Karen Buckland

Ex vivo gene-modified cell therapies present analytical challenges that differ fundamentally from viral vector-focused workflows. With each manufacturing run being patient-specific and starting material scarce, analytical teams must balance biological relevance, QC robustness, regulatory expectations, and feasibility.

This workshop uses a real paediatric gene‑modified cell therapy case study to show how a hospital‑based GMP team upgraded critical assays, completed concurrent validation under significant constraints, and managed QC risks for a first‑in‑child clinical programme. Building on this example, attendees will collaborate on core principles for designing assays that remain fit for purpose as programmes advance to later phases.

What attendees will explore through the case study:

• Evolving release assays from research grade to QC-suitable platforms, improving clinical decision-making confidence
• Designing and executing concurrent validation when conventional validation is impossible; how to justify risk and anticipate regulatory expectations in ultra rare or paediatric programmes
• Aligning manufacturing, QC and regulatory communication for complex ATMPS by reviewing how regulatory interactions were managed around assay changes, validation pathways, and manufacturing constraints, and how analytical teams can pre-empt regulator concerns