testing?
  • Are single organs enough or is there a strong need for multi-organ testing?

3D Models of Human Organs: Challenges and Prospects

Samira Musah, PhD, Dean's Postdoctoral Fellow, Wyss Institute at Harvard University and Harvard Medical School

  • Inducing stem cell differentiation, cell maturation, and functionality
  • Organoids, organs-on-chips, 3D-bioprinted tissues
  • Applications in disease modeling and therapeutic discovery

CRISPR/Cas9 for Drug Discovery Applications

Roderick Beijersbergen, Ph.D., Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

Stephanie Mohr, PhD, Lecturer, Genetics & Director, Drosophila RNAi Screening Center at Harvard Medical School

  • Impact of CRISPR/Cas9 for drug discovery in pharma and academia
  • Applications for functional screens, creating cell lines and disease models
  • Design and optimization of low- and high-throughput screens using CRISPR approaches
  • Application of CRISPR-knockout, -activation and -inhibition
  • Impact of new CRISPR technologies and reagents

Exploiting CRISPR, RNAi and Single-Cell Analysis: What You Need to Know Before and After

Sarah Boswell, PhD, Director of Sequencing Technologies, Laboratory of Systems Pharmacology and Director, Single-Cell Sequencing Core, Harvard Medical School

Jennifer Smith, Ph.D., Deputy Director, ICCB-Longwood Screening Facility, Harvard Medical School

  • Understanding inherent limitations and need for using complementary techniques
  • Examples of how multiple techniques have been put to good use for addressing biological questions
  • Evaluating and testing the reagents and tools
  • Insights on inherent challenges and ways to overcome it
  • Tackling data analysis

How to Design Machine Learning Models to Automatically Identify Biological Mechanisms

Greg Johnson, PhD, Scientist & Machine Learning Specialist, Allen Institute for Cell Science

  • Data representation
  • Model architecture
  • Designing an objective function

Challenges in Phenotypic, Target Agnostic Drug Discovery

Jesus Medina, PhD, Manager, Medicinal Chemistry, GlaxoSmithKline

  • Key challenges in cell-based phenotypic drug discovery efforts
  • Early assessment of the validity and viability of hits obtained from screens
  • What aspects of phenotypic drug discovery are the most advantages in drug discovery

MEDICINAL CHEMISTRY

Public-Private Partnerships in Drug Discovery

Dimitrios Tzalis, PhD, CEO, Taros Chemicals GmbH & Co. KG

  • How can collaborative public-private partnerships foster drug development?
  • What steps are being taken to close the "innovation gap"?
  • How can partnerships be beneficial for both parties?

Challenges in Phenotypic, Target Agnostic Drug Discovery

Jesus Medina, PhD, Manager, Medicinal Chemistry, GlaxoSmithKline

  • Key challenges in cell-based phenotypic drug discovery efforts
  • Early assessment of the validity and viability of hits obtained from screens
  • What aspects of phenotypic drug discovery are the most advantages in drug discovery CNS

Lost in Translation? Asking the Right Questions in the Right Language

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?

Improving the Success Rate of CNS Therapies

Takaomi C. Saido, PhD, Laboratory Head, Laboratory for Proteolytic Neuroscience, RIKEN Brain Science Institute

  • Why have more than 400 medication candidates for Alzheimer's Disease failed?
  • Which therapies have the best chance to succeed, and why?
  • Immunotherapy for neurodegenerative disease
  • What is the future of immunotherapy?

DRUG METABOLISM & TOXICITY

Gaps in Translating Preclinical Findings to the Clinic

William Proctor, PhD, Senior Scientist, Head of Investigative Toxicology, Department of Safety Assessment, Genentech

  • 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

Gary Gintant, PhD, Senior Research Fellow, Department of Integrative Pharmacology, Integrated Science and Technology, AbbVie

  • 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.

VESICLES & NANOPARTICLES

Best Source of Stem Cell Derived EVs

Paul Robbins, PhD, Professor and Director of the TSRI Center on Aging, Molecular Medicine, The Scripps Research Institute

  • Are EVs derived from embryonic stem cells as or more therapeutic than adult stem cells?
  • How similar in phenotype and function are the EVs from different stem cell sources?
  • Can allogeneic stem cell derived EVs be used clinically?

Challenges in EV Characterization

Alain Brisson, PhD, Emeritus Professor, UMR-CBMN CNRS, University of Bordeaux

  • Why do we need to better characterize EVs?
  • How reliable and efficient are the methods currently used for detecting, identifying, quantifying, isolating EVs?
  • What do we need for improving EV detection, identification, quantification, isolation?

* 活动内容有可能不事先告知作更动及调整。