Additional Relevant Details Our ex vivo Cell Engineering Platform and ProgramsCell death leads to the manifestations of many of the most serious and common diseases, such as heart disease, Type I diabetes, and advanced multiple sclerosis. We believe that the most effective mechanism to treat these patients is to replace their missing or damaged cells. Our goal is to manufacture genetically modified cells that are capable of both replacing missing cells and evading a patient’s immune system, which otherwise would recognize these cells as foreign and reject them. We believe our hypoimmune technology will enable us to manufacture cells cost-effectively, at scale, with product consistency, providing the potential for global patient access.Our hypoimmune technology combines the three gene modifications below to hide cells from the host immune system: Disruption of MHC class I and class II expression (which inactivates adaptive immune responses), and overexpression of CD47 (which hides cells from the innate immune system, including macrophages and natural killer (NK) cells). Pluripotent stem cells from healthy donors are used as the starting material and are then genetically modified with the hypoimmune edits. These edited cells are then differentiated into cell types of therapeutic interest, which are administered to the patient as “off the shelf” therapies.

We have shown across multiple species preclinically that our hypoimmune cells are able to hide from the immune system and avoid immune rejection, and we are now developing this technology to make ex vivo therapies to treat multiple diseases. NHP iPSCs modified with hypoimmune edits can survive in vivo when transplanted into an allogeneic recipient. Notably, the transplanted cells completely evade immune responses despite the presence of a fully intact immune system and absence of immunosuppression.