Navigating Next-Generation Quality Control Strategies For AAV Testing

Adeno-associated viruses (AAVs) are central to gene therapy, demanding rigorous quality control for safe and effective use. Critical testing methodologies underpin AAV-based therapeutics, ensuring robust process and product controls meet evolving regulatory standards.

Infectivity, a key quality attribute, is precisely measured using the TCID50 assay, now enhanced with molecular endpoints for accuracy and adaptability. Next-generation sequencing (NGS) identifies low-level variants and ensures product identity and purity, at both plasmid and final product stages, aligning with quality by design principles. Viral clearance studies are essential for mitigating contamination risks, evaluating downstream purification effectiveness, and complying with ICH Q5A(R2) guidelines. Potency assays—expression, functional bioassays, and relative potency assays—quantify biological activity, ensuring consistency and quality.

Integrating these advanced tools empowers manufacturers to navigate regulatory requirements, optimize processes, and deliver life-changing AAV-based therapies. Continuous refinement and adaptation of these methodologies are crucial for maintaining the highest safety and efficacy standards in gene therapy.