Training Session F2 - 7am to 5:30pm, Friday April 5, 2019

"The Gene Therapy Bioanalytical Challenges: PK/Biodistribution, Immunogenicity and Shedding Assays, qPCR, ELISPOT, Flow Cytometry in Bioanalytical labs, and Regulatory Perspectives"

What you need to know to develop bioanalytical assays for Gene Therapy and how Gene Therapy is impacting Bioanalysis in both Pharma and CRO - Multiple platforms & multiple approaches to deal with unsolved bioanalytical issues

  • Lesson 1:

    Evolution of WRIB Recommendations on Gene Therapy from 2017 WP to 2018 WP: Where are we now and what next?

Part 1: Biodistribution
  • Lesson 2:

    Drug Distribution Analysis in the Preclinical Development of Gene and Cell Therapy Products

  • Lesson 3:

    Biodistribution - Recent Development in Transgene Expression Quantification by Hybrid LBA/LCMS

  • Lesson 4:

    qPCR as integral part of a Bioanalytical Lab for Gene/Nucleic Acids, CAR-T, Oncolytic Virus, Gene Editing and other Emerging Therapies: Method development & Validation Strategies

  • Lesson 5:

    Gene Therapy: Utilization of molecular vs flow cytometry methods for PK assessments and MRD analysis, analytical considerations and challenges

  • Panel Discussion:

    Biodistribution Panel Discussion

Part 2: Immunogenicity
  • Lesson 6:

    Immunogenicity Strategies against Capsid and Transgene product in support of Viral Vector based Gene Therapy

  • Lesson 7:

    Crucial Importance of a Thorough and In-depth Evaluation of Pre-existing Abs to the Viral Capsid during Immunogenicity Assessment of Viral Vectors based Gene Therapy: Shire Experience

  • Lesson 8:

    Evaluation of Pre-existing Abs to the Viral Capsid during Immunogenicity Assessment of Viral Vectors based Gene Therapy: BMS Experience

  • Lesson 9:

    ELISPOT as the Gold Standard in Cellular Immunity Evaluation in AAV based Gene Therapy: Method Development and Validation Strategies

  • Lesson 10:

    Challenges in Cell-Based Nabs Assay development for Gene Therapy: Case Studies

  • Panel Discussion:

    Immunogenicity Panel Discussion

Part 3: Infectivity & Biomarkers Assays
  • Lesson 11:

    Viral Vector Shedding Assays in Gene Therapy Development: Quantitative & Infectivity Assays

  • Lesson 12:

    Biomarkers in Gene Therapy Clinical Development

  • Panel Discussion:

    Infectivity & Biomarkers Assays Panel Discussion

Part 4: Emerging Therapies
  • Lesson 13:

    CRISPR and Applications of Genome Editing: Bioanalytical Strategies & Challenges

  • Lesson 14:

    CRISPR Gene Editing Therapeutic: Preclinical Assays Strategies & Challenges

  • Panel Discussion:

    Emerging Therapies Panel Discussion

Finale: Ask the Regulators
  • Panel Discussion with the Regulators:

    Address your questions on Gene Therapies and interact directly with US FDA CBER



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