Specialized Workshop F2 - Friday June 23, 2023: 7am to 5:30pm - Discussion Topic List

"Gene Therapy, Cell Therapy and Vaccine - Latest Developments, Challenges, and Solutions"

NEW Case Studies on Immunogenicity of Gene/Cell/Vaccine Therapies; Regulatory Expectations on Whats Really Needed for Immunogenicity Assessment; TAb or NAb and when Companion Diagnostic (CDX) is needed; Continuing the Industry/Regulators Discussion on LNP & Viral Vectors Immunogenicity; Progress in Tracking CAR-T Cells; Biodistribution of Replication Competent Viral Vectors and Vaccines; Method Development & Validation Strategies for ELISpot; Novel Applications of Next Generation Sequencing (NGS) & NanoString in Gene Therapy; State-of-the-art Recommendations for qPCR & ddPCR Validation; Latest Progress in High-Performance ddPCR for Genetic Assays

Session 1: qPCR & ddPCR Assays Development/Validation

  • Topic 1:

    qPCR, ddPCR, chipPCR, Hybridization Assay for LNP-mRNA Quantification and Current Industry Standards and Recommendations: Pros & Cons of each Technology, Best/Preferred Technology, Comparability between different Technologies, What to use for Reference Standard ? ddPCR does not use Standard Curve, any impact on Quantitation?
    Dr. John Kamerud, Director, Pfizer

  • Topic 2:

    Lessons Learned on use of ddPCR Technology: Focus on Limitations & Potential Sources of Error, Consideration for the Correct Results Interpretation, Recommendation for Carefully Validate the assay before Drawing Conclusions, Discrepancies between qPCR and ddPCR data due to Formation of Concatemers vs Monomeric Circular Episomes
    Dr. Lin Yang, Sr Director, Regenxbio

  • Topic 3:

    Validation Approaches for PCR-based Diagnostics: Quantitative, Semi-quantitative, Qualitative Approaches for Vaccine Efficacy Studies, Validating Parameters and Established pre-defined Assay Criteria for Maintaining Long-term Performance, QC-Trending, Reagent bridging, Proficiency Panels
    Dr. Warren Kalina, Executive Director, Pfizer

Session 2: NGS & NanoString Assay Development/Validation

  • Topic 4:

    Implementing Automation for NGS under GCP Guidelines: Current Best Practices for Fit-for-purpose Validation of a New Automation Procedure, Troubleshooting and Optimizing the new NGS procedures, Importance of Data Metrics, Strategies for Implementation and Functional Redundancy, Data Flow and Data Integrity
    Mr. Serge Picard, Sr Manager, BMS

  • Topic 5:

    Control and Validation of  NGS Pipelines: How to Control Software NGS Pipelines? What Validation should be Performed to Ensure Proper Analysis and Reproducibility? Importance of Wet Lab Controls, Validation Datasets  Reference Standards, Software Engineering Tools, Positive Controls for Reproducibility, QC parameters for Assay Sensitivity
    Ms. Jessica Seitzer, Vice President, Genomic Operations, Intellia Therapeutics

  • Topic 6:

    NanoString Assay Development/Validation: Strategies for Assessing Gene Signature Panels, Quickly Emerging Technology, Development of Custom, Highly Multiplexed Gene Expression Assays, Advantages vs Traditional PCR & NGS Platforms, Implementation in GCLP Compliant Environment for Clinical Sample Analysis
    Dr. Keith Merdek, Director, Transcriptomics and Human Genetics, Sanofi

Session 3: Immunogenicity Assessment: Focus on Transgene and Vaccine

  • Topic 7:

    Characterization of Immune Response to Novel Vaccine Candidates: Development of Antigen Binding, Virus Neutralization and Hemagglutination Inhibition Assays, Strategies to Critical Reagents Generation, Approaches to Assay Qualification and Validation for a Combination of Influenza, RSV, and SARS-CoV-2 Vaccine
    Dr. Liang Zhu, Director, Moderna

