Sana Biotechnology's Breakthrough in Type 1 Diabetes: Islet Cell Transplantation Without Immunosuppression

Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, has announced promising clinical results from a first-in-human study of islet cell transplantation without immunosuppression in a patient with type 1 diabetes. The study, conducted in partnership with Uppsala University Hospital, demonstrated the survival, function, and immune evasion of HIP-modified primary pancreatic islet cells (UP421) for at least 28 days post-transplantation.

The study's principal investigator, Per-Ola Carlsson, MD, Senior Physician and Professor at the Clinic for Endocrinology and Diabetology at Uppsala University Hospital, expressed optimism about the potential of this approach. "These initial exciting results build upon the extensive preclinical and translational studies of Dr. Sonja Schrepfer and the team at Sana. The clinical data are highly promising for patients and provide the first evidence in humans for overcoming allogeneic and autoimmune rejection with pancreatic islet cell transplantation in type 1 diabetes with no immunosuppression," said Carlsson.

The study identified no safety issues, and the HIP-modified islet cells evaded immune responses, as demonstrated by the presence of consistent levels of circulating C-peptide, a marker of insulin production. C-peptide levels also increased with a mixed meal tolerance test (MMTT), suggesting insulin secretion in response to a meal. MRI scanning showed signals consistent with graft survival, further supporting the study's positive results.

Steve Harr, Sana's President and Chief Executive Officer, emphasized the significance of these findings. "We achieved our goals for the study, identifying no safety issues as well as demonstrating survival, function, and evasion of immune detection of HIP-modified primary pancreatic islet cells transplanted intramuscularly with no immunosuppression," said 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. Safe cell transplantation without immunosuppression has the potential to transform the treatment of type 1 diabetes and a number of other diseases."

Aaron J. Kowalski, Ph.D., CEO of Breakthrough T1D (previously known as JDRF), expressed enthusiasm about the potential impact of these results. "These initial clinical results show that cell therapies that replace insulin-producing cells without immunosuppression are approaching reality as a meaningful and potentially life-changing cure for type 1 diabetes," said Kowalski.



The success of Sana's hypoimmune (HIP) technology in this study represents a significant advancement over previous attempts at islet cell transplantation without immunosuppression. Unlike previous studies, Sana's HIP-modified primary pancreatic islet cells demonstrated survival, function, and immune evasion in a patient with type 1 diabetes for at least 28 days post-transplantation. This was evident by the presence of consistent levels of circulating C-peptide, a marker of insulin production, and increased C-peptide levels with a mixed meal tolerance test (MMTT). Additionally, MRI scans showed signals consistent with graft survival. The improvement can be attributed to the HIP technology, which enables the transplanted cells to avoid immune detection, function, and persist without the need for immunosuppression. This is a major breakthrough, as previous attempts at islet cell transplantation without immunosuppression have resulted in immune rejection and graft failure.

These results suggest that Sana's hypoimmune (HIP) technology could revolutionize type 1 diabetes treatment by enabling transplanted islet cells to avoid immune rejection without immunosuppression. This could lead to a scalable, curative treatment, potentially eliminating the need for insulin injections or immunosuppression. If successful in humans, this approach could significantly reduce the market for insulin and other diabetes therapies, as patients may no longer require daily insulin injections. Additionally, it could open up new opportunities for cell-based therapies in diabetes treatment, potentially transforming the market landscape.

However, longer follow-up is needed to assess the durability of these results and to identify any potential long-term risks. Sana must also consider the competitive landscape and the best path to regulatory and commercial success. The company is actively seeking a licensing partner for SC291 in oncology and exploring opportunities to spin out its SC379 program. These strategic decisions will help Sana focus its resources on areas with the greatest potential impact for patients and extend its expected cash runway into 2026.

In conclusion, Sana Biotechnology's hypoimmune (HIP) technology has shown promising results in a first-in-human study of islet cell transplantation without immunosuppression in a patient with type 1 diabetes. The success of this approach could transform the treatment of type 1 diabetes and open up new opportunities for cell-based therapies in diabetes treatment. However, longer follow-up and further development are needed to fully realize the potential of this breakthrough technology.