Topic 1:

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?

Topic 2:

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?

Topic 3:

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.

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.

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?

Topic 6:

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.

Topic 7:

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.

Topic 8:

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.

Topic 9:

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.

Topic 10:

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.

Topic 11:

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.

Topic 12:

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.

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

Panel Discussion with All the Regulators:

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