Leveraging Novel Analytical Approaches For Advanced Therapies

The characterization and analysis of advanced therapies, such as cell and gene therapies (CGTs) can be difficult, as these products are designed to function using complex mechanisms of action (MOA)s. There are a wide range of challenges associated with accurately assessing the potency and impurity profiles of these complex biologicals. As many CGT programs qualify for accelerated review pathways, novel approaches for analysis and characterization can help generate data that allows for real-time decision making and faster development timelines.

Catalent has developed a relative potency bioassay using quantitative polymerase chain reaction (qPCR) to assess relative transcription activity in cells treated with ligands or transgenic vectors. The assay platform can be used to qualify a repeatable, accurate, linear, and specific bioassay for assessing relative potency for CGTs, mRNA- and other nucleic acid-based therapies.

Review the advantages and limitations of transcriptional assays versus reporter gene assays and how the transcriptional assay compares to ddPCR and flow cytometry for analysis of cell and gene therapies.