Conference Day Two | Thursday, December 3
8:00 am Check In & Coffee
8:55 am Chair’s Opening Remarks
Global Alignment in Motion: Navigating Divergent Regulatory Frameworks for Strategic Advantage
9:00 am Panel Discussion: Navigating Global Regulatory Expectations for Cell & Gene Therapy Development
Synopsis
As cell and gene therapy programs become increasingly global, developers must align with diverse regulatory frameworks across regions. This session explores what’s required to successfully advance programs through non-clinical, CMC, and clinical milestones in markets such as Europe, Asia-Pacific, and Latin America.
- Understanding regional differences in CTA requirements for advanced therapies
- Preparing CMC and comparability packages for international regulatory review
- Leveraging accelerated pathways and early engagement opportunities outside the U.S.
- Opportunities for global harmonization and early agency engagement in advanced therapy programs
10:00 am Evolving CMC Expectations for Cell & Gene Therapies: From Initial Filing to Post-Approval Change
Synopsis
- Highlighting Health Canada’s approach to comparability and post-approval manufacturing changes
- Addressing the challenges of scaling CMC data from early-phase to BLA for complex gene therapy platforms
- Sharing insights on dossier readiness, common deficiencies, and opportunities for regulatory efficiency
10:30 am Morning Break & Networking
11:30 am Orthogonal Methods for Empty/Full AAV Characterization: Enabling Regulatory Confidence and Lifecycle Management
Synopsis
- Discuss the need for “regulatory confidence” in complex modality analytics. FDA (and other agencies) want reliable, orthogonal data for product quality attributes
- Discuss lessons learned from validating orthogonal methods (AUC, SEC-MALS, mass photometry, CD-MS, UV-Vis), and how the scientific community (including USP) approaches method selection, orthogonality, and dispute resolution
- Discuss harmonization efforts through CDMO working group on AAV methods
12:00 pm Establishing Regulatory Pathways for Cell & Gene Therapies in Brazil
Synopsis
- An overview of Brazil’s current regulatory framework for clinical trial and quality submissions, based on the speakers’ interpretation of publicly available guidelines
- Reflections on risk-based evaluation approaches, including considerations for comparability and raw material control in ATMP development
- Observations on international regulatory convergence and where future opportunities for alignment may exist
12:30 pm Lunch Break & Networking
Building Scalable & Compliant Manufacturing with Regulatory Confidence
1:30 pm Navigating Global Comparability Expectations in Cell & Gene Therapy CMC Development
Synopsis
- Harmonizing comparability approaches: contrasting FDA, EMA, and agencies such as Health Canada, ANVISA (Brazil), and CDE (China) to understand their impact on CMC strategy
- Risk-based frameworks for comparability assessment: leveraging analytical tools, potency assays, and totality-of-evidence to manage complex modality changes
- Proactive regulatory engagement: best practices for early discussions, submission planning, and addressing post-approval manufacturing evolution
Learning Through Experience: Mitigating Risk & Elevating Clinical Strategy in CGT
2:00 pm Leveraging Prior Knowledge to Streamline Process Validation & Post- Approval Specifications in Cell & Gene Therapies
Synopsis
- Using platform data and small-scale processes to reduce process validation batch requirements during tech transfer
- Addressing escalating post-approval regulatory demands with strategic comparability and potency management
- Optimizing commercial product specifications through real-world batch data linked to safety and efficacy in autologous therapies
2:30 pm Afternoon Break & Networking
3:30 pm From Bench to IND: De-Risking the Regulatory Journey for Cell & Gene Therapies
Synopsis
- Building a Scalable Regulatory Strategy Early: How to align CMC, preclinical, and clinical planning from day one to avoid costly delays at IND submission
- Common Pitfalls & Lessons from First-Time Filers: Real-world examples of what slows – or sinks – CGT INDs, from poor comparability planning to underpowered preclinical data
- Smart Use of Regulatory Interactions: How to make the most of Type B meetings, INTERACT meetings, and other FDA pathways to shape a clear and confident path to first-in-human trials
4:00 pm Regulatory Horizon Scan 2025: What’s Next for Cell & Gene Therapy Oversight
Synopsis
- Emerging Modalities Under Regulatory Watch: Identifying novel platforms drawing agency attention – including in vivo gene editing, programmable viral vectors, engineered iPSCs, and AI-driven design tools – and what early expectations are surfacing across agencies like EMA, FDA, and MHRA
- Forthcoming Guidance & Policy Shifts: Anticipated areas for new or revised regulatory guidance in 2025–2026, such as multiplex editing standards, next-gen potency assays, and decentralized manufacturing models
- Coordination Gaps & Global Signals: Where regulatory divergence is likely to widen (e.g., APAC vs. EU/US on platform designations or digital tools), and how developers can engage in proactive alignment and influence policy evolution through horizon scan engagement