Tech Tuesday: Engineered stem cells offer treatment strategy for Type 1 diabetes and more

University of Arizona researchers have developed a breakthrough gene-editing strategy to overcome one of the greatest challenges in regenerative medicine: immune rejection. Their innovation, non-immunogenic human pluripotent stem cells, offers a promising path toward their primary goal of developing a functional cure for Type 1 diabetes (T1D) without the need for lifelong anti-rejection drugs.

This technology is led by Deepta Bhattacharya, Ph.D., Professor of Immunobiology; Hannah Pizzato, Ph.D., Principal Scientist at the Center for Advanced Molecular and Immunological Therapies (CAMI); and Christopher Shen, Ph.D., co-founder and CEO of Aleutian Therapeutics. Together, the team is translating fundamental immunology discoveries into a scalable therapeutic solution with the potential to transform regenerative medicine.

Type 1 diabetes is a chronic autoimmune disease in which the body destroys its own insulin-producing beta cells. While automated insulin pumps and continuous glucose monitors have improved disease management, only 25 percent of patients achieve the A1c levels recommended by the American Diabetes Association. Poor glycemic control can lead to severe long-term complications, including cardiovascular disease, kidney failure, neuropathy and retinopathy, all contributing to an average 10-year reduction in life expectancy.

Although T1D can be managed, its burden is significant.

“Even though it's obviously a disease that can be managed at this point, you just recognize the impact that it has on people's lives and families, and just the enthusiasm and passion that people who have Type 1 diabetes in their families have towards this level of research and the potential cure is really inspiring,” said Bhattacharya.

Cell replacement therapies that entail implanting insulin-producing cells into patients have shown early signs of serving as a functional cure. However, current approaches require lifelong immunosuppressive drugs to prevent rejection. Because these medications suppress the immune system and increase the risk of serious infections and cancer, only a small subset of patients are considered appropriate candidates for this type of therapy.

The genetic engineering strategy developed at The University of Arizona removes the signals that normally cause the immune system to recognize transplanted cells as foreign and attack them. When differentiated into insulin-producing cells, they have the potential to survive without triggering rejection, eliminating the need for toxic anti-rejection medications.

In preclinical studies, the engineered pluripotent stem cells cells survived for the full six-month study duration in fully immunocompetent mice, suggesting they can overcome stringent immune barriers. Proof-of-concept studies in T1D mouse models are ongoing.

Approximately 2 million people in the U.S. and 9 million globally live with T1D, representing a $16 billion market growing at 6 percent annually. Beyond diabetes, the platform has potential across regenerative medicine by generating multiple specialized cell types.

The research team is actively seeking collaborations and strategic partnerships to accelerate development and clinical translation of this technology.

For more information about licensing and partnership opportunities, contact Leah Langlais, Manager of Venture Development at Tech Launch Arizona, at LeahL@tla.arizona.edu or (520) 626-0639.

Visit the TLA StartupHub to learn more and see a video about this technology.