Science

Exacis’ fully proprietary and next generation mRNA-based cell reprogramming and gene editing technologies allow the Company to create engineered cell therapies (ExaNK™, ExaCAR-NK™ and ExaCAR-T™) that overcome the limitations of the currently available therapies. Our ExaCELLs™:

• Are off the shelf (no manufacturing lead time) and can be administered in repeat doses and without requirement for HLA matching
• Are produced by a rapid and capital efficient manufacturing process owing to our avoidance of viruses and DNA as well as the ability to clonally expand our engineered iPSCs
• Are high-quality and uniform as our approach allows whole genome characterization followed by clonal expansion to ensure a homogeneous population of 100% edited cells with no off target effects within the genome – increases potency, avoids risk of mutagenesis, decreases risk of cytokine release syndrome and neurotoxicity (for ExaCAR-T™ cell products)
• Do not require preconditioning

Exacis is developing both NK and T cell treatments that contain genomic edits intended to decrease patients’ immune systems from recognizing and killing the cells (rejection resistance) and preventing the adopted cells from interfering with the host immune system (avoidance of graft vs. host disease). These cells are frozen and shipped for storage at point-of-care. They are administered off-the-shelf rather than requiring manufacturing on a single patient basis with the attendant costs, complexities, and timelines. Our approach includes editing components of the immune recognition system that render our products to be “stealth” cells. This avoids graft versus host attack and also increases persistence. This aspect of manufacturing, coupled with our ability to clonally generate large numbers of cells without requiring donors, supports our ability to stock our product on the shelves at multiple treating facilities.

ExaCELLs are created by “programming” or targeting, our programmable therapeutics platform to attack and kill individual tumor targets. The Programmable Therapeutics Platform consists of an iPSC backbone that is engineered for stealthing & performance. The engineering serves to decrease host immune surveillance, increasing persistence and potency. Because our products are derived from engineered iPSCs that are clonally expanded and differentiated, our products contain 100% edited cells. Our programming approach involves the following:

• ExaNK™ – the stealthed and performance-enhanced iPSC are directed to differentiate into highly metabolically active CD56+ / CD16+ NK cells to be administered in conjunction with monoclonal antibodies. The monoclonal antibodies serve to opsonize the tumor target and engage/activate the ExaNK™ cells for killing.
• ExaCAR-NK™ – the stealthed and performance-enhanced iPSCs are further engineered by the addition of a tumor-targeting CAR. The cells are then directed to differentiate into highly metabolically active CD56+ / CD16+ NK cells that can be administered as off-the-shelf treatments for specific tumors. 
• ExaCAR-T™ – the stealthed and performance-enhanced iPSCs are further engineered by the addition of a tumor-targeting CAR. The cells are then directed to differentiate into highly metabolically active CD4+ and CD8+ T cells.