ANALYTICAL REGULATORY FOCUS DAY

TUESDAY, MAY 25 2021

SESSION 1

10:10 am An introduction to an evolving regulatory landscape in the context of gene therapy analytical development

  • Barbara Bonamassa Quality Assessor & European Medicines Agency (EMA) Expert for ATMPs, Italian Medicines Agency (AIFA)

Synopsis

  • Current regulatory guidelines
  • Current trends in gene therapy analytical development: A regulators perspective
  • Expectations for future guidelines

10:40 am Panel: Assessing Common Analytical Pitfalls faced by the Industry

  • Cecilia Della Vale Associate Director Analytical Science, PTC Therapeutics
  • Barbara Bonamassa Quality Assessor & European Medicines Agency (EMA) Expert for ATMPs, Italian Medicines Agency (AIFA)
  • Anne Douar Chief Development Officer , Vivet Therapeutics

Synopsis

  • Addressing common challenges and errors seen by regulators
  • With a lack of detailed guidance, what is needed from a regulatory perspective?

SESSION 2

Regulatory Requirements for Analytics Depending on the Product’s Stage in Development

Synopsis

  • Hear different regulatory experts from industry and agencies share their opinions on the fields most important challenges at each
  • Stage of development with deep dive panel discussions focusing on the following areas.

11:10 am Panel: Pre-Clinical Regulatory Challenges

Synopsis

  • Preclinical Safety Evaluation
  • The vector/construct characteristics desired biological effects of the transgene(s)

11:40 am Panel: Clinical Development & Beyond Regulatory Challenges

Synopsis

  • Producing relevant potency assay data
  • The use of alternative infectivity assays
  • When to engage regulators regarding the analytic plan
  • An insight into the analytical data required for approval
  • Sharing common challenges seen with submitted data and comments on improvement

11:40 am Panel: CMC Regulatory Challenges

Synopsis

  • Length of the storage conditions, GMP status of the storage facilities • Process Controls and Validation
  • Appropriate product characterization and process definition to ensure robust comparability data.
  • Raw Material Qualification: Qualification processes and data for key starting materials
  • Analytical Test Methods

12:40 pm Lunch

1:40 pm Round Table Discussion

2:40 pm Round Table Feedback

3:00 pm End of Day