Establishing Reproducible Polymer Nanoparticle (PNP) Manufacturing

Polymer nanoparticles (PNPs) hold transformative potential for gene therapies by overcoming delivery challenges faced by traditional adeno-associated viruses (AAVs) and lipid nanoparticles (LNPs). With their large payload capacity (50+ kilobases), low immunogenicity, active targeting, and shelf stability, PNPs offer a versatile, cost-effective alternative for delivering genetic material, proteins, or drugs to specific cells or tissues. However, inconsistent manufacturing and lack of reproducibility have hindered clinical adoption.

Battelle’s HIT SCAN™ platform addresses these challenges by combining high-throughput synthesis, biological characterization, and machine learning to create reproducible, scalable PNP solutions. Using RAFT polymerization, HIT SCAN™ automates polymer library synthesis, ensuring precise, consistent particle properties. Advanced screening techniques identify high-performing candidates quickly, while machine learning predicts performance and optimizes designs. This iterative, AI-enabled process reduces variability and enables seamless scale-up for clinical use.

HIT SCAN™ demonstrated significant improvements in reproducibility and consistency, as presented at the 2024 Controlled Release Society Annual Meeting. Key advances included tighter control over polymer composition and improved biodistribution insights, paving the way for tailored PNP therapies.

Unlike AAVs and LNPs, PNPs unlock infinite design possibilities, allowing adjustments in size, charge, or degradation rates to enhance stability, targeting, and controlled drug release. With HIT SCAN™, Battelle is advancing non-viral delivery technologies, making PNP-based therapies a viable solution for treating genetic disorders, cancers, and previously untreatable conditions. This innovation positions PNPs to revolutionize gene therapy, providing safer, scalable, and more affordable treatment options.