ARPA-H selects teams to make made-to-order genetic medicine manufacturing possible

Five teams will produce automated, distributed biomanufacturing technology that doesn't currently exist — designed to increase access to genetic medicines for all Americans. 

Today the Advanced Research Projects Agency for Health (ARPA-H) announced the teams receiving contract awards from its Genetic Medicines and Individualized Manufacturing for Everyone (GIVE) program. GIVE is driving toward a new manufacturing paradigm: an automated network capable of producing individualized, ribonucleic acid (RNA)-based genetic medicines on demand, and replacing a slow, costly, centralized model with one created for speed, scale, and reach. ARPA-H will fund up to $125 million for the research and development program. 

Genetic medicines require centralized, specialized production that is traditionally slow and expensive, and cold-chain logistics keep patients waiting or without access altogether. GIVE is designed to close that gap by developing automated manufacturing and real-time quality control into a distributed biomanufacturing network, so treatment can be made close to the patient, empowering communities everywhere — not just those near major biomanufacturing hubs — to affordably produce this kind of cutting-edge treatment. 

“Individualized domestic biomanufacturing will give the United States an advantage and ensure that all patients have access to the latest therapeutic capabilities, no matter where they live,” said GIVE Program Manager John Schiel, Ph.D. “The breakthrough manufacturing and quality control technologies needed to enable these therapies to reach patients at scale doesn’t exist yet. This investment will catalyze domestic innovation for distributed manufacturing of RNA-based genetic medicines — and these teams will deliver it in a matter of years, not decades.” 

GIVE is establishing a domestic biomanufacturing platform that builds American supply chain and will meaningfully increase access for patients with cancer, rare genetic conditions, and chronic diseases. If successful, this effort will dramatically reduce costs and expand access to all communities nationwide. 

TA1 + TA2: Automated, Distributed Manufacturing Platforms with Integrated Quality Control 

These teams will create automated platforms capable of producing RNA-based genetic medicines and performing quality control testing at or near the point of care. 

• Centillion Biosciences Inc. – The team will develop a unified, end-to-end system combining cartridge-based technologies for DNA/RNA manufacturing, integrated fill, finish and quality control testing, and digital process management. It delivers scalable manufacturing (from personalized to large-scale), first-of-its kind measurement technologies, and the data-handling capabilities distributed RNA production requires.  
• HDT Bio Corp. – The team will develop a platform with an integrated architecture that combines automated DNA synthesis, chip-based RNA production, closed fill and finish, embedded process analytical technologies, and autonomous, robotics enabled quality control testing. Manufacturing and quality functions both operate inside a controlled enclosure with compliant electronic batch records.  
• Massachusetts General Hospital – The team will develop a single-use, all fluidics chip system and control unit hardware capable of continuous manufacturing and quality-control testing. By using a closed fluidic path, the system eliminates the need for open-air liquid handling and a cleanroom enclosure, representing a compact, efficient (<3 day), and cost-effective approach to advance distributed manufacturing with minimal hardware investment. 
• Waterfall Scientific – The team will develop a fully automated, benchtop, continuous-flow system for end-to-end production of RNA drug products, combining proprietary technologies from a consortium of expert organizations. Its core comprises four integrated, modular units: Cell-Free Enzymatic DNA Production, Continuous-Flow RNA Synthesis, Lipid Nanoparticle (LNP) Formulation, and Fill-Finish. The team will also develop an integrated quality control system for lot release, combining in-line, at-line and off-line characterization of drug product, fully autonomous and automated using proprietary software, robotics and the most advanced measurement technologies available. 

TA2: Integrated Quality Control 

This team will develop automated quality control systems that validate identity, purity, and potency in real time across distributed manufacturing sites. 

• University of Utah – The team will develop an all-in-one genetic medicine testing platform enabling full QC testing and lot release within as little as one day. Using digital microfluidics, advanced optics, and artificial intelligence, the platform will perform multi-parametric characterization of therapeutics at nanoliter- to microliter-scales in a compact, single-use cartridges format. 

GIVE will engage the U.S. Food and Drug Administration (FDA) throughout the program to co-develop the regulatory framework needed to bring distributed, individualized genetic medicine manufacturing to scale. 

“The proposed rule to modernize drug manufacturing reflects FDA’s recognition that the future of drug manufacturing is distributed, and our regulatory framework needs to evolve alongside it,” Karim Mikhail, B. Pharm., M.S., Acting Director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Programs like GIVE that are building the underlying technology for multi-site, individualized biomanufacturing are exactly the kind of innovation this framework is designed to support. We look forward to continuing to work with ARPA-H as this field matures.” 

Personalized treatments like Baby KJ’s revolutionary CRISPR cure currently take months to manufacture, cost hundreds of thousands of dollars, and require years of clinical trials to prove safe and effective. If GIVE succeeds, these therapies could be made locally and on demand, in a fraction of the time and cost. Faster manufacturing could, in turn, help trials move more efficiently. Patients with rare genetic conditions, cancers, and chronic diseases would gain faster access to medicine built around their own biology — wherever they live. 

For more on GIVE, visit the program page.