Topic 1:

Novel/Innovative approaches/strategies for PK, ADA and NAb Assays supporting Masked Cytokines; Clinical PK measurement of prodrug, active, and mask using IA-MS; Reagent strategy used to deliver on sub-ng/mL clinical sensitivity requirements using anti-peptide antibodies; Investigation performed to evaluate if ADA to the masked portion of the cytokine were able to be detected in the Tier 1 screening assay of the prodrug bridging ADA assay; Evolution of the Clinical Immunogenicity Strategy for the masked cytokine project; Importance of early Regulators' engagement.

Topic 2:

Challenges of Detecting ADA for Therapeutic Proteins with High Preexisting Antibodies; Critical aspects on immunogenicity evaluation and ADA assays validation; Regulators' requests to be addressed; Considerations in Establishing 5% false positive rate for therapeutic proteins with high preexisting antibodies; Use of 3-tier immunogenicity testing and alternative statistical approach to establish Screening CP and Reprocessing based on 2019 US FDA Guidance and reduction in % of preexisting & treatment emergent ADA. 

Topic 3:

How to overcome the Limitations of Developing ADA assays with non-representative Positive Controls (PC)? How to better understand what really generates an assay signal in ADA assay and better interpret results? Is it possible develop ADA assay that enable Quantitative ADA Detection and provide ADA concentrations? What possibilities would a Quantitative ADA Detection provide to better understand ADA Clinical Relevance? What are the alternatives to statistically-based Cut Point determination? 

Topic 4:

Understanding Patient Selection Strategies triggering need of Companion Diagnostic (CDx) to identify patients with Preexisting Immunity to AAV Capsids or with Specific Genetic Variants; Regulatory implications to consider in order to ensure successful Regulatory Approvals & patients' administration of Gene Therapies; What are key characteristics of GT requiring careful selection of patients during clinical trials and after regulatory approval? CDx Regulatory Implications from development to post-approval to ensure that GT can effectively reach patient populations with high unmet medical needs.

Topic 5:

Rapid Data Collection Benefits of using Established Diagnostic Assays (IVD) in supporting Exploratory PD Biomarker Endpoints versus limitations on Incomplete Assay Performance Characteristics to fully evaluate if assay is fit for intended use and with no information on assay metrics during sample analysis; If an IVD or CLIA assay is repurposed for an exploratory PD biomarker endpoint without further validation, what approaches may be used to ensure that the use of the diagnostic assay is suitable to support exploratory PD endpoints? 

Topic 6:

Biomarker Development Strategy for Targeted Protein Degradation with Molecular Glue clinical programs; Method Development & Validation of Multiple Technologies/Assays (Flow Cytometry, LBA, MSA) to monitor direct target degradation, downstream pharmacodynamic changes and treatment responses; Utility of Clinical Biomarkers in supporting clinical trials for Molecular Glue Degraders for small-molecule drugs to Target Undruggable Proteins as a novel approach to treat various autoimmune, inflammatory, oncological and neurological diseases

Topic 7:

Selection of Vaccine Biomarker Assays (LBA & CBA/Flow Cytometry) to align with immunological mechanisms of protection, capturing both Humoral and Cellular Responses for robust Correlate of Protection (CoP) identification; Using CBA to provide mechanistic insights linking Immune Activity to clinical protection endpoints and Multiplexed/High Throughput LBA to measure Immune Biomarkers; Standardization, cross-laboratory harmonization and validated reference standards critical for Comparability and Regulatory Acceptance of CoP defining bioanalytical data. 

Topic 8:

Current standards for the Interpretation of Predose Positivity observed in single-chain variable fragment (scFv) from CAR-T Therapies due to e presence of engineered domain structures interacting differently with endogenous serum components compared to traditional biologics; Effective mitigation strategies considering unique structural features, interfering factors, and propensity for nonspecific background signals; Careful evaluation of design/structure of scFv molecules used for capture/detection to minimize cross-reactivity and ensure specificity, which in turn reduces false positives and enhances overall assay performance.

Topic 9:

Unravelling the complexity of Cell-based Assays (CBA)/Biofunctional Assays due to complex multicomponent assays with multiple sources of variation; Dilutional Linearity as key performance parameter assessed during CBA Validation measured in heterogenous mix of antibodies in serum; Requirements for appropriate matrix as diluent for Virus Neutralization Serum-based Activity; Importance of Reference Standards implementations in CBA: True Reference, Normalizer, Cross-laboratory comparison and how the use of reference standards impacted Assay Performance.

Topic 10:

Advanced application of NGS Platform to differentiate hMPV subtypes using Amplicon and Whole Genome Sequencing methods; Evaluating RSV/hMPV mRNA Vaccine Efficacy using NGS; Limitations of GenMark assay to identify hMPV subtypes; Integration the digital workflow with the Bioinformatics Pipeline across multiple cloud platforms, implementation of a user-accessible interface for testing and executing the full end-to-end pipeline; Validation to evaluate key performance metrics, accuracy, precision, sensitivity, and specificity. 

Topic 11:

Newest experience on Novel CRISPR/Cas9 Gene Editing therapeutic development from non-clinical to late clinical stages; Monkeys to Patients PK/PD Translation; Build primary and secondary PD tests to link drug potency and PD effect to clinical endpoints; Demonstrate durability of PD effect and Clinical Benefit to patients after a single dose CRISPR/Cas9 gene editing therapy; Focus on PD Biomarker Assays and their suitability to better understand drug MoA and Disease Pathology.

Topic 12:

Lost in the Triangle of AAV-based Gene Therapy Bioanalysis: Immunogenicity against recombinant AAV capsid/transgene, Vector biodistribution/exposure/shedding, Transgene expression and Biomarkers; State-of-the-art technologies, expertise, alternative approaches; Analysis of rAAV exposure by a Novel Ultra-sensitive Capsid Quantification compared with the biodistribution by dPCR; Application of generic Anti-AAV assay to analyze anti-capsid immune responses on vector biodistribution; Correlation of Anti-transgene protein response with transgene expression/exposure and Target Engagement biomarkers.

Topic 13:

Latest strategies for High-quality & Reliable Molecular/Genomic Assays (PCR & NGS) for producing cost-effective, high-impact data in Gene & Cell Therapies development; Maximizing Bioanalytical Sensitivity & Specificity, minimizing sample/reagent use, ensuring reproducible results across operators/sites; AI/ML-driven Assay Design, validation experiments optimization, failures prediction; DoE Simulation, LoD and variance modelling, samples prioritization via active learning, QC thresholds and protocol generation; AI/ML impact on Tissue Use Reduction, improved robustness, accelerated validated deployment.

2026 White Paper on Ligand-Binding, Cell-Based, and Molecular Assays

Consensus & Conclusions on Ligand-Binding, Cell-Based, and Molecular Assays for 2026 White Paper