Topic 1:

Present challenges in trying to assess Preexisting Antibodies against Novel Capsid utilized for development of Heart Disease Gene Therapy programs due to its unique heart tropism; Impact of Preexisting Antibodies on the binding to Novel Capsid remains unclear; Importance of developing reliable LBA to evaluate anti-AAV capsid IgG seroprevalence in healthy donors as they can neutralize the vector or form immune complexes, compromising therapy efficacy and raising safety concerns.
Dr. Michael Franti, Director Viral Platforms, Boehringer Ingelheim

Topic 2:

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 3:

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 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 designed to broaden clinical indications while enhancing safety profiles and therapeutic efficacy through innovative engineering strategies; Essential Bioanalytical Assay Requirements for supporting the development and characterization of Complex Cell Therapies; Importance/challenges of Cellular Kinetic Data in non-blood matrices for evaluating tissue-specific persistence and its implications for safety and efficacy; Approaches and alternative strategies to overcome challenges in complex matrices to improve data quality, applicability, and reliability.
Dr. Nanda Balasubramanian, Associate Director Precision Medicine, Bioanalytical & Translational Science, Bristol Myers Squibb

Topic 11:

Sophisticated technologies used in the development of Next-Generation Vaccine Therapy; Innovative application of Molecular Assays used in design, development, monitoring of vaccines; Leveraging NGS for high-throughput molecular analysis to achieve greater speed, precision, efficacy in vaccinology; Application of NGS to allow for rapid/comprehensive genetic analysis of Pathogen Genome Analysis; Analyzing host B-cell & T-cell receptor (TCR) to understand how the immune system responds to a vaccine to identify Correlates of Protection (CoP); Applications of AI & Deep/Machine Learning for structure-guided antigen design and predicting optimal stabilizing mutations in viral proteins; How Molecular Assays are accelerating the development of new vaccine platforms, mRNA, DNA, and NP-based Vaccines.
Dr. Qiang Xu, Vice President Global Genomics Services, Frontage

Topic 12:

State-of-the-art qPCR & ddPCR Cellular Kinetics Strategy for Novel Cell 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 mevelopment/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 - invited)
• Dr. Vijaya Simhadri (US FDA - invited)
• Ms. Leslie Wagner (US FDA - invited)
• Dr. Surender Khurana (US FDA)
• Dr. Joshua Xu (US FDA)
• Dr. João Pedras-Vasconcelos (US FDA - invited)
• Dr. Mohanraj Manangeeswaran (US FDA - invited)
• Dr. Shirley Hopper (UK MHRA)
• Dr. Luke Lindsay-Hill (UK MHRA)
• Dr. Anna Nowocin (UK MHRA)
• Dr. Alessandra Buoninfante (EU EMA)
• Mr. Christian Mayer (Austria AGES/ 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)