Specialized Workshop Th2 - Thursday May 09, 2024: 7am to 5:30pm - Discussion Topic List

"Advanced Strategies for Cell-Based Assays"

Cell-Based Assays Applications in Cellular Immunogenicity/NAb Assays, Cellular Kinetics for Cell Therapy/CAR-T Cells and Vaccine Functional Assays; Issues in Reporting NAb Data from Cell-Based Assays; Recent Approaches in CBA Development/Validation and Technology Advancements; Regulatory Expectation on CBA for Submitted Studies; Newest Innovations in High Dimensional Flow Cytometry/Mass Cytometry; Novel Applications of Artificial Intelligence (AI) & Machine Learning (ML) for Flow Cytometry; New Development in Flow Cytometry for Exploratory & Clinical Biomarkers/RO/TE; Current Status of Single-Cell Assays for High Dimensional Cytometry in Clinical Trials

Session 1: High Dimensional Flow Cytometry: Implementing AI/ML/Automation, Ensuring Quality, Improving Manual Processing & Data/Gating Analysis

  • Topic 1:

    Advancements in AI/ML-based High Dimensional (HD) Flow Cytometry as Essential Feature of New Drug Development Programs: State-of-the-art applications to assess Cellular Drug Target Binding and pathway Target Engagement TE/PD , cell types deep exploration/phenotyping, signaling events in treated subjects, need for Artificial Intelligence (AI) and Machine Learning (ML) to relieve the data analysis and gating bottleneck
    Dr. Steve Keller, Senior Director, Biomarker Bioanalysis, AbbVie

  • Topic 2:

    Up-to-date Implementation of High Dimensional (HD) Flow Cytometry in Global Clinical Trials and Fit for Purpose (FFP) Stream-lined Gating: need for Exhaustive Manual Analysis strategies including 1000s of pre-specified reportable per sample, novel approach which includes the pre-specification of tiered reportable based on project impact to be delivered within a specific turn-around-time, FFP gating strategies focused on addressing key questions
    Dr. Bill O'Gorman, Director of Clinical Cytometry, Genentech

  • Topic 3:

    Minimizing Manual Errors of HD Flow Cytometry Manual Process in Discovery & Clinical Applications using Automation: Increased errors on cocktail preparation due to HD Flow Cytometry increased number of markers per assay, focus on common errors in the creation of master mixes containing all the assay antibodies/controls, novel application of automated cocktailing liquid handling to combat the problem of errors
    Dr. Michael Nathan Hedrick, Senior Scientific Director Clinical Flow Cytometery, Bristol Myers Squibb

Session 2: Biomarker & Cell Therapy: Innovative Flow Cytometry Applications for RO/TE & CAR-T PK-like Assays

  • Topic 4:

    Considerations in the Development/Validation of Flow Cytometry Assays to directly detect Target Engagement (TE) Pharmacodynamic (PD) Biomarkers in a clinical trial, importance of TE/PD data to inform dosing decisions to predict clinical efficacy, use of TE data to provide proof of mechanism data in a clinical study, limitations of TE/PD data in informing both dosing decisions and proof of mechanism data in a clinical setting
    Dr. Erin Stevens, Head of Clinical Biomarker Technologies, Translational Clinical Sciences, Pfizer

  • Topic 5:

    Novel Strategies in Employing Surrogate Materials & Iterative Approaches to demonstrate the Fit for Purpose (FFP) Performance of the Flow Cytometry assay for Biomarker and Cell Therapy Applications, limitations of contrived samples in validation, comparison between surrogate material used in Receptor Occupancy (RO) assays and study material, cell surrogate used in quantitative Cell Therapy/CAR-T PK-like assays validation
    Dr. Steven Eck, Director, Integrated Bioanalysis, Astra Zeneca

  • Topic 6:

    Cutting-edge Applications of Flow Cytometry Receptor Occupancy (RO) Development/Validation and Implementation in Clinical Trials, challenges/mechanisms in/for stabilizing blood samples and current technologies available, strategies for PBMC-based RO assay format and frozen blood RO assay format, specific design to quantify binding of drugs to their cell surface targets to generate Pharmacodynamic (PD) Biomarker data
    Dr. Katharine Grugan, Associate Scientific Director, Bioanalytical Discovery and Development Sciences, J&J Innovative Medicine

