Specialized Workshop F2Friday September 30, 2022: 7am - 5:30pm - Lecture List

"Latest Advancements in Gene Therapy, Cell Therapy, and Vaccine Bioanalysis"

Immunogenicity & Risk Assessment of Gene, Cell Therapies & Vaccines; LNP & Viral Vectors for Oligonucleotide-based Therapy/Vaccines; qPCR/ddPCR Method Development & Validation Strategies; NGS & nanoString New and Specific Developments; Current Regulatory Expectations

Part 1: Immunogenicity & Risk Assessment for Gene Therapy, Cell Therapy and Vaccines

  • Lecture 1:

    What are the Expectations for Immunogenicity Risk Assessment for Gene Therapy programs?

  • Lecture 2:

    Immunogenicity on Novel Drug Modalities: Lesson Learned based on Novel Data from Non-clinical and Clinical Studies

  • Lecture 3:

    Current Development/Validation challenges of in vitro Human PBMC Cytokine Release Assays for Immunogenicity Risk Assessment: How can these assays be Applied across Multiple Modalities to Justify Bioanalytical Strategy?

  • Lecture 4:

    Recent Developments in Vaccine Immunogenicity Strategies: Harmonization Process, China NMPA Draft Guidelines for Vaccine Immunogenicity and Novel Case studies

Part 2: LNP and Viral Vectors Immunogenicity for Oligonucleotide-based Therapies & Vaccines

  • Lecture 5:

    Immunogenicity Assessment for Oligonucleotide-based Therapeutics: Lesson Learned based on Novel Data from Non-clinical and Clinical Studies

  • Lecture 6:

    Are LNPs used for Oligonucleotide-based Therapies & Vaccines Immunogenic? State-of-art Industry Experience and Novel Data in this field

  • Lecture 7:

    Comparing & Contrasting Cell-based vs LBA for pre-existing anti-AAV TAbs and NAbs Assays: How to select a suitable assay platform?

Part 3: qPCR/ddPCR Infectivity, Vaccine & Biodistribution Assays

  • Lecture 8:

    Recent and Novel Developments on Universal Genomic Assay Strategy to Characterize the Biodistribution in Gene Therapy by qPCR/ddPCR

  • Lecture 9:

    Updated Strategies for qPCR Infectivity Assays Development/Validation for Gene Therapy and Oncolytic Virus

  • Lecture 10:

    Advanced approaches in Molecular Assays to Support Vaccines Studies: qPCR, ddPCR and NGS Applications

Part 4: NGS and NanoString Assays

  • Lectures 11, 12:

    Next Generation Sequencing (NGS) Bioanalytical Applications in Genetics & Genomics from Discovery to Clinical Development of Cell & Gene Therapy

    • Understanding the fit-for purpose Validation for NGS in drug development - Lecture 11
    • Customizing NGS Bioanalytical Applications to Different Phases of Drug Development: What to consider in NGS use in Discovery vs Clinical Monitoring - Lecture 12
  • Lecture 13:

    Recent Developments in Using NanoString Technology as an Alternative to qPCR: Understanding the Technology and its Potential Application in Bioanalysis

Part 5: White Paper in Bioanalysis

  • 2022 White Paper on Gene Therapy, Cell Therapy and Vaccine Topics:

    Consensus & Conclusions on Gene Therapy, Cell Therapy and Vaccine Topics for 2022 White Paper


  • Panel Discussion with All the Regulators:

    Have an Open Dialogue with the Regulators including US FDA, EU EMA, UK MHRA, Health Canada Japan MHLW
    Ask the Regulators any Questions You Have on Gene Therapy, Cell Therapy, Vaccines and Hear Their Feedbacks on Submitted Studies and Inspections/Audits Outcomes

Agenda at a Glance Agenda at a Glance