Potency Assay Development Guide for Cell and Gene Therapy Products
Potency assays are among the most challenging analytical methods to develop for CGT products. As regulatory expectations continue to evolve, developers must establish assays that not only measure biological activity but also demonstrate consistency, product quality, and readiness for clinical and commercial release. Pharmaron’s guide to potency assay development, now in its 3rd edition, walks through the progression for AAV gene therapies and CAR-T cell therapies.
Download this comprehensive guide to learn:
- How potency assay expectations evolve from preclinical development through commercialization
- The difference between expression-based and functional potency assays
- Strategies for implementing a potency assurance framework aligned with regulatory guidance
- Best practices for assay qualification, validation, and GMP readiness
- Approaches for AAV, gene-modified cell therapy, and CAR-T potency testing
- Common challenges that can delay development and how to overcome them
- Key considerations from ICH Q2(R2) validation requirements
- Real-world case studies demonstrating potency assay development and validation approaches
Inside the guide:
Potency Assurance Strategies
Explore how potency testing fits within an overall potency assurance framework and learn how assay requirements evolve throughout development. The guide discusses recent regulatory thinking and practical approaches for maintaining potency throughout a product’s lifecycle.
Roadmap for Potency Assay Development
Learn how developers progress from early proof-of-concept testing to qualified and validated assays suitable for clinical and commercial release.
Functional Potency Assays for Gene and Cell Therapies
Capabilities
Gain insights into developing assays for:
- AAV gene therapies
- Viral and non-viral gene delivery platforms
- CAR-T and other engineered cell therapies
- Transgene expression and biological activity assessment
Validation and Regulatory Readiness
Understand the critical validation parameters outlined in ICH Q2(R2), including accuracy, precision, specificity, linearity, range, robustness, and system suitability.
Case Studies
The first is the validated potency assay for Luxturna, published in Molecular Therapy Methods & Clinical Development with co-authors from Pharmaron’s Exton, Pennsylvania site. The second looks at CAR-T activation and cytotoxicity measured on several analytical platforms. Complete the form to download the guide.
Accelerate Potency Assay Development with Confidence
Whether you’re establishing an early-stage potency strategy, preparing for clinical development, or advancing toward BLA submission, this guide provides practical insights to help navigate one of the most critical aspects of cell and gene therapy product development. Download the eBook to learn how a well-designed potency assay strategy can support regulatory success and streamline commercialization.
Learn about AAV and CAR-T assay design tools, an ICH Q2(R2) validation summary and two case studies.
References
1. FDA. Potency Tests for CGT Products (2011).
2. FDA. Potency Assurance for CGT Products, draft (2023).
3. FDA. Q2(R2) Validation of Analytical Procedures (ICH).
4. High KA, et al. Validation of a cell-based relative potency assay for LUXTURNA. Mol Ther Methods Clin Dev. 2025;33(1):101423.