Accelerating Gene Therapy With HITSCAN Platform

Gene therapy holds life-changing potential for the approximately 25–30 million Americans affected by one of over 7,000 rare genetic diseases—yet only about 500 of these diseases have approved treatments. A key barrier to progress is the effective delivery of therapeutic payloads. Battelle’s HIT SCAN™ (High Throughput Synthesis, Characterization and Assessment of Nanoparticles) platform addresses this by rapidly designing, screening, and optimizing polymeric nanoparticle (PNP) delivery vehicles.

HIT SCAN integrates automated polymer synthesis, high-throughput in vivo screening, and machine learning to identify the most effective drug delivery systems. This closed-loop platform follows a Design-Build-Test-Learn cycle: automated RAFT polymerization enables consistent production of diverse PNPs; robotic screening rapidly evaluates thousands of candidates; and predictive models guide the optimization process by analyzing biodistribution, bioactivity, and toxicity data.

Compared to traditional delivery vehicles like AAVs and LNPs, PNPs offer several advantages, including lower toxicity, greater compositional and payload diversity, repeat-dose potential, and no need for cold-chain storage. Additionally, PNPs demonstrate promise in extra-hepatic delivery—extending treatment options beyond the liver to other tissues.

With capabilities to handle large and complex payloads (up to 20kB), and support for RNA, DNA, and proteins, HIT SCAN is revolutionizing how non-viral gene therapies are developed. By combining high-throughput experimentation with AI-driven insights, Battelle’s platform significantly accelerates the path from concept to clinical application—unlocking new hope for millions of patients with rare diseases.