Potency Assays: The 'Bugaboo' of Stem Cell Therapy

Area of Science:

• Biotechnology
• Pharmacology
• Regulatory Science

Background:

• Cell-based products for human use are regulated as medicines, necessitating thorough characterization.
• Potency is a critical quality attribute that must reflect the intended mechanism of action and biological effect.
• Defining and validating potency assays is challenging due to poorly understood mechanisms of action.

Purpose of the Study:

• To outline strategies for demonstrating the potency of cell- and gene-based therapies.
• To discuss the regulatory framework governing potency testing.
• To highlight the need for standardization in potency assay development.

Main Methods:

• Review of current strategies employed by developers of cell- and gene-based therapies.
• Analysis of the regulatory landscape for innovative medicines.
• Discussion of challenges and considerations in designing potency assays.

Main Results:

• Identified common approaches used by developers to establish potency for novel cell and gene therapies.
• Highlighted regulatory expectations and challenges in potency assay validation.
• Emphasized the importance of assay standardization for ensuring product quality and efficacy.

Conclusions:

• Potency assessment is a critical yet complex aspect of cell- and gene-therapy development.
• Standardized and well-defined potency assays are essential for regulatory approval and patient safety.
• Further research and collaboration are needed to refine potency testing methodologies.