Nasser Sadr

Company: uniQure

Job title: Sr Director Global Analytical Development

Seminars:

The Road to Approval: Gene Therapy Analytical Package From Early IND to Approval 9:00 am

Lean release analytical package for the initiation of Phase 1/2 clinical trials: minimum requirements and common challenges Evolution of the analytical package through different stages until filing for marketing approval: extended vs characterization vs release Regulatory expectations at the time, of BLA/MAA approval: first hand, learnings from parallel filing in the US, and EURead more

day: Day One AM

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

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…Read more

day: Regulatory Focus Day