Conference Day Two - Wednesday | October 30, 2024

Day One

Tuesday, October 29

Day Two

Wednesday, October 30

8:00 am Check-In & Coffee

8:50 am Chair’s Opening Remarks

  • Fubao Wang Senior Vice President, Head of Regulatory Affairs, Prime Medicine

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

  • David Uguen Senior Vice President & Head of Regulatory, Orchard Therapeutics

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

  • Annelise Brossel Director, Regulatory Affairs & Quality Assurance, Vivet Therapeutics
  • Seraphin Kuate Director, CMC, CAR-T Cell Therapy, Bristol Myers Squibb
  • Keith Wonnacott Vice President, Regulatory Affairs, Lexeo Therapeutics

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

  • Sagi Nahum Global Senior Director, Analytical Sciences CMC, Cell & Gene Therapy, Orgenesis

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11:45 am Fireside Chat: Analyzing the Most Recent FDA Draft Guidance on Potency Assays to Navigate Differences & Obstacles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  • Comparing the newly released draft guidance with previous versions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

12:30 pm Lunch Break & Networking

Taking a Global Perspective in Regulatory CMC to Discuss How Raw Materials, Batch Failures, & Platform Technologies are Regulated Across the World

1:30 pm Roundtable Discussion: Addressing Batch Failures & Benchmarking Regulatory Proceedings in Such Scenarios

  • Wade Atkins Regulatory Affairs Specialist, National Institute of Health

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

  • Fubao Wang Senior Vice President, Head of Regulatory Affairs, Prime Medicine
  • Monica Veldman Director, Global Regulatory Policy, Alliance for Regenerative Medicine
  • Shirley Bartido Director, Global Regulatory Affairs & Cell Therapy Oncology, Takeda Pharmaceuticals

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

3:15 pm Chair’s Closing Remarks

  • Fubao Wang Senior Vice President, Head of Regulatory Affairs, Prime Medicine

3:30 pm End of Conference

Conference Day One
See Full Agenda
Partner