Accelerating Gene Therapy With The HIT SCAN Platform

Battelle’s HIT SCAN™ (High Throughput Synthesis, Characterization and Assessment of Nanoparticles) platform is revolutionizing gene therapy by tackling one of its biggest challenges: delivery. While approximately 7,000 rare genetic diseases affect 25–30 million Americans, only about 500 of these conditions currently have approved treatments. The HIT SCAN platform was developed to accelerate the discovery and development of safe, effective delivery vehicles for these therapies—especially for the majority of patients who still lack treatment options.

Unlike viral vectors, polymer nanoparticles (PNPs) offer several advantages, including lower toxicity, better immunogenicity profiles, shelf stability without cold storage, and the potential for repeated application. They also have a high payload capacity (up to 20 kilobases), can deliver a variety of payloads (RNA, DNA, proteins), and allow for targeting beyond the liver.

HIT SCAN leverages automated polymer synthesis via RAFT (reversible addition-fragmentation chain transfer) polymerization and high-throughput robotic screening to create and test thousands of PNP candidates. Machine learning is then applied to predict and optimize physical traits, bioactivity, and in vivo biodistribution. This learn-by-iteration approach enables the rapid identification of ideal nanoparticles tailored for specific therapeutic needs.

The platform’s comprehensive workflow—design, build, test, and learn—creates a powerful feedback loop that dramatically speeds up the discovery process. With its versatility, scalability, and efficiency, HIT SCAN represents a transformative solution in the quest to bring gene therapy to more patients suffering from rare and underserved genetic disorders.