Gene Therapy

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.  AAV has proven to be a powerful tool for gene therapy purposes for its broad tropism, inability to replicate on its own in vivo, minimal immunogenicity, and ability to deliver effective and long-lasting results.

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.  While there are several methods for AAV production, including transient transfection in human (HEK293) cells, baculovirus driven production in insect (Sf9) cells, or recombinant helper viruses such as HSV in mammalian cells, the importance of precise and predictive analytics remains regardless of the production system.

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. Offering rapid, label-free single-cell analysis, Radiance® enables real-time data collection for monitoring cell health, transfection, and viral vector production, eliminating the need for time-consuming offline assays. LFC™ proves valuable for AAV transduction measurement, providing a precise and efficient alternative to traditional assays like TCID50 that are often slow, labor-intensive, and challenging to standardize.