Sana Biotechnology has announced positive initial results from its first-in-human study of UP421, an allogeneic primary islet cell therapy engineered with the company's hypoimmune (HIP) technology, in a patient with type 1 diabetes. The study, conducted in partnership with Uppsala University Hospital, achieved successful islet cell engraftment, avoidance of immune response, and insulin production for up to 28 days post-transplantation without the use of immunosuppressive drugs.
Breakthrough in Type 1 Diabetes Treatment
The results represent a significant step forward in the treatment of type 1 diabetes, a condition affecting over 1.7 million Americans. Current treatments often involve lifelong insulin injections and management of blood glucose levels. Sana's approach aims to provide a scalable, curative treatment by transplanting engineered cells that can evade immune detection, potentially eliminating the need for insulin injections or immunosuppression.
Key Findings from the Clinical Trial
Four weeks after cell transplantation, the study demonstrated 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 increased with a mixed meal tolerance test, consistent with insulin secretion in response to a meal. MRI scanning further confirmed a sustained signal at the site of transplanted cells over time, consistent with graft survival. Importantly, no safety issues were observed, and the HIP-modified islet cells did not elicit an immune response.
Analyst and Investor Reactions
The positive data led to a surge in Sana Biotechnology's stock price, with shares increasing over 300% following the market close on the day of the announcement. TD Cowen upgraded SANA from Hold to Buy, with analyst Mark Frahm noting that the results "meaningfully derisk" type 1 diabetes and other applications of the company's Hypoimmune platform. BofA Securities analysts reiterated their Buy rating on Sana Biotechnology stock, maintaining a price target of $7.00. Citi increased its price target for Sana from $8.00 to $15.00 due to progress in its Type 1 diabetes trial.
The Science Behind Hypoimmune Technology
Sana's HIP technology is designed to overcome the immunologic rejection of allogeneic cells. In type 1 diabetes, it also evades the autoimmune rejection of pancreatic beta cells. UP421 cells were transplanted without immunosuppression, and the survival of the islet cells provides evidence that these cells evade allogeneic and autoimmune detection. According to Sana CEO Steve Harr, "As far as we are aware, this is the first study showing survival of an allogeneic transplant with no immunosuppression or immune-protective device in a fully immune competent individual."
Future Directions
Sana expects to continue following the patient transplanted with hypoimmune-edited islet cells and will share additional data at a future medical meeting. The company is also advancing its SC451 program, a HIP-modified, stem cell-derived pancreatic islet cell therapy, and is prioritizing its UP421 and SC451 programs for Type 1 diabetes and its SC291 program for B-cell driven autoimmune diseases.
