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.

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.

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?

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.

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.

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.

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.

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?

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?

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.

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.

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.

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

Panel Discussion with All the Regulators:

Have an Open Dialogue with the Regulators including US FDA, EU EMA, UK MHRA, Austria AGES/ EU EMA, Health Canada
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