Topic 1:

Important lesson learned in dealing with AAV antibodies in clinical trials based on the Selected Route of Administration; Assessment of Preexisting anti-AAV antibodies and monitoring of treatment-induced anti-AAV9 antibodies following  4 different routes of administration of AAV-based Gene Therapy: into the Cerebrospinal Fluid (CSF), Intravenous administration; Subretinal administration and Suprachoroidal administration of AAV of clinical participants; Thorough data evaluation, considerations and recommendations.
Mr. Michele Fiscella, Vice President Translational & Bioanalytical Sciences, Regenxbio

Topic 2:

Latest perspectives/comparison on Characteristics of Neutralizing Antibodies (NAb) and Total Antibodies (TAb) Assays against the Viral Vector: Should we update previous Industry/Regulators' recommendations on NAb/TAb assays? Splitting samples into 4 categories from data correlation from same sample set tested in NAb/TAb assays; If/how can the preclinical data be used to determine the best approach for inclusion /exclusion based on pre-existing antibodies status in the first in human study?
Dr. Nancy Ramia , Bioanalytical Lead, UCB

Topic 3:

Overcoming current challenges in Assessing Preexisting Antibodies for AAV-GT: TAb/NAb Assay development for multiple AAV Serotypes or species; TAb/NAb assay fit-for purpose validation; Understanding the impact on Gene therapy Efficacy of pre-existing anti-AAV TAbs /NAbs and AAV-GT dependence on vector transduction efficacy; Importance of screening for TAb/NAb in preclinical models; In depth considerations on Vector Quality Variability and MOI adjustments; Challenges in detecting low-level immunity and distinguishing treatment effects.
Dr. Danielle Salha, Senior Executive Director Global Immunology, Altasciences

Topic 4:

Continue development of NGS Bioanalytical Assays in support of CRISPR Gene Editing Therapies; Advanced methods developed to characterize sgRNA Sequence Impurities; Important factors to consider when evaluating NGS sgRNA sequencing impurity data; Challenges NGS when analyzing sgRNA molecules with Complex Secondary Structures/Modifications; Current clinical trials evaluating Off-target Profile across different lots of sgRNA material throughout the lifecycle of process development and manufacturing.
Ms. Jessica Seitzer, Vice President Genomics & Analytical Development, Intellia

Topic 5:

Novel progresses on SARS-CoV-2 Whole Genome Sequencing performed on nasopharyngeal/nasal swab samples to monitor for the incidence of resistance mutations; Data Investigation revealing that mutations were called in instances where there was strand bias (reads from only one strand); Updating the Bioinformatics Pipeline for Variant with a filter to remove artifacts from strand bias to significantly decrease in the number of false mutations arising from strand bias.
Dr. Elizabeth Dushin, Associate Director, Biomarker Clinical Assay Lead, Pfizer

Topic 6:

Current Challenges in the development of Multiplex PCR Assays; How can Reproducibility & Sensitivity be maximized for detecting transcripts over a broad dynamic range; Optimizing Mutiplex Molecular Assay for assessing gene expression for in Gene, Cell, Vaccine Therapies response monitoring; Development of both Multiplex qPCR and NanoString assays involves distinct technical challenges, centered on assay design complexity, achieving specificity and sensitivity, and data analysis; Multiplex qPCR struggles with primer interactions/competition, while NanoString faces issues with data normalization, quality control for low-input samples; How to ensure precise measurement of gene expression in Multiplex Molecular Assays as crucial component for evaluating therapeutic effectiveness.
Dr. Nathan Riccitelli, Associate Director Molecular Innovations, Navigate BioPharma

Topic 7:

Advances in Monitoring RNA Therapies Immunogenicity by evaluating Innate Immune Responses and Biomarkers to ensure safety in therapeutic development; PCR-based Bioanalysis to ensure data Integrity & Compliance in a rigorously controlled Regulated Laboratory Environment; Challenges with RNA immunogenicity assessment in Positive Control Generation; Clinical Context of Immunogenicity Data Interpretation by exploring whether Sensitivity & Drug tolerance should be prioritized or if meaningful ADA monitoring is the goal.
Dr. Maria Jadhav, Director GCP Bioanalysis Team Lead , Novartis

Topic 8:

Low Immunogenicity Risk & Clinical ADA reported for siRNA modality; What value does routine ADA testing provide? Understanding the role of siRNAs metabolites on-target & active and with similar metabolic profile in non-clinical/clinical; What value does metabolite ID in clinic provide? Liver-targeted siRNAs mostly metabolized in the plasma/liver, and minimally in kidneys: Why do we need to test in urine? What value do clinical renal impairment studies provide?
Dr. Michael Partridge, Senior Director Bioanalytical Sciences & Immunogenicity Strategic Lead, Regeneron

