Sana Biotechnology Reports Positive Results from Type 1 Diabetes Islet Cell Transplant Study

Sana Biotechnology, Inc. has unveiled initial findings from a groundbreaking study involving their hypoimmune (HIP) technology. This study marks the first human trial of UP421, an allogeneic primary islet cell therapy engineered to avoid immune rejection, which was administered to a patient with type 1 diabetes without immunosuppression. Conducted in collaboration with Uppsala University Hospital, the research offers promising insights into managing type 1 diabetes without the need for immune-suppressing drugs.

The four-week post-transplant results indicate successful survival and functionality of the transplanted pancreatic beta cells. This was evidenced by the detection of circulating C-peptide, a key indicator of insulin production by the transplanted cells. Furthermore, C-peptide levels rose following a mixed meal tolerance test (MMTT), aligning with typical insulin responses to food intake. MRI scans reinforced these findings, showing consistent signals indicative of graft survival at the transplantation site. Importantly, no safety concerns were identified, and the HIP-modified islet cells demonstrated an ability to avoid immune detection.

Dr. Per-Ola Carlsson, the study's principal investigator and a senior medical professional at Uppsala University Hospital, emphasized the significance of these results. He noted that the study provides the first human evidence of overcoming both allogeneic and autoimmune rejection without immunosuppression in the context of pancreatic islet cell transplantation for type 1 diabetes. These findings fuel hope for a scalable, curative treatment that could eliminate the need for insulin injections and immunosuppressive therapy in patients.

Steve Harr, President and CEO of Sana Biotechnology, highlighted the study's achievements, noting the successful demonstration of cell survival, function, and immune evasion without the need for immunosuppressive drugs. He remarked on the potential transformation this approach could offer in treating not only type 1 diabetes but also other diseases. The insights gained are expected to advance the development of Sana's HIP-modified, stem cell-derived pancreatic islet cell program (SC451) for type 1 diabetes.

Aaron J. Kowalski, CEO of Breakthrough T1D, praised the study's promising clinical outcomes, which suggest that cell therapies replacing insulin-producing cells without immunosuppression are nearing practical application. Breakthrough T1D, previously known as JDRF, has been a supporter and investor in such translational research projects, recognizing their potential to revolutionize type 1 diabetes treatment.

Traditionally, islet cell transplantation in type 1 diabetes requires immunosuppression to avoid the immune system's rejection of donor cells. Sana's HIP technology aims to bypass this requirement by engineering cells that can evade immune detection, thus reducing the risk of rejection without compromising the immune system.

The study, supported by a grant from The Leona M. and Harry B. Helmsley Charitable Trust, investigates whether HIP-engineered pancreatic cells can be safely transplanted to restore insulin production in type 1 diabetes patients without immunosuppression. UP421 was developed using Sana's HIP platform at Oslo University Hospital and involves the intramuscular transplantation of HIP-engineered islet cells into the forearms of type 1 diabetes patients. The primary goal is to ensure the safety of UP421 transplantation, with secondary objectives assessing cell survival, immune evasion, and C-peptide production.

Overall, the study's initial results showcase the potential of HIP technology to enable cell therapies that overcome immune barriers, paving the way for innovative treatments that could significantly impact the management of type 1 diabetes and potentially other diseases.