Designing a Gene Therapy CMC Architecture for the Full Product Lifecycle

Process Development and Contract Services Partner Decision-Making

While the gene therapy sector is growing rapidly and making significant strides toward fulfilling its promise of providing highly effective, often curative therapeutics for an expanding number of disease states, the industry is working to navigate numerous challenges.

For example, proof-of-concept and other early-phase work are commonly conducted in highly academic settings using manual techniques. Transitioning these approaches to streamlined, reproducible, scaled, and ultimately good manufacturing practice (GMP) processes can be challenging.

Additionally, gene therapy companies are often structured as virtual organizations, commonly with no built-in CMC teams at the early stage and with smaller CMC teams as they move through regulatory approval processes, late-phase development, and commercial manufacturing phases.

Currently, gene therapies have several unique qualities that may or may not change as the therapeutic modality becomes more conventional. For example, gene therapies are generally envisioned as one-time treatments, and today’s primary focus is rare diseases with low clinical numbers. Therefore, smaller commercial batch sizes are often appropriate. However, gene therapies are expanding into large patient population conditions, which could deliver new challenges such as scaling manufacturing, managing networks of point-of-care production capabilities, and reducing the cost of goods to make gene therapies more accessible.

This guide is designed to help gene therapy innovators gain a high-level perspective on the series of process development and contract services selection decisions to be made throughout the phases of development. Download the full guide here.

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