A multitude of cellular therapy candidates are quickly progressing to clinical stage manufacturing. Advances in automated processing (e.g., Prodigy TCT) have substantially reduced the development efforts needed to support a GMP-compliant manufacturing strategy. However, many therapeutics are delayed getting into the clinic due to lack of suitable analytical methods at an appropriate level of quality.
While some release tests such as sterility, mycoplasma, and endotoxin are standardized via compendial methods, most cellular therapies entering the clinic are characterized using a suite of complex and often bespoke assays. Flow cytometry is the most widely used modality in cell therapy, employed to determine incoming product phenotype and composition for in-process control, as well as to evaluate cellular identity and purity of final drug products for release testing and as “for-information-only” (FIO) characterization. RT-PCR is used to determine vector copy number, as well as surrogate assays to detect the presence of replication competent viral vectors. In addition, functionality assays are typically expected as part of final product FIO studies in early phase, with the expectation at BLA filing of a fully validated characterization suite.
A combination of release testing and FIO characterization are also employed in early phase development for in-use and long-term storage studies as part of product stability qualification.
Miltenyi Biotec is now focusing efforts towards standardization and automation of analytical characterization technologies on many of the techniques used, which will allow a more streamlined development paradigm, and ultimately, a faster path to clinical and approval.
Attendees will hone their understanding of: