REGULATORY FOCUS DAY - Wednesday, October 30, 2024

8:30 am Check-In & Coffee

9:00 am PANEL: When Should We Start Measuring Potency? Navigating Potency Measurement & Assay Setup Considerations

Synopsis

  • Understanding the implications of the new FDA draft potency guidance on the necessity and implications of potency assays in phase 1 trials
  • Determining best practice for optimizing controls, reference standards, and AAV serotype selection to establish robust potency assays that satisfy regulatory demands
  • Overcoming limited batch size to develop appropriate documentation and address gaps in CMC packages for potency assays

10:00 am Mediating Effective Communication Between QC & R&D Teams to Ensure Successful IND Submissions

Synopsis

  • Understand the role of the QC team in developing, qualifying, and validating assays received from R&D
  • Discussing how to effectively work with CMC teams to draft appropriate documentation for regulatory filings
  • What is required during the application review process?

11:00 am Networking Break & Refreshments

12:00 pm COLLABORATIVE SESSION: Sharing Stories About the Implementation of the New Potency Guidance

  • David Schwalb Director - Analytical Development Cell and Gene Therapy, Pacira Pharmaceuticals Inc
  • Natalie Palmer-Deverill Senior Scientist, MSAT Analytical Cell Development, MeiraGTx

Synopsis

A series of mini case studies outlining the implementation of the new draft FDA guidance into analytical strategies. Attend for discussions and recommendations on how the new guidelines should be interpreted and implemented to guide novel assay design approaches.

1:00 pm Lunch & Networking

2:00 pm Voluntary Consensus Standards & Their Use in the Regulatory Process

  • Katie Zander Director, Standards Education & Outreach, Standards Coordinating Body

Synopsis

• What are voluntary consensus standards, their benefits, and how to get involved

• How voluntary consensus standards fit into the regulatory process

• Understanding the FDA CBER standards recognition program

2:45 pm ROUNDTABLE: Accelerating Cell-Based Bioassays for Robust Safety Assessment that Meets Regulatory Demands

  • Kashyap Pandya Associate Director - Analytical, Chemistry, Manufacturing, Controls & Gene Therapy, PTC Therapeutics

Synopsis

  • Introducing strategies to expedite the replication competent AAV qPCR assay timeline without compromising sensitivity
  • Brainstorming techniques for overcoming detection limitations, increase the sensitivity of the assay and, enhance the detection of replication competent AAV
  • Understanding regulatory guidelines surrounding rcAAV and residual WT AD5 safety testing to guide method development and implementation
  • Determining the emerging role of NGS/ICHQ5a in safety testing: how likely is this to become an industry standard?

3:45 pm Close of Regulatory Focus Day