Process Performance Qualification

Overview

Regulatory requirement for biologics manufacturing demonstrating a process consistently produces product meeting its specifications; the FDA mandates prospective PPQ for gene therapy manufacturing components, and Prime Medicine pioneered 'PPQ-as-you-go' to align validation with clinical timelines. Process performance qualification (PPQ) is a CMC bottleneck for gene therapy programs because the FDA mandates prospective — not retrospective — validation of mRNA and guide RNA manufacturing processes even when material amounts needed are small and existing inventory may be adequate. Prime Medicine and Aldevron developed 'PPQ-as-you-go': making the first manufacturing lot under prospective PPQ conditions within approximately 2.5-3 months by explicitly designating in protocols which validation activities could be deferred to later without compromising compliance. (Segment 5 (20:00-25:00) and Segment 6 (25:00-30:00))