Accelerate Gene Therapy with Precision AAV Transfection Analysis

Gene therapy, which delivers DNA into a patient’s cells to replace or add genes that may be abnormal or missing, is starting to deliver results in the clinic after 30+ years of development.  With the FDA approving five new gene therapies in 2023, it is truly an exciting time in the field.  

Among available delivery methods, adeno-associated virus (AAV) has emerged as a leading vector for gene therapy due to its broad tropism, inability to replicate independently in vivo, minimal immunogenicity, and long-lasting effectiveness. As adoption grows, the demand for accurate gene therapy analysis and real-time monitoring of AAV transfection and transduction is increasing; helping ensure therapeutic dose accuracy, quality, safety, and reproducibility in manufacturing.

There are several challenges related to the development and manufacturing of AAV-based processes, including characterization, quantification, and downstream purification. Manufacturing challenges in particular include the need for the consistent production of high purity, high potency, and robust safety for AAV products, all while maintaining acceptable large-scale manufacturing costs.  

Multiple approaches exist for AAV production, including transient AAV triple transfection in human (HEK293) cells, baculovirus-driven production in insect (Sf9) cells, or recombinant helper viruses such as HSV in mammalian cells. Regardless of the system, the lack of real-time, predictive gene therapy analysis makes it difficult to ensure reproducibility, dose accuracy, and regulatory compliance, underscoring the need for advanced analytical tools to guide gene therapy process development.

Radiance®, utilizing the advanced Laser Force Cytology™ (LFC) technology, addresses analytical challenges in gene therapy development. As a Process Analytical Technology (PAT), it serves as an in-process monitoring tool for AAV, adenovirus, and other gene therapy vectors.

With rapid, label-free single-cell analysis, Radiance® enables real-time monitoring of cell health, AAV transfection efficiency, and viral vector production, eliminating reliance on slow, labor-intensive offline assays. LFC™ also delivers highly precise measurement of AAV transduction (without the need for a helper virus) and viral titers, offering a faster, more reproducible, and scalable alternative to traditional assays like TCID50. This real-time insight ensures therapeutic dose accuracy, product quality, and manufacturing consistency, helping accelerate gene therapy process development from research to clinical production.