  • Topic 7:

    Innovative Development of a Dual Flow Cytometry Assay Format for RO Bioanalysis in Circulating T Cells in Whole Blood: Building phospho-Flow Cytometry Assay for Pharmacodynamic (PD) Biomarkers monitoring of mTOR-inhibitor, multi-parameter assessment of the immunomodulatory effect, evaluating kinetics of biomolecules uptake in primary splenocytes for de-risking, conducting RO analysis based on bound drug assay and total target modulation
    Dr. Polina Goihberg, Discipline Lead, Quantitative Biomarkers and Biomeasures, Pfizer

Session 3: Immunogenicity & Oncology: Advanced Applications of Cell-Based Assays and Flow Cytometry

  • Topic 8:

    Key Steps in the Development of an in vitro Cell-Based Assay (CBA) to Investigate the Ability of ADA to Block Function of Endogenous Receptor to Address Safety Concerns: Format & appropriate controls selection, cell line choice, proper endpoint of the measurement, optimization of assay parameters, characterization of ADA cross-reactivity with endogenous T cell co-stimulatory receptor ligands to understand potential safety observations
    Dr. Grzegorz Terszowski, Cell-Based Assay Team Lead, Novartis

  • Topic 9:

    Sophisticated Procedures for Single Cell Isolation for Investigating Human Tumor Infiltrating Leukocytes (TIL) by Flow Cytometry in Oncology for Drug Development: Challenges in the assessment of the immune compartment in tumor environment, tissue isolation from solid tumor site, influence of pre-analytical factors in tissue preparation and quality for immuno-phenotyping, assay validation in investigating TIL
    Mr. Paul Trampont, Senior Scientific Director, Clinical Biomarker Laboratory, Moderna

  • Topic 10:

    Sophisticated Measurement of Cell Mediated Immunogenicity (CMI) Elicited by Vaccines: Assay selection based on clinical purpose and reportable parameters, strategies for High Parameter Spectral Cytometry development/validation and deployment for functional immune cell profiling assays, key considerations in clinical testing design, critical reagents, Focus on systematic data analysis & gating analysis, impact of PBMC quality to monitor functional cellular responses
    Ms. Shannon McGrath, Director, Flow Cytometry, Clinical Biomarker Laboratory, Moderna

Session 4: Breaking through Complex Panels & Technology Barriers to Enable Advanced HD Flow/Mass Cytometry Applications

  • Topic 11:

    Strategies for Designing Complex Panels in High Dimensional (HD) Flow Cytometry: Evolution of output data from simple panel with few reportable to complex panel with many reportable, Flow Cytometry data mapping during clinical trial from LB domain in the past to Cell Phenotyping domain now, controlled terminology explanation using Flow Cytometry panels, how data are converted internally/externally and CDISC rules for test codes
    Dr. Shuyu Yao, Associate Director, Group Lead Cellular Biomarkers, GlaxoSmithKline

  • Topic 12:

    Successful Handling of Complex Panels for HD Mass Cytometry with 40+ Markers and Multiple Gated Subsets to be Measured: Needs for Complex Panels correlation marker by marker manually when utilizing automated analysis and asses for performance assay to assay, novel validated & automated pipeline correlated with manual gating strategy and a weighted batch/marker scoring system to ensure data quality
    Mr. Greg Hopkins, Associate Manager, Analytical Sciences, Regeneron

  • Topic 13:

    Breaking through Technology Barriers to Enable HD Mass Cytometry for in vivo studies for the use in PK/PD and Modeling: Correcting for batch effects in large sample studies with multiplexed barcoding to increase throughput, method development for longitudinal in vivo HD PK/PD studies, developing high dimensional/intracellular/PD readouts to enable dose projection modeling, and target dose modeling on tissue specific deep phenotyping
    Mr. Chad Stevens, Senior Scientist, Cellular Biomeasures, Pfizer

Session 5: 2024 White Paper in Bioanalysis

  • 2024 White Paper on Advanced Strategies for Cell-Based Assays

    Consensus & Conclusions on Advanced Strategies for Cell-Based Assays for 2024 White Paper

Agenda at a Glance Agenda at a Glance