Topic 1:

Current role of in Vitro Models in developing Genetic Medicine: Emphasize the critical role that in vitro translational models play in the early stages of genetic medicine development; Providing insights into gene expression, safety, efficacy. Examine challenges in predictability: Successes/limitations experienced in mRNA-LNP; Innovative strategies/technologies, advanced bioengineering methods, Artificial Intelligence & Machine Learning (AI & ML) integration, and enhanced model systems.
Dr. Swati Gupta, Executive Director Clinical Immunogenicity & Translational Science, AbbVie

Topic 2:

Can Patients be Redosed with an in vivo CRISPR/Cas9 Genome Editing Therapy?  Consideration on the delivery system via Lipid Nanoparticle (LNP) instead of AAV and AAV GT cannot be re-dosed vs LNP Targeting TTR gene re-dosed for ATTR patients; Low Immunogenicity Risk Assessment in non-clinical and clinical studies for anti-drug antibodies and anti-Cas9 protein antibodies after the 1st dose and re-dose for NHP to human translation.
Dr. Yuanxin Xu, Senior Vice President Early Development & Translational Medicine, Intellia

Topic 3:

State-of-the-art Approaches used to assess Immunogenicity for mRNA-LNP and Unique Challenges: Developing anti-Protein and NAb assays with multiple confirmation assays for multiple mRNA derived proteins; anti-PEG assay development how changes in antibody isotypes can affect the interpretation of results from clinical studies; anti-LNP assay development, ensuring LNP is coated onto the plate, identifying optimal confirmation conditions, and reducing effects of LNP stickiness.
Dr. Melissa Taylor, Associate Director Bioanalytical & Molecular Assays, Moderna

Topic 4:

Challenges & Opportunity for Next Generation Cell Therapy Bioanalysis on Global Scale: Latest and unique bioanalytical considerations in drug development, discovery & clinical; Crucial importance of Cellular Kinetics; Immunogenicity & Immune Response characterization for complex cell therapies including Cytokine Release Syndrome (CRS) & Allogenicity; Acceleration through holistic, global strategies with a diverse Cell Therapy portfolio.
Ms. Daisy Yuill, Executive Director Integrated Bioanalysis, AstraZeneca

Topic 5:

Cytokine Release Syndrome (CRS): Systemic inflammatory response caused by the rapid release of cytokines from immune cells following Cell Therapy: Importance to independently evaluate the relationship of Anti-Therapeutic Antibody (ATA) with the anticipated or relevant Adverse Events (AE) driven by product-specific Immunogenicity Risk Assessment and Risk Management Plan; If an AE is driven by Cell Therapy and not by ATA, should it be part of the output?
Dr. Vibha Jawa, Executive Director Translational Medicine, Bristol Myers Squibb

Topic 6:

Highlighting the Immunogenicity Clinical Relevance on development of Autologous CAR-T Cell Therapy based on indications: Bioanalytical strategies/methodologies on assessing immunogenicity; Clinical relevance related to PK, efficacy, and safety; How to mitigate clinical immunogenicity for Next Generation Cell Therapy Design based on probability of success; Challenges on employing cell therapies for solid tumor indications and impact of immunogenicity on PK, efficacy and safety.
Dr. Tong-Yuan Yang, Senior Director Preclinical Sciences & Translational Safety, J&J Innovative Medicine

Topic 7:

Translatability/Synergy between CMC Bioassays and Bioanalytical Assays to accelerate Gene and Cell Therapy Development: Comparing technologies/processes used for method development and validation; Development of NGS off-target Detection Assay to support both drug product characterization and safety monitoring of patients in a Cell Therapy clinical trial; Target Engagement (TE) Assay using RT-qPCR expression in animals tissue samples adopted by CMC for potency assay probe/primers sequences.
Dr. Yanmei Lu, Vice President Biomarker & BioAnalytical Sciences, Sangamo

Topic 8:

Advancing RSV Vaccines with Innovative Bioanalytical Approaches; Efficacy evaluation with a three-assay Customized PCR Algorithm for detecting the vaccine strain in negative samples; mRNA Vaccine efficacy evaluation by NGS for using nasal swabs. Comparison of NGS amplicon sequencing and NGS whole genome sequencing; Advantages of whole genome sequencing utilizing random primers to make it less susceptible to the effects of viral mutations.
Dr. Rocio Murphy, Senior Director & Head of Global Clinical Immunology, Sanofi