  • Topic 8:

    Issues and Considerations in Developing Transgene Immunogenicity Assays: Recent Developments in Gene Therapy Clinical Immunogenicity Prediction & Assessment, Choice for Surrogate Transgene Product, Impact of Auto-Antibodies on Assays Development, Importance of Outlier Factors to determine appropriate Cut Points
    Dr. Weiping Shao, Sr. Director & Head of US GxP Testing Labs, AstraZeneca

  • Topic 9:

    Immunogenicity to the Expressed Transgene in Gene Therapies: Where do we stand now? What type of Assays to use (TAb or NAb)? Is a Companion Diagnostic (CDx) needed? Should Cellular Response be tested? Current Recommendations and Lack of Regulatory Guidance on Transgene Immunogenicity Testing 
    Dr. Michael Partridge, Director, Regeneron

  • Topic 10:

    Challenges in Total Antibody (TAb) Assay Development to Characterize Pre-existing and Treatment Emergent anti-AAV Antibodies (TE-ADA): What Assay Format to use ? What is the best Positive Control? How to set Cut Points in the presence of High Pre-existing Reactivity? How to deal with TE-ADA when Pre-existing Reactivity is present? 
    Dr. Nicoletta Bivi, Sr Director, Eli Lilly

Session 4: PK & Biodistribution for Virus-vector Vaccine/Therapies and CAR-T Cells

  • Topic 11:

    Biodistribution for Vaccines: Challenges to develop Infectivity Assay to Detect Tissue-residual Infectious virus in all Virus-vector Based Vaccines, Optimized Homogenization Protocol to Release the Virus while Preserving its Activity, Issues with Homogenate Toxicity to the downstream Cell-based Assay and Lack of Real Quality Controls
    Dr. Weifeng Xu, Director, Merck

  • Topic 12:

    Replication Competent Viral Vectors: Application of Novel Bioanalytical Approaches to Understand the PK and Biodistribution, Limitations of Standard Techniques to Decouple Input Drug from Replicated Drug Products, Development of Hybridization Assay using Strand-specific Probes to assess Spatiotemporal Distribution in Tissues
    Dr. Adam Vigil, Director, Boehringer Ingelheim

  • Topic 13:

    Tracking Cellular Kinetics & Biodistribution of CAR T-Cells: Development of Cellular Kinetics Assays based on Clinical Trial Considerations, Highly Sensitive Technologies to Track Biodistribution of CAR T-cells, Development of Bioanalytical Tools for Tracking CAR-T Localization, Reducing Patient Visits and Specimen Volume by Universal Assay Frameworks   
    Dr. Naveen Dakappagari, Head, Biomarker & Diagnostic Development, Navigate Biopharma, a Novartis Subsidiary

Session 5: 2023 White Paper in Bioanalysis

  • 2023 White Paper on Gene Therapy, Cell Therapy and Vaccine - Latest Developments, Challenges, and Solutions

    Consensus & Conclusions on Gene Therapy, Cell Therapy and Vaccine - Latest Developments, Challenges, and Solutions for 2023 White Paper

Session Finale: ASK THE REGULATORS! Interactive Panel Discussion with All the Regulators

  • Regulatory Feedbacks on Submitted Studies and Inspection/Audit Outcomes on Gene Therapy, Cell Therapy & Vaccines

    Regulatory Panelists:

    • Dr. Nirjal Bhattarai (US FDA CBER)
    • Dr. Heba Degheidy (US FDA CBER)
    • Dr. Leslie Wagner (US FDA CBER)
    • Dr. Joshua Xu (US FDA NCTR)
    • Dr. Alessandra Buoninfante (EU EMA)
    • Dr. Christian Mayer (Austria AGES/EU EMA)
    • Dr. Sandra Diebold (UK MHRA-NIBSC)
    • Dr. Omar Tounekti (Health Canada)
    • Dr. Yoichi Tanaka (Japan MHLW)

Agenda at a Glance Agenda at a Glance