ARPA-H teams prepare to take the "200-degree leap" and end cold-chain dependence for life-saving cell therapies

Funded teams will develop room-temperature preservation technology that could reduce costs and expand patient access to cell therapies and cancer drugs

The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services (HHS), today announced four contract awards through its BioStabilization Systems (BoSS) program, funding three teams to develop new methods to produce, store, and transport cell-based biologic medicines at room temperature. The BoSS program also awarded one independent verification and validation (IV&V) partner to provide test materials and performance assessments at critical junctures throughout the program. ARPA-H’s commitment is up to $87 million across the four-year program.  

The awards mark a major step toward the program’s goal of taking the "200-degree leap," from ultra-cold storage (around -196°C) to close to room temperature (around 21°C). This technology would end the reliance on the cold chain for some of medicine's most promising treatments and unlock the future of regenerative medicine. The technologies developed from BoSS, will save lives by making critical biologic medicines as shelf stable as aspirin, reducing the cost of and improving accessibility to cellular medicines for all Americans.

"Many of the newest therapeutics on the market rely on very cold storage temperatures, which contribute to unacceptably ballooning price tags," said BoSS Program Manager Gloria Elliott, Ph.D. "If we can eliminate the need for deep freezing, it will unlock the full, life-changing potential of cell-based therapies for patients who currently do not have access to them. To do that, we need to develop biocompatible materials that can prepare cells for stabilization and ensure successful re-animation, and we also need new processing approaches that are gentle enough to achieve that suspended animation state at scale without loss of cell viability."

Medicines manufactured from living cells or organisms (including cell- and gene-based therapies), called biologics, are a rapidly growing class of therapeutics with the promise of treating—and even curing—cancers, chronic autoimmune disorders, and other rare diseases. However, these therapies can be prohibitively expensive for the average American patient. This is in part due to the logistical requirement to store fragile biologics in an unbroken cold chain of ultra-cold storage, from the manufacturing site to the hospital. A single dose worth hundreds of thousands of dollars can be ruined by a shipping delay or cryogenic container failure. The reliance on frozen storage adds tens of billions of dollars annually and represents a significant logistical barrier to innovation and access. All BoSS performers will be working with transition partners to apply their technology developments to a cell therapy towards the end of the project.

The selected BoSS performer teams will work across two technical areas: interventions to stabilize and restore cells, and the creation of novel processing systems that mitigate chemical and physical stress to cells during manufacturing. The following teams have been selected. Performer awards vary in funding amount per awardee and are contingent upon each team meeting aggressive research milestones.

• DesiCorp will leverage high-throughput screening to identify biocompatible formulations that slow cellular metabolism and process optimization assisted by artificial intelligence (AI) to develop a thin-film freeze-drying system that stabilizes cells at room temperature.
• Charles Stark Draper Laboratory will use AI-assisted design of experiments to optimize formulation, quiescence induction parameters, hydrogel composition, and recovery media. They will use freeze-assisted droplet drying to achieve a stable product.
• University of California, Davis will induce a low metabolic state in cells using light-responsive gels and microfluidic processing techniques to produce cells that are stable at room temperature.

ARPA-H is committed to the rigor, translation, and adoptability of these technologies. The American Type Culture Collection (ATCC) is selected to serve as the IV&V partner. ATCC will provide working cell banks of government-selected cell types that will be used for demonstrations by performers. ATCC will provide unbiased assessments of both the viability and function of the preserved products and the design features of the preservation systems.  

If successful, work under the BoSS program could enable lower costs for cell-based drugs, reduce the risk of therapeutic and pharmaceutical product losses, and enable domestic reserves for public health preparedness. Awardee teams will progress through a four-year, multi-phase program structure, with performance evaluated against increasingly stringent benchmarks for cell viability, production speed, and shelf-life stability.

To learn more about BoSS, visit the program page.