Sana reports six-month follow-up results from islet cell transplantation study

Sana Biotechnology (SANA) announced six-month follow-up results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with Sana’s hypoimmune technology, into a patient with type 1 diabetes without any immunosuppression. The study is being conducted in partnership with Uppsala University Hospital. The results are consistent with and build upon the previously reported four-week and 12-week clinical results. Results of the study at six months after cell transplantation demonstrate the survival and function of pancreatic beta cells as measured by the presence of circulating C-peptide, a biomarker indicating that transplanted beta cells are producing insulin. C-peptide levels also increase with a mixed meal tolerance test, consistent with insulin secretion in response to a meal. 12-week PET-MRI scanning also demonstrated islet cells at the transplant site, a forearm muscle. The study identified no safety issues, and the HIP-modified islet cells evaded immune responses. “Durable survival, function, and immune evasion of transplanted allogeneic pancreatic islet cells with no immunosuppressive medicines, particularly in the context of a pre-existing autoimmune response to these cells, represents a transformative and necessary step to making cellular and transplant medicine more accessible,” said Steve Harr, CEO. “Type 1 diabetes currently impacts over nine million people globally, and there have been relatively few transformational advances in this disease since the discovery of insulin over 100 years ago. The data presented today bring our vision-treating diabetes with a broadly available therapy leading to normal blood glucose control without either insulin or immunosuppression-closer to reality. We are incorporating the immune evasion learnings and technology from the current UP421 trial to develop SC451, a HIP-modified, stem cell-derived islet cell therapy, for which we intend to file an investigational new drug application as soon as next year.” Primary islet cell transplantation with immunosuppression is an established procedure in type 1 diabetes in which allogeneic pancreatic islet cells are isolated from a deceased donor’s pancreas and transplanted into a patient with a goal of normal blood glucose control and insulin independence. As with whole-organ transplants, suppression of the recipient’s immune system has historically been required to prevent immune rejection of the allogeneic transplanted cells and resurgence of the inciting autoimmune attack. Sana’s HIP technology is designed to overcome immunologic rejection of allogeneic cells, and in type 1 diabetes, to evade the autoimmune rejection of pancreatic beta cells as well. UP421 cells were transplanted with no immunosuppression, and the survival of those islet cells provides evidence that they evade both allogeneic and autoimmune detection.