Standardizing material management in cell and gene therapy: A consensus framework for quality, risk, and regulatory alignment

Cell and gene therapies (CGTs) represent a paradigm shift in modern medicine, yet their journey from discovery to commercialization is fraught with challenges that extend beyond scientific innovation. Early-stage designs often overlook critical factors such as scalability, regulatory compliance, and material quality, leading to costly delays and rework. This presentation explores a holistic framework for integrating commercial viability, quality assurance, and regulatory strategy into therapeutic design from the outset.

Key takeaways

• Quality by Design (QbD) principles for raw material qualification and lifecycle management.
• Harmonizing global regulatory expectations (FDA cGMP, ISO 13485, USP <1043>) to streamline compliance.
• Risk mitigation strategies for ancillary materials and single-use systems, addressing sterility, traceability, and performance claims.
• Practical approaches to supplier qualification and documentation that reduce filing times and enhance operational agility.