SBIR Phase I: Intracellular Delivery for Rapid Point-of-Care Labeling of Therapeutic Cells

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The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase I project involves leveraging an innovative approach to deliver and monitor therapeutics more effectively. Real-time monitoring and tracking of immune and stem cell therapies is in its infancy. However, without the capability to precisely know where therapeutic cells localize, clinicians cannot assess safety, design optimal doses, or make clinical decisions related to therapeutic approaches. As the multi-billion-dollar cell therapy market is expanding rapidly in its race to develop the next generation of life-saving, cost-effective, lower toxicity cell therapy products there is a heightened requirement for safety risk management to monitor on-target, on-tumor delivery of cell products in patients. This proposal addresses a critical unmet need in the cell and gene therapy industry for rapid point-of-care labeling of therapeutic cells specifically to enable tracking in the body. The addressable market for preclinical and clinical cell tracking in clinical trials involving therapeutic cells in the United States is estimated to reach approximately $1.14 billion by 2024.This Small Business Innovation Research (SBIR) Phase I project involves an innovative approach to intracellular delivery where mechanoporation or cell-squeezing is achieved simply by propelling cells, in the presence of exogeneous cargo, through a proprietary mechanoporation element that resides in a column designed to be processed in a centrifuge (spin column). End users will realize significant advantages over conventional and microfluidic approaches to intracellular delivery including: 1) Convenient spin column format; 2) Short cell processing times; 3) Ease of scale-up and multiplexing; 4) Applicability to numerous cell types; and 5) Compatibility with automated/robotic cell production systems including autologous cell production systems designed for clinical settings. Three objectives are proposed involving the generation of prototype spin columns and their use in optimizing aspects of element design to afford efficient intermolecular delivery of iron-based nanoparticles (magnetic resonance imaging contrast agent) into T-cells and Mesenchymal Stem Cells. The study will enable rapid point-of-care labeling of therapeutic and regenerative cells for in vivo tracking, empowering clinicians to make timely decisions with respect to treatment strategies, thus ensuring better clinical outcomes.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.