POTENCY ASSAY FOCUS DAY

8:30 am Developing Appropriate Reference Standard Materials for Potency Assays to Enhance Batch Comparability

Synopsis

2h Interactive Deep-Dive

  • Discussing strategies for developing, characterising, and qualifying in-house primary reference material and working reference materials
  • Qualifying methods for ensuring batch-batch comparability and titre consistency.
  • Establishing reference standard materials for both AAV serotypes and lentiviruses to enhance lab to lab reproducibility
  • Brainstorming the challenges of bridging new reference standards during potency assay development

10:30 am Morning Break & Networking

11:00 am (CASE STUDY) Implementing a Tailored Potency Assay Strategy to Ensure GMP Compliance & Overcome Regulatory Concerns

Synopsis

  • Discussing the optimisation of cell lines to reduce background protein expression and ensure GMP compliance
  • Building novel cell models to improve potency assay efficiency and reduce costs
  • Divulging methods to ensure a potency strategy remains robust through to endpoint analysis
  • Implementing appropriate statistical tests for batch-batch comparability

12:00 pm Lunch

1:00 pm PANEL DISCUSSION: Discussing Strategies to Correlate the Outputs of a Potency Matrix Approach to Manage Regulatory Expectations

  • Uditha DeAlwis Vice President, Analytical Development and Quality Control, Sarepta Therapeutics
  • Louise McCormick Principal Scientist, BioMarin
  • Tony Bou Kheir Head of Analytical Development and Quality Control, Purespring Therapeutics
  • Rajeev Boregowda Associate Director, Bioassay & Molecular Analytical Development, Sanofi

Synopsis

  • Exploring the potency matrix and understanding the relevance of the constituent assays
  • Aligning measures of infectivity, mRNA, protein, and biological activity to generate robust potency assay data
  • Understanding correction factors and correlation coalitions in potency assay read-outs
  • Upper and lower limits: discussing regulatory perspectives on infectivity titres

2:00 pm Assay Optimisation Throughout the Early to Late-Stage Transition to Facilitate Phase-Appropriate Potency Analysis

Synopsis

  • Using potency matrices throughout early and late-stage potency assay design
  • Understanding how to use MoAs to select potency assay methods during early clinical development
  • Formulating robust validation protocols during potency assay upscaling

3:00 pm Interactive session: How Do we Best Measure Potency? Discussing the Implementation of Appropriate Statistical Tests & Success Criteria To Meet Regulatory Expectations

  • Rajeev Boregowda Associate Director, Bioassay & Molecular Analytical Development, Sanofi

Synopsis

  • Determining appropriate analytical methods for measuring product expression
  • Implementing model-specific system suitable criteria to assess potency
  • Understanding how to use appropriate statistical tests (line vs sigmoid analysis) when designing a potency strategy to competently assess comparability