Exacis Biotherapeutics is using mRNA to reprogram somatic cells to create induced pluripotent stem cells (iPSCs) in-house. We further engineer these high quality iPSCs using mRNA to vectorize our proprietary gene editing protein NoveSlice™ to create engineered iPSCs that are allogeneic, potent and safe. We then differentiate these cells into highly active engineered iNK cells for treatment of malignancies with high unmet medical needs.
Exacis is democratizing access to cell therapies for cancer treatment by employing non-viral, capital-efficient manufacturing processes to decrease the cost of treatment while at the same time improving the safety of cell therapies. Exacis has exclusively in-licensed a robust portfolio of patents for producing engineered NK and T cells for oncology. The technology is applied to generate a programmable therapeutics platform that can be modified, using our in-licensed library of gene editing protein building blocks, to target virtually any cancer antigen. We plan to treat both solid and hematologic tumors with a goal to improve the patient experience while improving outcomes and decreasing the cost of care.
Exacis Biotherapeutics employs key proprietary technologies that work together to allow us to develop the most potent and safe cell therapies:
- mRNA-based cell reprogramming – Exacis’ approach creates fully rejuvenated, footprint-free iPSCs using mRNA to reprogram human somatic cells to iPSCs in ~3 weeks. Our iPSC are highly metabolically active with fully restored telomeres and the ability to be differentiated, using directed differentiation protocols, into many different cell types, including NK and T cells.
- mRNA-based gene engineering with proprietary editing protein – Exacis’ proprietary technologies are mRNA based (RiboSlice™) and avoid the use of DNA and viruses to perform knock-in, knockout, and single base correction as needed to generate the potent and safe cell therapy products. Exacis’ proprietary gene-editing protein, NoveSlice™ is fully patent-protected, efficient, and specific in its editing activities.
- Exacis uses its proprietary fusogenic lipid delivery system (ToRNAdo™) to deliver single complex lipid-mRNA to target cells. The fusogenic nature avoids endosome formation thereby avoiding challenges with endosomal escape.
Exacis is developing allogeneic NK and T cell therapies for solid tumors and hematogenous malignancies by employing its platform technologies as follows:
- Exacis creates iPSCs using mRNA-based cell reprogramming technologies that yield fully restored and rejuvenated iPSCs that are fully genomically characterized prior to the next step.
- Stealthing edits and performance enhancements are engineered using proprietary mRNA-based gene editing tools (RiboSlice™ and NoveSlice™) in order to increase persistence and potency. These enhancements comprise the engineered iPSC backbone used to generate all our cell therapy products. These engineered iPSCs are fully genomically characterized and banked for use to create individual products.
- Chimeric Antigen Receptors (CAR) are then added using mRNA vectoring approaches (avoiding the use of viruses and DNA) for products that include CARs. Non-CAR bearing products (ExaNK™) bypass this step.
- The fully enhanced iPSC are then expanded clonally to yield large numbers (billions) of homogenously edited cells which are then differentiated into the final product (ExaNK™; ExaCAR-NK™; ExaCAR-T™) which is frozen for shipment and administration to patients.