FDA

Overview

US regulatory agency that mandates prospective process performance qualification for gene therapy manufacturing components including mRNA and guide RNA; released a new draft guidance on gene therapy and gene editing on June 10, 2026. The FDA requires prospective rather than retrospective process performance qualification for mRNA and guide RNA materials used in gene therapy products, even when Prime Medicine argued these were critical reagents rather than drug substance and that existing inventory was sufficient. On approximately June 10, 2026 (the day before the summit session), the FDA released a new draft guidance for gene therapy including gene editing, addressing manufacturing validation requirements that align with the PPQ-as-you-go approach. (Segment 5 (20:00-25:00) and Segment 6 (25:00-30:00))