Cell-Based Potency Assays Explained

What is a Phase-appropriate Potency Assay?

Phase appropriateness of a potency assay ensures that its scientific and regulatory rigor aligns with the clinical development phases. For more insights, listen to our Webinar on Destination BLA, First Stop IND!

Is a Gene Expression Assay Sufficient for Early-phase Clinical Trials?

While the FDA requires a Quality Target Product Profile (QTPP) with a mechanism of action (MOA), i.e., activity-based potency assay, gene expression assays are acceptable in the early stages if developing an activity assay is challenging. Regulatory agencies may sometimes require expression and activity assays for product approval.

Is the RT-qPCR Assay Acceptable as a Potency Readout?

Although recommended, a physiologically relevant cell line isn’t always necessary. A non-physiological cell line may suffice if the MOA can be determined in vitro. For example, standard cell lines like HEK293 can mimic the MOA for ocular products.

For early-phase clinical trials, mRNA-based expression assays are generally accepted when sensitivity or critical reagent availability is an issue. Co-developing an activity assay to correlate mRNA expression with transgene activity is recommended.

Offerings

How to Choose the Right Platform?

Select the analytical platform based on your target molecule type:
- RT-qPCR for mRNA
- ELISA, MSD, or Flow Cytometry for proteins
- LC-MS/MS for amino acids, lipids and hormones
Consider the platform’s sensitivity, robustness, reagent availability, throughput, and compliance software

What If an Activity Assay in vitro Is Not Feasible?

If in vitro activity isn’t measurable, explore unconventional assays like protein-protein interactions or cell viability. In vivo potency assays might also be an option.

Choosing the Right CRO/CDMO for Potency Assays

Select a service partner with a strong scientific team and a rigorous QA-controlled GMP team for assay validation and product testing for lot release.

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