Topic 1:

Importance of Immunogenicity Risk Assessment & Mitigation for Gene Therapy (GT) and mRNA-Lipid Nanoparticle (mRNA-LNP): Understanding the potential immune responses triggered and their impact on safety and efficacy, methods using in vitro tools, assays for measuring immune cell activation & cytokine profiling, use of immunomodulatory agents, managing immunogenicity in clinical trials and real-world settings
Dr. Swati Gupta, Executive Director, Clinical Immunogenicity, AbbVie

Topic 2:

Are Risk-Based Approaches for Immunogenicity Assessment Relevant for Gene, Cell, and Vaccine Therapies as they are for Biotherapeutics? Understanding the drug design & clinical development strategies to differentiate Vaccine Wanted immunogenicity vs Gene & Cell Therapies Unwanted one, technical challenges & alternative approaches for high-risk products, exhaustive interpretation of clinical data
Dr. Yuanxin Xu, Senior Vice President, Intellia

Topic 3:

How Immunogenicity Risk Assessments for Gene Therapy can be used to rate the Risk for Potential Untoward Immune Activation: Current risk assessment paradigm that may not apply to Gene Therapy ( three-tiered assays, cut points calculations, need for NAb assays) challenging the thinking on applying the current immunogenicity testing paradigm to Gene Therapy vs customizing current practices to address unique risks for GT vs immune-related risk specific for GT
Dr. Christine Grimaldi, Director, Assay Development Group, Regeneron

Topic 4:

Lesson Learned on Immunogenicity Assessment for Cell Therapy Including Incidence and Impact on PK: Immunogenicity on-set and cellular kinetics, best way to determine impact of immunogenicity on cellular kinetics, method validation to determine if the method meets the necessary characteristics to allow the use of S/N over titer, best to present data to Regulatory Agencies to seek feedback for future study support
Dr. Johanna Mora, Senior Director, Bioanalysis, Bristol Myers Squibb

Topic 5:

New Considerations for Allogenic Cell Therapies Immunogenicity Assays: What aspects are critical for anti-Extracellular Domain (ECD) assays? What is critical to examine for CAR ECD immunogenicity assay design and specificity? What are the key aspects for Anti-HLA antibody analysis and correlation with HLA-typing? Challenges with generation of Critical Reagents for the individual/armoring components?
Dr. Christopher Beaver, Senior Director, Cell Therapies Immunogenicity and Immunoassays, Takeda

Topic 6:

Interpretation of the 2023 EU EMA Guideline on Clinical Evaluation of Vaccines and its requirement for a Fully Validated Method used in pivotal trials to measure Immune Parameters as primary and/or major secondary endpoints, What does it mean to perform a Full Validation vs Fit for Purpose (FFP) Validation for Vaccine Clinical Assays? FFP Validation can be very limited or very extensive based on assay intended use when considering a specific study objective
Mr. Francis Dessy, Director and Clinical Assay Senior Advisor, GlaxoSmithKline

Topic 7:

Current Best Practices for Method Development/Validation of Next-Generation Sequencing (NGS): Guidance for Fit-for-Purpose Validation, software pipeline for genetic alteration calls, important factors to consider in developing NGS technologies in support of Gene, Cell and Vaccine Therapies, NGS applications for discovery vs clinical monitoring, gene editing risk assessment, similarities/difference between NGS & NanoString Validation
Dr. Nathan Riccitelli, Associate Director, Molecular Assays, Navigate

Topic 8:

Expanded Applications of Next-Generation Sequencing (NGS) in a Regulated Environment: A new paradigm for Regulatory Agencies to consider, need for high depth sequencing of both patient somatic and germline mutations is required with high quality, need for regulatory frameworks, focus on the up front sequencing that is required as part of Individualized Neoantigen Therapy (INT) sequencing on a per patient level
Dr. Jean-Claude Marshall, Vice President & Head of Clinical Assays, Supply and Logistics, Moderna

Topic 9:

Current NGS/Genomic Analytical Tools to Enable Cell Therapy and in vivo CRISPR Clinical Studies: Integrative genomics approach combining biochemical, cell-based, and computational methods to support CRISPR Gene Editing Therapies, incorporating genomic diversity in sgRNA sequence selection to ensure efficacy and specificity across the patient population,characterization of in vivo Gene Editing Therapies
Ms. Jessica Seitzer, Vice President Genomic Operations, Intellia

Topic 10:

Development, Optimization and Validation of Automated NGS/Genomic Assays for Clinical FFPE Sample Analysis: Leveraging novel approaches to monitor clinical-grade genomic assay quality, evaluation of emerging genomics technologies to integrate within the framework of a clinical genomic lab, exploratory genomics for developing biomarkers, risks of shifting from an exploratory genomics platform to a Regulated/IVD platform
Dr. Mark Stern, Senior Director, Clinical Genomics & Molecular Assay Development, Bristol Myers Squibb

Topic 11:

Development of a 2-tiered Approach where PCR Sensitivity is coupled with Sequencing for Optimal Diagnostic Accuracy: Amplicon sequencing used to increase specificity when used with orthogonal diagnostic approaches, use of very specific primers in validation by leveraging both contrived and real samples for derivation of specificity, sensitivity, and LOD, assay intended use where specific targets are validated
Dr. Warren Kalina, Executive Director Diagnostic Assays, Pfizer

Topic 12:

Challenges in the Development of Quantitative Dengue Detection PCR: Thorough understanding the PCR process and key steps involved in the multiplex process, common issues that arise during qPCR method development, focus on how a change in reagent lot can impact qPCR assay and affect the sensitivity, troubleshooting PCR issues in Dengue detection, highlighting the importance of careful experimentation
Dr. Dewal Jani, Director, Clinical Assay Development, Vaccine Research and Analytics, Takeda

Topic 13:

Advantages of Using qPCR rather than bDNA for mRNA-Derived Protein Quantification to support PK evaluation: Comparison of data and performance, strategy for conversion from bDNA to quantitation by qPCR, challenges associated with choosing an appropriate reference standard for quantitation of mRNA, bioanalytical support for mRNA therapeutic including selection of endpoints and lifecycle management of assays
Mr. Jason DelCarpini, Director, Bioanalytics, Moderna

2024 White Paper on Advanced Strategies for Molecular Assays and Immunogenicity of Gene Therapy, Cell Therapy and Vaccine

Consensus & Conclusions on Advanced Strategies for Molecular Assays and Immunogenicity of Gene Therapy, Cell Therapy and Vaccine for 2024 White Paper

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

Regulatory Panelists:

• Dr. Zuben Sauna (US FDA CBER)
• Dr. Vijaya Simhadri (US FDA CBER)
• Ms. Leslie Wagner (US FDA CBER)
• Dr. Joshua Xu (US FDA NCTR)
• Dr. Alessandra Buoninfante (EU EMA)
• Mr. Christian Mayer (Austria AGES / EU EMA)
• Dr. Shirley Hopper (UK MHRA)
• Dr. Meenu Wadhwa (UK MHRA)
• Dr. Sandra Diebold (UK MHRA)
• Dr. Sandra Prior (UK MHRA)
• Dr. Omar Tounekti (Health Canada)
• Dr. Sarah Wassmer (Health Canada)
• Dr. Chad Irwin (Health Canada)
• Dr. Julie Joseph (Health Canada)
• Dr. Adrian Wong (Health Canada)