FDA's New Gene Therapy Rule Book

6 chapters covering the updated FDA framework

From what changed in the guidance to exactly what FDA now expects at BLA — mapped to the avoidable hurdles that stall gene therapy programmes.

• Chapter 1 FDA's Updated Gene Therapy Guidance: What Changed and Why It Matters
• Chapter 2 Post-Market Performance Study Requirements: Scope and Triggers
• Chapter 3 RMAT Designation: Criteria, Application Strategy, and Common Pitfalls
• Chapter 4 IND Meeting Playbook: Pre-IND, Type B, and End-of-Phase Interactions
• Chapter 5 LTFU Requirements: Duration, Registry Design, and Reporting Thresholds
• Chapter 6 CMC Considerations in the Updated Guidance: What FDA Now Expects at BLA