Tackling CMC and Manufacturing Challenges of Complex LNP-based Biopharmaceuticals for Oncological, Gene Editing and Vaccine Applications

Join this interactive workshop with hands-on demonstrations to learn and discuss about nanoparticle (LNPs) manufacturing and CMC challenges. LNPs are the most established platform for delivery of nucleic acid payloads. Their tunability and streamlined manufacturing facilitated an unprecedentedly rapid scale-up during the COVID-19 pandemic. However, being multicomponent, complex systems also poses a challenge of controlling their stability, quality and safety. Emerging novel LNP formulations often include complex mixtures of different cargoes which require a different approach to manufacturing and characterization. Such formulations, possibly also with surface modifications, are powerful for advanced therapies and active targeting, but additionally increase the complexity of CMC and manufacturing. Next generation orthogonal methods for analytics and purification methods are needed to address these challenges.
CIM® (convective interaction media) monolithic columns offer a unique solution for intact chromatography of large biomolecules. Such shear-less process is ideal for unstable assemblies like LNPs. Advanced chromatographic platforms based on this technology can address some of these CMC challenges, using multiple analytical columns and multiple detectors, operating without sample pre-treatment. Multiple cargo formulations, like those in use for CAR-T therapies, CRISPR gene editing and combination vaccines require precise analytical handling. This involves separating the cargos, a process effectively accomplished through chromatography. On larger scales, efficient purification of large biomolecules is achieved using monoliths, excelling in high recovery and integrity of purified molecules. This approach enhances the correlation between in vitro characteristics and therapeutic efficacy, accelerating LNP-based therapeutics and vaccines.