Sana Biotechnology Presents Data at ISSCR 2021 Virtual Annual Meeting Showing Survival of Transplanted Hypoimmune Stem Cells Without Immunosuppression in Non-Human Primates

First demonstration of the survival of allogeneic iPSCs transplanted into an immunocompetent non-human primate model without the need for immune suppression

Transplanting allogeneic cells into a primate without immune suppression represents a key step toward widespread treatment of disease using engineered cells

SEATTLE, June 26, 2021 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, today presented data showing survival of transplanted stem cells without immunosuppression in non-human primates (NHPs). The transplanted cells were induced pluripotent stem cells (iPSCs) with Sana’s hypoimmune gene modifications that enable immune evasion. Data were presented by Sonja Schrepfer, M.D., Ph.D., Head of Hypoimmune Platform at Sana, during the plenary session on Cellular Therapy and Tissue Engineering at the International Society for Stem Cell Research 2021 Virtual Annual Meeting.

“These findings represent a major breakthrough in cell transplantation, as demonstration of immune evasion and durable cell survival in non-human primates with a healthy immune system has not been achieved before,” said Steve Harr, Sana’s President and Chief Executive Officer. “Since the debut of organ, tissue, and cellular transplantation, immune rejection of allogeneic transplants has been a significant challenge preventing widespread utilization of these therapies. Our team has now shown the potential of these gene-modified cells in multiple animal models, and the next step is to apply this technology in humans in several therapeutic contexts, with our first investigational new drug (IND) application as early as next year.”

Transplanting cells or tissues from a donor to a different recipient currently requires intense immunosuppression to prevent rejection of the transplant. Sana’s hypoimmune (HIP) platform’s goal is to eliminate the need for immunosuppression by cloaking cells from immune recognition. The platform disrupts major histocompatibility (MHC) class I and MHC class II expression to hide cells from the adaptive immune system, which includes antibody and T cell responses. However, these changes make cells susceptible to innate immune cell killing, in particular by natural killer (NK) cells. Sana’s HIP platform additionally provides for the overexpression of CD47, a molecule that protects HIP-modified cells from innate cell killing involving either NK cells or macrophages. HIP-modified pluripotent stem cells can serve as the starting material for manufacturing cell-based therapeutics, differentiating into specialized cell types. Sana’s goal is to use these HIP-modified cells to replace damaged or missing cells in the body in a number of different diseases, including cancer, type I diabetes, cardiac disease, and others.

In this study, allogeneic iPSCs were transplanted intramuscularly into healthy NHPs without immunosuppression (n=8), split into two cohorts. The first cohort received unmodified allogeneic iPSCs, while the second cohort received HIP-modified allogeneic iPSCs. The unmodified cells disappeared rapidly in all NHPs, with significant T cell activation and antibody production. The HIP-modified iPSCs survived in all four monkeys for the duration of the study (up to four months at data lock), and there was no evidence of a systemic immune response, including no T cell activation, antibody production, or NK cell activity (p