Topic 9:

Immunogenicity impact on the latest RNA Therapeutics (Oligonucleotides & Vaccines); ADA Assessment as critical component of Clinical Study Design to measure the safety & efficacy of RNA therapeutics; What are the current unresolved challenges involved in measuring ADA responses to RNA therapeutics? Why are Regulatory Agencies requiring Immunogenicity assessment for ASO & siRNA? What are the strategies and specific design to ensure that ADA assays development, validation, sample analysis for RNA Therapeutics meet Regulatory Expectations?
Dr. Lynn Kamen, Executive Director Immunology & Scientific Officer, Bioagilytix

Topic 10:

Emerging Next-generation CAR-T Therapies in Oncology designed to broaden clinical indications while enhancing safety profiles and therapeutic efficacy; Emphasize the growing need to measure CAR-T cells in non-blood matrices such as Bone Marrow Aspirate (BMA) and Cerebrospinal Fluid (CSF) critical for addressing questions related to safety, malignancies, efficacy in indications involving the central nervous system or hematologic malignancies.  Bioanalytical Strategies for supporting the development and characterization of Complex Cell Therapies in complex tissue environments and challenging patient populations; Approaches and alternative strategies to overcome challenges in complex matrices to improve data quality, applicability, and reliability; Highlighting emerging assay development strategies, including the selection and validation of critical reagents, optimization of sample processing workflows, and solutions to overcome analytical challenges in these complex matrices; Use of a genomic equivalence approach that enhances DNA isolation and enables more sensitive detection and quantification of CAR-T cells in CSF. 
Dr. Nanda Balasubramanian, Associate Director Precision Medicine, Bioanalytical & Translational Science, Bristol Myers Squibb

Topic 11:

Sophisticated technologies used in the development of next-generation Personalized Cancer Vaccines; Innovative application of Molecular Assays used in design, development, monitoring of cancer vaccines; Accurate identification of clonal, immunogenic neoantigens is essential for inducing durable antitumor immune responses; Avoiding sequencing errors, and limited resolution of tumor clonal architecture, leading to inclusion of low-confidence or subclonal mutations that compromise vaccine efficacy; Use of Integrated paired whole-exome sequencing (WES) framework optimized for cancer vaccine applications; NGS-based cTMB and clonal neoantigen profiling platform to enable precise, identification of vaccine targets and supports patient selection in clinical trials of therapeutic cancer vaccines.
Dr. Qiang Xu, Vice President Global Genomics Services, Frontage

Topic 12:

State-of-the-art qPCR & ddPCR Cellular Kinetics Strategy for Novel CAR-T Therapies in Cancer Treatment; Advanced Molecular Assay approaches to evaluate  growth/persistence of CAR-T cell in circulation by evaluating pros/cons of PCR technologies; Optimization of Lymphodepletion pretreatment to minimize impact on readout and increase consistency across patients; Lesson learned from measuring cellular kinetics of Cell Therapies using different types of PCR reagents; Overcoming ddPCR method development/validation challenges applying innovative approaches to Increase Sensitivity requirements.
Ms. Tracy Iles, Global Head of Method Development Molecular BioAnalysis, Labcorp

2026 White Paper on Gene, Cell, and Vaccine Therapies Immunogenicity & Technologies

Consensus & Conclusions on Gene, Cell, and Vaccine Therapies Immunogenicity & Technologies for 2026 White Paper

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

Regulatory Panelists:

• Dr. Zuben Sauna (US FDA)
• Dr. Vijaya Simhadri (US FDA)
• Ms. Leslie Wagner (US FDA)
• Dr. Surender Khurana (US FDA)
• Dr. Joshua Xu (US FDA)
• Dr. Mohanraj Manangeeswaran (US FDA)
• Dr. Seth Thacker (US FDA)
• Dr. Shirley Hopper (UK MHRA)
• Dr. Anna Nowocin (UK MHRA)
• Mr. Christian Mayer (Austria AGES/ EU EMA)
• Dr. Ingrid Schellens (Dutch MEB / EU EMA)
• Dr. Omar Tounekti (Health Canada)
• Dr. Chad Irwin (Health Canada)
• Dr. Sarah Cummings (Health Canada)
• Dr. Huixin Lu (Health Canada)
• Dr. Adrian Wong (Health Canada)