Molecular therapies are providing new hope to patients with genetic diseases, but they also present new challenges for process development and control. The sheer size and complexity of products like pDNA, mRNA and AAV present a new challenge for “at-line” analytics, like for example, distinguishing sc/oc/lin pDNA or empty from full AAV capsids. At BIA Separations, we are passionate about providing the best solutions for gene therapy purification, by providing a platform for analysis and in-process control of both upstream and downstream processing steps, using our PATfix HPLC platform.
To achieve this, we have built our platform on 4 key pillars:
CIMac™ analytical columns, are a perfect tool for chromatography of large gene therapy molecules due to convective laminar flow, a large accessible surface area required for binding of GT products and a variety of surface chemistries. These in turn allow us to tailor the PATfix platform for best results
Featuring bio-inert ceramic pump heads, and flow rates up to 10 mL/min (quaternary) or 50 mL/min (binary), PATfix is designed to manage the analytical parameters for a wide range of biological products. A highly sensitive, multi-wavelength UV detector, as well as a fluorescence detector, capture component peaks, even with the steepest gradients. The binary version is designed for easy transfer of analytical insights to semi-prep chromo steps, simplifying scale-up.
PATfix is powered by PATfix software which offers an elaborate, yet user-friendly toolbox for the automated analysis of chromatographic data sets, while providing rapid feedback to help you make critical process control decisions. PATfix delivers the speed, power, and robustness required for process analytical technology (PAT) in the development and production of molecular therapies.
Its not just about the individual building blocks, its knowing exactly which blocks are needed and how they need to work together to achieve their stated goal – provide analytical insight at every step of gene therapy process development and production. Gaining this level of insight requires a lot of trial and error over years to perfect. We have developed and transferred methods for both analytics and production of gene therapeutics to many customers. The experience gained on such a variety of products and projects has allowed us to develop a set of chromatographic methods, that can encompass a variety of products and can be applied to key steps of PD and production.