Bioanalytical support for AAV therapies
As adeno-associated virus (AAV) therapies gain traction in the gene therapy market, the need for specialized bioanalytical support becomes increasingly critical. Bioanalysis in pre-clinical studies focuses on evaluating the biodistribution, expression levels and potential immunogenicity of the AAV vectors. Techniques like quantitative PCR, ELISA and immunohistochemistry are employed to track vector presence, transgene expression and immune response. These assessments are crucial for understanding the vector’s safety profile and therapeutic potential.
In pre-clinical safety assessment, we monitor adverse effects, gene expression and off-target impacts. This comprehensive analysis helps predict human responses and guides dose selection for clinical trials. Robust bioanalytical methods are essential for ensuring the safe and effective translation of AAV-mediated gene therapies from the laboratory to clinical applications.
In this Spotlight, we will explore the expanding role of bioanalysis in AAV therapies, focusing on biodistribution, transgene expression, immunogenicity, ELISpot and flow cytometry. With AAVs becoming more niche, there is a growing demand for detailed analysis to understand their safety profiles and their broader applications across different therapeutic areas. Our discussion will highlight the significant contributions of bioanalytical methods to the development and assessment of AAV therapies.
This feature was produced in association with:
