Notes on CMC for cell, gene, and RNA medicines - tech transfer, process validation, drug product, CDMO oversight, and regulatory writing, from someone who has done the work.
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How to run deviations, root cause investigations, CAPA effectiveness verification, and change control across a network of contract manufacturers, from what the quality agreement actually buys you to the metrics that reveal a partner's quality system drifting.
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How to run the technical assessment, capability, capacity, culture, equipment fit, analytical readiness, and data systems, that turns CDMO selection into a defensible technical agreement instead of a leap of faith.
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What INDs, NDAs, and BLAs actually require for process validation content, how to write S.2.5 and P.3.5 sections and PPQ summaries a reviewer can follow, and how to handle review-cycle questions and pre-approval inspection readiness.
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How to build a Stage 3 CPV program that catches process drift before it becomes an OOS, covering parameter selection, control charts versus capability indices, escalation rules, and right-sizing for batch volume.
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How to qualify the cold chain for cell and gene therapies: shipper types, shipping study design, real-time monitoring and alert escalation, reverse logistics, customs and import contingency planning, and how a temperature excursion actually gets investigated.
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How to build a sterilizing-grade filter validation package: bacterial retention studies with the manufacturer, product-specific versus matrix bracketing, the extractables handoff, routine integrity testing, and the traps that catch low-volume programs.
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How formulation logic shifts from proteins to oligonucleotides to cells to AAV vectors: stability drivers, excipient choices, custom cryoformulation development, mixing equipment selection, lyophilization cycle basics, and what changes at high concentration heading toward a prefilled syringe.
Ready-to-use documents
- Risk Assessment ALCOA+ Data Integrity Risk Assessment Worksheet
- Protocol Analytical Method Transfer Protocol
- Log Anticipated Regulatory Questions Log
- Checklist Automation and Control System Migration Verification Checklist
- Form Bioreactor and Unit Operation Scale Translation Worksheet
- Record CAPA Effectiveness Verification Record
- Risk Assessment CCIT Change Control and Revalidation Trigger Assessment
- Risk Assessment CCIT Method Selection and MALL Risk Assessment