Conference Day One | Wednesday, December 3
8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
9:00 am Panel Discussion: From First Filing to Final Approval: Inside the Regulatory Journey of Cell & Gene Therapies
Synopsis
- Key inflection points, setbacks, and accelerators across IND, BLA, and global filings
- Building productive relationships with regulators across regions – what worked, and what didn’t?
- Managing internal alignment and external expectations during late-stage development and approval
Achieving Lifecycle Excellence & Managing Post-Approval Changes to Ensure Continuous Compliance and Market Success
10:00 am Wins & Lessons Learned from FDA BLA Submission & CRL Issuance, Through to BLA Approval of a Gene-Corrected Cell Therapy
Synopsis
- Presenting a case study on US registration of an autologous RVV-based gene-corrected cell therapy for treatment of an ultra-rare, life-threatening skin disorder
- Evaluating the dossier for major risks and how to mitigate these
- Sharing wins and lessons gained during BLA preparation, FDA review, and approval
10:45 am Morning Break & Speed Networking
Synopsis
Reconnect with fellow CGT regulatory professionals to share insights, discuss challenges, and build lasting connections in this dedicated networking session.
11:45 am Round Table Discussion: Managing Significant Change & Post-Approval Optimization in Cell & Gene Therapy
Synopsis
- Clarify what constitutes a “significant change” and how to meet global regulatory expectations with risk-based data strategies
- Understand why post-approval optimization is critical for CGT products, including manufacturing and assay refinements
- Learn from real-world case studies on balancing innovation with regulatory compliance throughout the product lifecycle
Exploring Future Modalities & New Regulatory Territory While Advancing Innovation & Navigating Emerging Challenges
12:15 pm Navigating Regulatory Pathways for iPSC-Derived Cell Therapies in Neurology
Synopsis
- Regulatory considerations unique to iPSC-derived allogeneic therapies for CNS indications
- Regulatory considerations of management of investigational device for cell therapy administration
- Opportunities for accelerated pathway
12:45 pm Lunch Break & Networking
1:45 pm Real-World Use of AI in Cell & Gene Therapy Regulatory Affairs: From Efficiency Gains to Risk-Aware Applications
Synopsis
- Practical examples of how AI is being used today to draft regulatory documents, streamline publishing, and manage submissions
- Identifying “low-risk” use cases for AI in regulatory processes through a patient safety lens
- Lessons learned from implementing AI tools in regulatory and clinical workflows: what’s working and what’s not?
Realigning with the Regulators: What the FDA’s Next Chapter Means for CGT Development
2:15 pm New Tools, Old Questions: Applying FDA Initiatives & Evolving CMC Expectations in CGT
Synopsis
- How companies can best leverage meetings with FDA in cell and gene therapy development
- Practical implications for submission planning, regulatory flexibility, and engagement tools such as INTERACT and Type D meetings
- Uses for FDA-led programs such as Project FrontRunner, the Commissioner’s National Priority Voucher program, and RMAT designation
- What’s changing in CMC expectations: comparability, potency methods, and analytical characterization
2:45 pm Afternoon Break & Networking
3:45 pm Live Insights Lab: Navigating FDA & CBER Leadership Changes Impacting CGT Development
Synopsis
Join this interactive session to reflect on a year of FDA changes, explore their impact, and collaborate in small groups to craft key questions and discussion points, then watch them come to life in a dynamic panel conversation.
- Shifts in trial design expectations: from single-arm studies to randomized controlled trials and innovative alternatives
- Evolving CMC and analytical requirements: balancing regulatory rigor with manufacturing realities
- Changing agency interactions, global harmonization challenges, and the growing role of real-world evidence and patient engagement