Many life-saving biologics rely on ultra-cold storage and transport. The BioStabilization Systems (BoSS) program wants to change that.
The U.S. Department of Health and Human Services' Advanced Research Projects Agency for Health (ARPA-H) announced contract awards through its BoSS program to develop new methods to produce, store and transport cell-based biologic medicines.
The University of Texas at Austin joins a six-member team led by DesiCorp, Inc. to develop an integrated platform that combines transient, reversible cellular modifications with a closed-loop bioprocessing system to enable room-temperature storage and on-demand reanimation of cells.
Cell-based biologics are advanced medicines that use living cells to treat cancers, chronic autoimmune disorders and other serious diseases. Keeping those cells viable until they reach the patient requires ultra-cold storage at temperatures as low as -196 °C (-320 °F) and transporting these materials is expensive, unwieldy and vulnerable to delays and equipment failure.
"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."
Thin-film freeze-drying (TFFD)—invented and developed on the UT Austin campus—could help make that cold-chain dependence a thing of the past.
TFFD is an advanced drying technology that rapidly freezes liquid solutions into thin sheets on a cold surface before removing the ice under a vacuum. Applied successfully to living cells, TFFD could enable room-temperature storage and transport, greatly reducing costs and allowing more flexible transport options.
Zhengrong Cui, Ph.D., will lead optimization of thin-film freeze-drying processes. Cui serves as Division Head and Professor of Molecular Pharmaceutics and Drug Delivery (MPDD) in UT Austin's College of Pharmacy and developed TFFD for applications with eukaryotic cells.
Key investigators also include fellow MPDD Professor Robert O. (Bill) Williams III, Ph.D., who invented TFFD, and Assistant Professor Alexander Marras, Ph.D. in the Department of Mechanical Engineering at the Cockrell School of Engineering, who will lend his expertise in materials science.
As part of the DesiCorp-led team, UT Austin researchers will help optimize the TFFD critical process parameters (CPPs) and composition/material attributes (CMAs), while also contributing expertise in aseptic processing and scaling up the technology. UT Austin has intellectual property demonstrating that TFFD can preserve human monocyte cells with intact membranes after rehydration.
"We thank ARPA-H for its visionary BioStabilization Systems program supporting the development of technologies to enable the production, storage and shipment of cell and gene therapies without ultra-cold storage requirements," says Cui. "We also appreciate the opportunity to collaborate with DesiCorp and other team members to further optimize UT Austin's TFFD-enabled technology."
DesiCorp will lead integration of the overall workflow by using a high-throughput screening platform to rapidly identify biocompatible formulations that enable cells to survive TFFD and resume function after rehydration. These efforts will be led by Brett Janis, Ph.D. and David Grimm, Ph.D.
For Cui, that potential is ultimately about access. He is excited to contribute to the BoSS program's goal of making "the seemingly impossible problem of room temperature biologics possible."
Developing lifesaving treatments is only part of the challenge; those treatments also must reach the patients who need them. By reducing the cost and complexity of storing and transporting cell-based medicines, technologies like TFFD could help end cold-chain dependence and expand patient access to these advanced therapies.