Revolutionizing Treatment: How Nanoparticle Platforms Are Unlocking the Potential of Genetic Medicine

Genetic medicine is advancing rapidly, offering the potential to treat and even cure complex diseases through modalities such as antisense oligonucleic acids (ASOs) and CRISPR-based gene editing. However, progress in delivery technologies has lagged, largely due to limited throughput in screening new delivery vehicles. Battelle’s HIT SCAN™ platform (High Throughput Synthesis, Characterization, and Assessment of Nanoparticles) addresses this gap by enabling researchers to screen hundreds of polymer-based delivery candidates simultaneously, rather than just a few at a time.

Traditional delivery methods like lipid nanoparticles (LNPs) and adeno-associated viruses (AAVs) face limitations in navigating biological complexities. Polymeric nanoparticles (PNPs) have emerged as a promising alternative, offering improved payload protection, capacity, and targeting specificity. They can be engineered for diverse genetic materials and therapeutic goals, providing greater adaptability than LNPs.

Regulatory pathways are also evolving to support these innovations. The FDA’s Platform Technology Designation Program recognizes PNPs as platform technologies rather than single-use components, allowing developers to leverage existing data to accelerate approvals. This approach facilitates “combination products” that integrate drug and device, streamlining regulatory review and reducing time to market.

HIT SCAN enables rapid, automated synthesis and evaluation of PNPs, identifying optimal carriers for specific payloads and delivery sites. This high-throughput approach reduces costs, increases efficiency, and accelerates patient access to critical therapies.

With ongoing research expanding PNP capabilities, these carriers are poised to transform genetic medicine by enabling more targeted, efficient, and safe delivery systems—bringing the next generation of genetic therapies closer to widespread clinical use.