Pacing the Journey: A Framework to Commercial Readiness

Jey Bouc, Ph.D., and Keerthana Subramanian As gene therapy manufacturing continues to mature, the focus is shifting. Most gene therapy developers begin this work running a sprint, racing to reach first-in-human data as quickly as possible. But as programs advance toward commercialization, the discipline required starts to look less like a sprint and more like a marathon.

Early development once focused primarily on demonstrating that a vector could be produced reproducibly and at sufficient yield. Today, as programs advance toward pivotal studies and commercialization, the challenge shifts to a thorough, steady journey. Regulatory agencies no longer ask whether a product can be manufactured.

They ask whether the process and product are truly understood in a way that supports consistency and safety. Process characterization (PC) provides the scientific foundation needed to build the data set that enables developers to identify critical process parameters, establish robust manufacturing control strategies and demonstrate process understanding as programs advance toward commercialization. Like training for a marathon, PC is not about a single explosive effort; it’s the sustained, disciplined work, mile after mile, that builds the endurance a program needs to go the distance to commercial launch.

It transforms a process that operates reliably in the hands of its developers into one that can be justified, reproduced, and trusted by health authority reviewers. When executed well, PC reduces downstream risk by strengthening validation strategies, supporting PPQ readiness, and enabling a smoother path to commercial launch. When treated as both a regulatory requirement and a scientific exercise, it can become a foundational source for information requests during BLA or MAA review.

Within the development lifecycle, process characterization serves as the critical link between process development and commercial process validation: It is where the empirical, exploratory work of early development is formalized into a defensible control strategy, grounded