In the Zone: AAV characterization and analysis for gene therapy

Gene therapy is transforming how we treat genetic disorders, offering unprecedented hope for patients with conditions like spinal muscular atrophy and hemophilia. Adeno-associated viruses (AAVs) have become the leading vectors for gene delivery, thanks to their strong biosafety profile, low immunogenicity, and ability to enable long-term gene expression in target tissues.

But bringing these life-changing therapies from lab to patient requires comprehensive analytical testing to ensure AAV gene therapies are safe, effective and consistent. From understanding different types of viral titers to measuring capsid interactions with target cells, discover how advanced characterization methods like qPCR, Bio-Layer Interferometry, and HPLC are accelerating the path to regulatory approval and ultimately, to patients who need these breakthrough treatments.

[Video by James Harvie]

INFOGRAPHIC

AAV characterization: the what, why and how

This infographic maps the complete AAV development journey from the initial discovery of disease-causing genetic mutations to the final characterization of therapeutic vectors for clinical use. Explore how cutting-edge analytical platforms identify therapeutic targets, how specialized cell lines are engineered to produce high-quality AAV particles, and why rigorous purification and characterization steps are essential for patient safety.

INTERVIEW

Expanding the AAV analytical toolkit for gene therapies

AAV vectors have revolutionized gene therapy, however, the journey from laboratory concept to clinical application presents numerous challenges that must be overcome to ensure safety, efficacy and consistency. In this interview, we spoke with Stefano Boi to learn how some of these challenges can be addressed, with a focus on analytical methods, quality control, and process optimization.

RESOURCES

Unveiling the secrets of AAVs: novel high-throughput approaches for quantification
Rapid and non-disruptive AAV capsid ratio determination in complex biological matrices

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