Explore the Agenda
8:30 am Check in & Light Breakfast
8:50 am Chair’s Opening Remarks
Improving Analytical Development Efficiency & Confidence in Decision Making Through Better Use of Advanced Data, Modelling & AI
9:00 am Using Large-Scale Single-Cell Data & Virtual Cell Models to Accelerate & De-Risk Analytical Assays
- Using whole-transcriptome single-cell profiling to reveal cell-state distributions and off-target populations that bulk and targeted assays miss
- Applying single-cell readouts and virtual cell models to detect subtle state shifts after process changes and strengthen analytical comparability
- Using virtual cell models trained on large-scale perturbation data to prioritize which conditions to test, as decision support upstream of validation
9:30 am Roundtable Discussion: Adopting AI Effectively in Analytical Development to Reduce Unnecessary Cost & Complexity
- Exploring where AI genuinely reduces workload versus adding validation burden, cost, or review time
- Using historical data responsibly to support credible AI-assisted decisions, including where the GxP boundary sits and what documentation is required either side of it
- Comparing how large and small organizations are adopting AI differently, and what each can learn from the other’s constraints
10:15 am Session reserved for Agilent
10:45 am Morning Break & Networking
Cell Therapy Track
Supporting Better Functionality & Potency Decisions Through Mechanistically Relevant Analytical Approaches
11:45 am Improving Potency Decision Making When Data Does Not Clearly Correlate Across Development Stages
- Examining gaps between in vitro preclinical and clinical CAR-T data to understand where potency signals break down
- Establishing assay specification and standardizing potency assay qualification
- Interpreting imperfect potency data to make more confident development and release decisions
12:15 pm Advancing Mechanistically Relevant Potency Assessment Through Functional Disease Models
- Navigating updated guidance for mechanistically relevant potency assays
- Addressing the challenge of measuring potency for cell therapies with multifactorial mechanisms of action
- Deconstructing Mechanism of Action (MoA) via disease modeling
- Translating biology into quantifiable bioassays using new approach methodologies (NAMs)
Gene Therapy Track
Advancing Gene Therapy Potency Testing Through Robust & Release Ready Analytical Approaches
11:45 am Development of Multiplex qPCR Platform Technology for Gene Therapy Product Potency Assessment
- Transition approaches of a dual-target AAV gene therapy potency assay from two separate reactions to a single multiplex PCR
- Channels independence validation and establishing proper controls
- Analytical performance qualification
12:15 pm Bridging Biology to Quantitation: A Roadmap to Meaningful Potency Measurement For AAV-Based Gene Therapy
- Critical considerations for AAV gene therapies include cell line selection, culture and transduction conditions, plate configuration, sample concentration & dilution strategies, assay controls, and data analysis approaches.
- In this presentation, we will describe the development of a robust potency assay for a novel AAV9-like viral vector.
- The presentation will highlight assay development, data analysis strategies, qualification outcomes, and the demonstration of stability-indicating properties, providing a practical roadmap for developing a potency assay suitable for drug release and stability studies.
12:45 pm Lunch Break & Networking
Cell Therapy Track
Reducing Variability & Strengthening Product Quality Through Better Understanding of Starting Materials & Raw Materials
1:45 pm Leveraging Transcriptomic Approaches to Support Cell Therapy Characterization and Critical Quality Attribute Identification
- Leveraging transcriptomic technologies, including qPCR, bulk RNA sequencing, and single-cell RNA sequencing, to characterize cell therapy products
- Applying transcriptional profiling to evaluate process-related differences, enhance product understanding, and support identification of candidate critical quality attributes
- Translating transcriptomic insights into analytical strategies that strengthen product characterization and support development and lifecycle decision-making
2:15 pm Applying Genome Editing to Xenotransplantation to Advance Human Compatible Organ Solutions
- Investigating how genome editing is being used to make porcine organs compatible with humans to give you insight into one of the most pioneering clinical translation applications in the field.
- Mapping the regulatory & safety considerations specific to xenotransplantation programs to help you understand the unique challenges of translating genome editing into organ compatibility.
- Highlighting real-world progress in xenotransplantation to equip you with a broader perspective on how genome editing is expanding the boundaries of therapeutic application
Gene Therapy Track
Maintaining Confidence in AAV Quality Assessment as Product Complexity & Analytical Requirements Continue to Evolve
1:45 pm Roundtable discussion: Improving Assay Transferability Through Stronger QC & Analytical Development Alignment
- Establishing early-stage QC criteria that support downstream development and IND readiness
- Aligning assay design, SOP development, and analytical requirements across teams
- Reducing variability by improving AAV quality standards, supplier oversight, and method transferability
2:15 pm Characterization of Host Cell DNA Impurities in AAV Products: A Product Specific Qualification Method & DNA Fragment Size Assessment
Details TBA
2:45 pm Afternoon Break & Networking
Enabling Successful Analytical Method Transfer & QC Readiness Through Robust Assay Development and Strategic Planning Across the Product Lifecycle
3:45 pm Panel Discussion: Navigating Analytical Method Development & Tech Transfer to Support Successful Cell & Gene Therapy Programs
- Sharing lessons from analytical method development across cell and gene therapy programs
- Exploring approaches to transferring analytical methods across internal teams and external partners
- Discussing considerations for supporting regulatory submissions through effective analytical strategies
4:15 pm Improving LNP Potency Assay Robustness to Enable Reliable QC Transfer
- Understanding regulatory expectations shaping potency assay design and validation
- Anticipating transfer requirements when developing mRNA LNP assays for future QC
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- Comparing base and targeted LNP approaches to inform robust assay development