Conference Day Two - Wednesday | October 30, 2024
8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
Utilizing Lessons Learned from Commercialized Therapies to Accelerate Clinical Products to Approval
9:00 am Sharing Lessons Learned After Approval to Drive Gene Therapy Products Towards Approval
Synopsis
- Presenting a case study on the EU and US registration of an ex-vivo LVV-based gene therapy for the treatment of a rare neurodegenerative disease
- Identifying areas for improvement and optimizing efficiency by recommending hurdles to strategically plan for
- Summarizing key lessons learned during the approval process and sharing insights
Supercharging Regulatory CMC by Overcoming Challenges to Ensure Safe & Efficacious Advanced Therapies
9:30 am Panel Discussion: Discussing Comparability Guidance & Gaining Insights on Negotiating with Regulatory Bodies
Synopsis
- Understanding regulatory expectations for comparability protocols to ensure alignment with criteria
- Providing examples of negotiating comparability strategies with regulatory bodies to apply these approaches in real-world scenarios
10:15 am Morning Break & Networking
Diving into Potency Assay Regulation to Uncover Hurdles in the Latest Regulatory Guidance
11:15 am Exploring Potency Assay Regulation to Ensure Clarity in Developing Efficacious & Guideline-Compliant Assays
Synopsis
- Distinguishing cell and gene therapy potency assays from other therapies to contextualize and provide high-level insights into associated challenges
- Addressing common regulatory pitfalls in potency assays to benchmark best practices for overcoming bottlenecks
- Showcasing examples of successful potency assays that comply with guidelines
11:45 am Roundtable Discussion: Analyzing the Most Recent FDA Draft Guidance on Potency Assays to Navigate Differences & Obstacles
Synopsis
- Comparing the newly released draft guidance with previous versions
- Removing uncertainties stemming from the draft guidance to determine clear next steps
- Discussing strategies for updating and aligning potency assay techniques with the new guidance
Taking a Global Perspective in Regulatory CMC to Discuss How Raw Materials, Batch Failures, & Platform Technologies are Regulated Across the World
12:35 pm Lunch Break & Networking
1:30 pm Roundtable Discussion: Addressing Batch Failures & Benchmarking Regulatory Proceedings in Such Scenarios
Synopsis
- Exploring global procedures for handling batch failures to enhance efficiency in overcoming challenges
- Improving communication among sponsors, regulators, sites, and patients in the case of a batch failure to streamline dialogue
- Sharing case examples of batch failure and how it was managed effectively
2:00 pm Panel Discussion: Exploring Recent Guidance on Platform Technologies to Clarify Frameworks for Approaches Used Across the Cell & Gene Field
Synopsis
- Considering what should be included on a platform basis or potentially excluded from a CMC, non-clinical, or procedural basis
- Enhancing the developmental process and efficiency by understanding the versatility of platform technologies and clarifying guidance on approaches
- Leveraging standardized systems to expedite the production of several cell and gene therapy products
2:45 pm Discovering Material Challenges to Untangle Regulatory Advice to Gain a Global Perspective
Synopsis
- Highlighting the regulatory requirements and strategies for the materials used in cell and gene manufacturing, from raw and starting materials, to gene editing components.
- Spotlighting the differences in material guidelines in Europe & other regions to enable smooth applications for approvals globally