Current And New Technologies To Characterize Advanced Therapies: Making The Right Decisions Upstream To Ensure Downstream Success

By Avi Nandi, VP, Process Development, Brian Tomkowicz, Sr. Director, R&D, and Dana Cipriano, SVP, Testing & Analytical Services

The accurate characterization of cell and gene therapies is currently mission-critical for the industry. The past two years have seen a swathe of rejections, delays, and suspensions from the FDA because of CMC issues many driven by a lack of product characterization and insufficient understanding of mechanisms of action (MoA). In addition, cell therapies continue to diversify in terms of cell type, so additional development is required to characterize these new, unique functional characteristics effectively and consistently.

Mechanisms of action of many advanced therapies are, in general, poorly understood and assay design is not robust, therefore, developers are finding it hard to provide the proof that regulators need to approve products. The FDA has been increasingly calling for manufacturers to develop more consistent ways to measure product quality. To bridge this gap, developers need access to services and tools that allow for sensitive and specific assays, with the flexibility to determine quality, potency, and safety throughout the manufacturing process.

One approach to reducing the risk associated with such therapies is to build a manufacturing process that includes validated analytical procedures and assays to promote enhanced assay performance and process knowledge to ensure high-quality and consistent products.

This article will explore the latest strategies and technologies to develop a comprehensive, optimal approach to assay development and product characterization.