Topic 9:

Novel Streamlined Approach for Normalizer Gene Identification using Next-Generation Sequencing (NGS) methods and differential gene expression analysis: Addressing variability from the target disease, tissue pathology, inflammation, patient population, cell type, genetic differences, different RNA species, cell type composition, biopsy handling and RNA quality; Accuracy of PCR-based methods that measure mRNA transcript expression improvement with a rigorous normalization strategy.
Dr. Andrew Melton, Senior Director BioAnalytical Sciences, BioMarin

Topic 10:

Leveraging Artificial Intelligence (AI) based bioinformatics NGS pipelines to interrogate genomics data pertaining to somatic analysis of tumor samples and germline samples: Best Practices for Implementing AI in Clinical Genomic Labs by leveraging AI to enhance genomic analysis by ensuring robust data integration, maintaining high-quality standards, and providing continuous training for lab personnel on new analytic tools; Integrating Information with Wet Lab Workflows.
Dr. Mark Stern, Senior Director Clinical Genomics & Molecular Assay Development, Bristol Myers Squibb

Topic 11:

What are main reasons to evaluate Anti-transgene Protein Immune Response and how this information can inform development of Gene Therapies? What are the critical factors that can determine the need and value of assessment of anti-transgene protein immune response? What is the Regulatory Position on this assessment and when is it asked for? What are the risk-based recommendations and current industry experience?
Dr. Boris Gorovits, Vice President Non-clinical & Clinical Bioanalysis, Regeneron

Topic 12:

How do we identify a preexisting anti-AAV Threshold that maximizes Gene Therapy Efficacy in all patients? What non-clinical data would help identify the correct anti-AAV threshold, in vitro transduction, passive immunization, pharmacology studies? What clinical data would help identify the most appropriate anti-AAV thresh-hold? Data from TAb/NAb and TI Assays, stepwise increase in titer for inclusion; IVD, IDE and CDx requirements? What strategies can we use to permit AAV Redosing?
Dr. Michael Partridge, Senior Director Bioanalytical Sciences, Regeneron
Dr. Susan Irvin, Senior Staff Scientist, Regeneron

Topic 13:

Lesson Learned on Applying/Non-applying the Current Paradigm for Immunogenicity Testing for Gene Therapy; Immunogenicity Assessment & Clinical Relevance with Focus on Regulatory Feedback on TAb/NAb Assays; Agreement on recent recommendations of not using NAb Assays for Gene Therapy; Advices on Putting Together the Integrated Summary of Immunogenicity (ISI); Latest agreements/disagreements on why we should not follow the ADA guidance/3-tiered paradigm designed for Biotherapeutics; Which immunogenicity assays are providing useful information in the clinic?
Mr. David Escobar, Associate Director Ligand Binding Assay Technologies , Navigate

2025 White Paper on Gene, Cell, and Vaccine Therapies Immunogenicity & Technologies - Latest Advances, Challenges and Solutions from Internationally Recognized Key Opinion Leaders

Consensus & Conclusions on Gene, Cell, and Vaccine Therapies Immunogenicity & Technologies - Latest Advances, Challenges and Solutions from Internationally Recognized Key Opinion Leaders for 2025 White Paper

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

Regulatory Panelists:

• Dr. Zuben Sauna (US FDA)
• Ms. Leslie Wagner (US FDA)
• Dr. Wojciech Jankowski (US FDA)
• Dr. Binsheng Gong (US FDA )
• Dr. Wenming Xiao (US FDA)
• Dr. Alessandra Buoninfante (EU EMA)
• Mr. Christian Mayer (Austria AGES / EU EMA)
• Dr. Claire Kerridge (UK MHRA)
• Dr. Anna Nowocin (UK MHRA)
• Dr. Sandra Prior (UK MHRA)
• Dr. Omar Tounekti (Health Canada)
• Dr. Sarah Wassmer (Health Canada)
• Dr. Chad Irwin (Health Canada)
• Dr. Bradley Scott (Health Canada)
• Dr. Adrian Wong (Health Canada)