21% Of CGT Clinical Holds Stem From CMC Issues. What Does This Tell Us?

Cell and gene therapy (CGT) trials account for nearly 40% of all clinical holds reported by the NIH, with over 21% caused by CMC issues, often delaying approvals by over 6 months.

A major contributing factor? Potency assay challenges.

Potency testing is among the most complex CMC requirements. Clinical holds related to potency assay issues can cause severe delays and financial setbacks, making early assay development a critical factor in avoiding these costly interruptions.

Our white paper explores the complexities of potency assay development, underscoring the FDA’s recommendation for early initiation as part of the Investigational New Drug (IND) process to allow adequate time for testing, refinement, and regulatory alignment. Given the technical hurdles and unique requirements of CGTs, a well-defined strategy that begins early can significantly reduce the risk of clinical holds, enhance product quality, and ensure smoother progression through regulatory milestones.