Explore the Agenda
8:00 am Check in & Light Breakfast
8:50 am Chair’s Opening Remarks
Navigating Regulatory Expectations & Aligning Analytical Approaches to Support Long-Term Decision Making Before & After Approval
9:00 am Scientific Advisory Board Fireside Chat: Aligning on Future Analytical Standards to Reduce Risk & Improve Decision Making Across Cell & Gene Therapy
Meet the Scientific Advisory Board whose expertise has helped shape a highly relevant, industry focused agenda for this year’s meeting.
They will be exploring topics such as:
- Defining priorities that shift analytical development from support function to core risk management
- Identifying where challenges are similar and different across modalities and what that means for teams
- Clarifying practical gaps that still limit progress across analytical development and QC
10:00 am When Comparable Does Not Mean Equivalent: Designing Analytical Comparability Studies That Withstand Regulatory Scrutiny in ex vivo Lentiviral Gene Therapy
- Framing comparability as characterization, not equivalence, when a process change is expected to improve the product
- Anchoring acceptance criteria to the clinical therapeutic window rather than assay variability alone
- Separating dose-finding from formal assessment to keep pre-specification auditable under regulatory scrutiny; using Type C meetings to align before executing
10:30 am Morning Break & Speed Networking
Cell Therapy Track
Reducing Development Delays & Strengthening Commercial Readiness Through Smarter Analytical & Control Strategy Decisions
11:30 am Building A Control Strategy for Cell Therapies That Remains Robust from Phase 1 Through BLA to Minimize Costly Delays
- Evaluating when early characterization assays should be retained, removed or moved into release testing
- Refining specifications and acceptance criteria as development progresses across phases
- Aligning analytical decisions with evolving CMC and regulatory strategy on the path to BLA
12:00 pm Analytical Strategy & Lifecycle Execution Roadmap & Examples to Support Commercial Readiness & Operational Execution Success
- Using an enhanced analytical development program via appropriate analytical target profiles (ATP) and development staging opportunities to optimize analytical performance.
- Aligning analytical development, manufacturing, and QC controls strategy conditions.
- Pre-/post-commercial capacity expansion and validation considerations for QC operations for autologous cell therapies.
Gene Therapy Track
Keeping Analytical Strategies Fit-for- Purpose as Gene Therapy Programs Evolve & New Innovations Emerge
11:30 am Adapting Analytical Strategies to Keep Pace with AAV Complexity & Maintain Confidence
- Addressing how capsid and genome changes are affecting the reliability of existing assays
- Redesigning analytical and stability methods to reflect new product behaviors and risks
- Maintaining consistency in measurement to support confident decision making despite variability
12:00 pm Platform approach for AAV analytics
- Defining what constitutes a platform analytical method in AAV programs and identifying which assays and attributes can realistically be leveraged across products
- Establishing phase-appropriate strategies for applying platform methods, with clear expectations for method development, qualification, and validation as programs advance
- Creating practical criteria for method reuse, redevelopment, and revalidation to reduce redundant validation efforts and enable efficient, scalable analytical approaches across similar AAV therapies
12:30 pm Lunch Break & Networking
Cell Therapy Track
Strengthening Decision Making Around Quality, Safety & Regulatory Risk to Advance Access to Novel Cell Therapies
1:30 pm Confirming gene modifications on release using Flow Cytometry assays
- Measuring knock out knock in or knockdown expression as a functional way of confirming whether the gene modification occurred.
- Using PCR-based methods to assess genomic knock out or knock-in efficiency as an orthogonal measure on characterization.
- Reducing measurement of redundant attributes on release to prevent assay deviations in QC
2:00 pm Panel Discussion: Are We Measuring the Right Things? Defining Meaningful Flow Cytometry Readouts for Cell Therapy Development
- Identifying which attributes truly influence product quality and clinical performance
- Designing assay roadmaps that translate defined QTPPs and CQAs into fit for purpose analytical strategies
- Understanding where organisations still struggle to connect characterisation, potency, and clinical outcomes
2:30 pm Roundtable Discussion: Improving Consistency by Embedding Donor Selection into Early Autologous Cell Therapy Development
- Identifying donor driven variability to reducing inconsistency in assays and final product quality
- Using donor characterisation to predicting manufacturing performance earlier in development
- Aligning cross functional decisions to minimising rework failed batches and variability in release
Gene Therapy Track
Improving Confidence in AAV Safety & Performance by Advancing How Full to Empty Capsid Ratios Are Measured & Used
1:30 pm Optimizing AAV Product Understanding by Defining Meaningful Full to Empty Capsid Thresholds
- Establishing how full to empty capsid ratios impact gene and protein expression in a defined model system
- Identifying the threshold where expression begins to decline and how this informs analytical decision making
- Exploring how empty capsids may influence safety assessments through in vitro evaluation strategies
2:00 pm Improving AAV Product Quality by Looking Beyond Empty & Full Measurements
- Identifying how capsid integrity and DNA leakage provide additional quality insights
- Understanding why empty and full measurements alone can miss critical product attributes
- Evaluating how better formed viruses could reduce total viral load while maintaining activity
2:30 pm Session reserved for Catalent
3:00 pm Afternoon Break & Poster Session
Building Assays That Remain Robust, Transferable & Regulatory Ready to Reduce Costly Late-Stage Rework
4:00 pm Navigating Assay Re-Development & Transfer to Reduce Risk & Delays in GMP Testing
- Addressing challenges in transferring assays from research into GMP testing environments
- Managing redevelopment of complex assays and adapting to new readout platforms
- Aligning timelines and coordinating qualification across internal teams and CRO partners
4:30 pm Session reserved for SGS
4:40 pm Avoiding Late-stage Failure by Delivering Potency Assays That Meet Regulatory Expectations
- Prioritizing early development of functional potency assays to reduce late-stage risk and rework
- Demonstrating dose response and reproducibility to supporting smoother validation and approval
- Learning from case studies to improve assay robustness and reducing variability