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Bridging bench to bioprocess for extrahepatic LNP gene delivery

04 Nov 2026
Gene Therapy Manufacturing
  • Scale up for In Vivo Extrahepatic Targeting

As we move beyond liver-targeted therapies to extrahepatic tissues via novel surface ligands or specialized formulations, how do we balance complex bioconjugation strategies with the need for simplified, reproducible downstream purification and scalable operations?

  • Translating from Lab to Clinical Manufacturing: What's Missing?

When moving a promising non-viral formulation from benchtop research into development and manufacturing, where are the biggest gaps in current equipment, standardized analytics, or regulatory guidance?

When scaling lipid nanoparticle (LNP) or polymer-based formulations from bench-scale microfluidics to commercial-scale continuous processing, how are you maintaining particle size distribution, encapsulation efficiency, and critical quality attributes (CQAs) without compromising throughput?

  • Expanding Beyond Standard LNPs

As the field pushes beyond standard LNP formulations, what alternative non-viral platforms—such as polymeric nanoparticles, extracellular vesicles/exosomes, or hybrid lipid-polymer systems, or sonoporation—show the most promise for clinical translation and tissue-specific delivery?

  • One-Shot Encapsulation vs. Post-Loading Strategies

When evaluating one-shot simultaneous formulation against post-loading nucleic acids into preformed empty LNPs, how to balance payload stability, encapsulation efficiencies, and analytics?

Industry Expert
Pedro J Cejas, Senior Director - Discovery, Delivery Platforms - Roche