The Advanced Research Projects Agency for Health has selected five teams for a program intended to develop automated, distributed manufacturing systems for individualized RNA-based genetic medicines, with up to $125 million available for research and development.
The Genetic Medicines and Individualized Manufacturing for Everyone, or GIVE, program seeks to replace centralized production models with technology that could manufacture genetic medicines closer to patients. The systems would combine medicine production, quality-control testing and digital process management at or near points of care.
ARPA-H said current genetic-medicine production is often specialized, centralized, costly and dependent on cold-chain logistics. The program aims to develop domestic manufacturing capabilities that could support patients with cancer, rare genetic disorders and chronic diseases.
Centillion Biosciences, HDT Bio Corp., Massachusetts General Hospital and Waterfall Scientific will develop automated distributed manufacturing platforms with integrated quality-control systems.
Centillion will develop a cartridge-based system for DNA and RNA manufacturing, fill-finish operations, quality testing and process management. HDT Bio plans an enclosed platform that combines automated DNA synthesis, chip-based RNA production, fill-finish processes and robotics-enabled quality testing.
Massachusetts General Hospital will develop a single-use fluidics-chip system designed for continuous manufacturing and quality testing in less than three days. The proposed closed-fluidic pathway would avoid open-air liquid handling and the need for a cleanroom enclosure.
Waterfall Scientific will develop a benchtop continuous-flow system incorporating cell-free enzymatic DNA production, RNA synthesis, lipid nanoparticle formulation and fill-finish operations. Its platform would also include automated systems for drug-product characterization and lot release.
The University of Utah will focus on standalone quality-control technology. Its proposed platform is designed to validate the identity, purity and potency of genetic medicines and enable lot release within as little as one day through digital microfluidics, advanced optics and artificial intelligence.
ARPA-H said it will work with the Food and Drug Administration during the program to help develop a regulatory framework for individualized, multi-site genetic-medicine manufacturing.
The agency said personalized genetic therapies can currently take months to manufacture and cost hundreds of thousands of dollars. GIVE is intended to reduce production time and cost while expanding patient access.