Explore the Agenda
8:30 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
Balancing Innovations in Novel Vector Technologies with QC & Manufacturing Readiness
9:00 am Manufacturing & QC Considerations for TfR1-Targeted AAV Capsids: Balancing Innovation with Process Robustness
- Identifying key bottlenecks in purification and how adjustments in chromatography and filtration improve recovery and yield
- Evaluating the role of advanced analytical methods in confirming identity and consistency
- Sharing lessons on establishing platform QC methods that remain predictive and reproducible when applied to engineered capsids
9:30 am Re-Engineering AAV9 to Minimize Off Target Effects & Improve Efficacy
- Discussing techniques to maintain process robustness across diverse capsids and payloads
- Highlighting Affinia’s use of single-use clarification, affinity purification and UAC to achieve full capsid enrichment to ensure batch consistency and regulatory confidence
- Presenting lessons from the transfer of AFTX-201 high yield process to a CDMO partner, including reproducibility of yield and capsid quality across sites
10:00 am Advancing Viral Vector Manufacturing: NGS-Driven Solutions for Manufacturing Robustness, Product Safety & Regulatory Compliance
- Addressing analytical gaps in viral vector identity, genome integrity, impurity profiles and batch comparability
- Evaluating the role of sequencing in identifying truncated genomes and sequence integrity that impact potency and safety
- Discussing how NGS can be adopted in cGMP environments to meet identity, purity, safety, and comparability requirements
10:30 am Morning Break
Industrializing Vector Supply: Meeting the Demands of Global Cell & Gene Therapy Commercialization
11:00 am Fireside Chat: From Clinical Supply to Commercial Launch: Ensuring PPQ Readiness & BLA Success in Viral Vector Manufacturing
- Outlining what regulators expect from PPQ runs in viral vector manufacturing: consistency, reproducibility, and demonstration of process control across multiple consecutive batches
- Sharing strategies for scaling processes developed at early-stage to commercialready platforms without compromising quality or timelines
11:30 am The Clock is Ticking: Meeting Regulatory Burdens in a Patient-Centred Timeframe
- Reviewing how to design GMP-compliant viral vectors tailored for a single patient’s treatment needs
- Discussing strategies for releasing viral vectors in <3 weeks for single-patient treatments
- Anticipate scaling up processes from n=1 vectors to 20-40 patient batches
12:00 pm Roundtable Discussion: Triple Transfection, Transient Systems, or Stable Cell Lines – Which Pathway Best Supports Commercialization?
- Comparing triple transfection and transient systems for their ability to rapidly support early-stage programs, while evaluating their scalability limits at clinical and commercial scales
- Exploring how plasmid-heavy approaches create supply challenges at large scale, and whether dual plasmid systems or stable cell lines can reduce dependency on GMP plasmid supply
- Debating which transfection or cell line strategy best supports PPQ runs, regulatory alignment, and robust COGs to ensure long-term viability of gene therapies
12:45 pm Lunch Break & Networking
Reducing Losses in Downstream: How to Recover More Product Without Raising Costs
1:45 pm Roundtable: Discussing Strategies to Design Potency Assays Earlier in The Pipeline to De-risk IND & Clinical Trial Readiness
- Discussing how to integrate potency assay design into early R&D so that assays are relevant, reproducible, and fit for regulatory expectations at IND stage
- Evaluating how early potency assay choices reduce risk of late-stage delays, bridging studies, or regulatory feedback loops
- Reviewing emerging tools, including ddPCR-based functional assays, FACS, in vitro surrogate models, that allow faster and more reliable potency readouts
Advancing Global Regulatory Harmonization for Viral Vector Manufacturing
2:15 pm Overcoming Global Regulatory Misalignment to Increase Efficiency in Viral Vector Manufacturing
- Clarifying definitions of raw materials, starting materials, and ancillary components to enable global alignment and consistent quality expectations
- Discussing how industry and regulators can collaborate through pharmacopeial channels to anticipate evolving expectations
- Highlighting gaps and inconsistencies across regional guidance and how standardsetting organizations can help bridge interpretation differences
2:45 pm Aligning Post-Approval Change Management Across FDA, EMA & PMDA
- How ICHQ12 provides a shared foundation for post-approval change management
- Comparison of post-approval variations framework across the FDA, EMA and PMDA
- Adoption of risk-based CMC review models across the three agencies