What’s Happening?
A new publication in The New England Journal of Medicine provides more detail about the first person with type 1 diabetes (T1D) treated with Sana Biotechnology’s gene-edited islets. This person is still making insulin 14+ months after their islet transplant—without immunosuppressants!
What to Know About Sana’s Gene-Edited Islets
Sana’s novel cell therapy approach is designed to help transplanted islet cells evade immune attack while continuing to produce insulin. The strategy uses gene editing to create hypoimmune islet cells, which are cells engineered to avoid detection by the immune system while maintaining their insulin-producing function. This approach is one of several next-generation cell therapy strategies being prioritized by Breakthrough T1D to help overcome one of the biggest barriers to cell replacement therapies: immune rejection.
In this first-in-human study, deceased-donor islets were genetically modified to become immune-evasive and then transplanted into the forearm. Because this is a phase 1 trial, the primary goal is to assess safety, while also monitoring islet function through C-peptide, a marker of the body’s own insulin production.
Importantly, the transplant included only about 5% of the number of cells typically needed to fully restore insulin production, reflecting the exploratory nature of the study.
The Results So Far
The results so far are encouraging. After 60 weeks, there have been no severe adverse events, meeting the trial’s primary safety endpoint. At 14 months after transplantation, the participant continued to produce detectable C-peptide, indicating that the transplanted cells remained alive and functional. The islet cells were also visible in the participant through PET and MRI imaging.
Researchers also observed that C-peptide levels temporarily declined after about one year, likely due to beta cell exhaustion, but subsequently recovered. This is an encouraging signal that the transplanted cells may be capable of regaining function.
Importantly, the participant had no detectable immune response to the transplanted islet cells. While the levels of T1D autoantibodies remained unchanged, this had no impact on the survival and function of the gene-edited islets.
What’s Coming Next
While still very early, these findings provide important proof of concept that gene-edited, immune-evasive islet cells can survive and function in a person with T1D. If confirmed in larger studies, this approach could help move the field closer to cell therapies that work without long-term immune suppression—a major goal for the future of T1D cures.
Excitingly, Sana plans to translate their hypoimmune gene-editing technology to manufactured islets in a new clinical trial, meaning that they are combining their unique immune protection strategy with a scalable approach. This is in line with our Project ACT initiative, which aims to dramatically speed development, access, and adoption of islet cell therapies for everyone with T1D who wants them.
Project ACT
Scientific progress takes time, resources, collaborations, and effort. To get islet cell therapies to people with T1D faster than ever, Breakthrough T1D launched Project ACT (Accelerate Cell Therapies) to simultaneously advance research, development, regulatory policies, access, and adoption of islet cell therapies that do not require broad immunosuppression.
Driving Research into T1D Cell Therapies
Sana is a portfolio company of the T1D Fund: A Breakthrough T1D Venture. Through equity investments, we have helped Sana grow their T1D pipeline, leading to their gene-edited islet cell therapy being investigated in human clinical trials. Even more, the T1D Fund recently invested in Century Therapeutics, another company developing manufactured islet cell therapies with immune-evasive capabilities. We will continue to work closely with Sana and other promising companies to support their product development and accelerate scalable islet cell therapy approaches that do not require immunosuppression.
This article was written by Sandy Vogt, Ph.D., and Brian Herrick.
