Sana Biotech’s Cell Therapy without Immunosuppression is a Gamechanger

Earlier this month, Sana Biotechnology, a clinical-stage company, released trial results in which it implanted insulin-producing islet cells into the arm muscle of a type 1 patient without triggering a rejection response.

Led by Per-Ola Carlsson at the University of Uppsala Hospital in Sweden, the study involved cadaver-sourced, insulin-producing cells (UP421) modified with Sana’s Hypoimmune (HIP) Technology platform. This first-time study proved safe cell transplantation without immunosuppression, and it is supported by funding from the Leona M. and Harry B. Hemsley Charitable Trust.

Since type 1 diabetes (T1D) is an autoimmune condition where the body attacks the pancreas’ insulin-producing cells, the challenge has been to either encapsulate the functioning cells or take immunosuppressive agents. Unfortunately, these anti-rejection medications leave individuals vulnerable to side effects and an increased risk of infection.

The ideal cure is cell transplantation, where the cells are protected.

Exciting Breakthrough

Although only one participant was involved in the study and researchers had only one month’s data, this is exciting for T1Ds. Cell protection through gene modification without immune meds is big news.

 Here’s why…

When the scientists transplanted functioning islet cells into the forty-two-year-old man with T1D, the cells began producing insulin immediately. They also responded positively to food intake, generating more insulin after meals— without needing immune-suppression drugs.   

Twenty-eight days after the procedure, an MRI scan revealed healthy insulin-producing cells not under attack by the body’s immune system, consistent with graft survival.

This first-in-human study proved that Sana’s Hypoimmune (HIP) Technology enabled the transplanted islet cells, allogeneic islets engineered with Sana’s HIP technology, to avoid immune rejection and continue producing insulin. 

The transplanted UP421 cells evaded both allogeneic and autoimmune detection.

The HIP-engineered pancreatic islet cells produced “consistent levels of circulation C-peptide, a biomarker of insulin production, and increased C-peptide levels with a mixed meal tolerance test (MMTT).”

The study, conducted in partnership with Uppsala University Hospital in Sweden, found no safety issues, as the islet cells evaded immune responses.

“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 Per-Ola Carlsson, MD, Study Principal Investigator, Senior Physician and Professor at the Clinic for Endocrinology and Diabetology at Uppsala University Hospital.

How is the body accepting these new cells?

The gene-edited hypoimmune cells have lower recognition from the immune system by disrupting the expression of MHC proteins that trigger the immune system attack and amplifying CD47, a cell receptor that releases a protection signal of ‘don’t eat me’ to other potential attackers.

Breakthrough T1D Support

“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 Aaron J. Kowalski, Ph.D., CEO of Breakthrough T1D (previously JDRF).

The study involves donor-derived, HIP-engineered islet cells in the forearm of T1D patients. Results from the four-week study demonstrated the survival and function of pancreatic beta cells at each weekly blood draw.

Large-Scale Cell Production

Typically, when islet cell transplantation is combined with immunosuppression, the cells are isolated from a deceased donor’s pancreas. Similar to organ transplants, immunosuppressive drugs are required to prevent immune rejection.

Sana is applying its HIP technology to pluripotent stem cells, which can be differentiated into multiple cell types, including pancreatic islet cells and donor-derived allogeneic CAR T cells. In the future, they hope to mass-produce these cells for large-scale expansion.

About Sana Biotechnology

Sana Biotechnology is a life science corporation that develops engineered cells to treat diseases. Its gene therapy technologies repair and control genes in cells, and its cell replacement restores missing or damaged cells in the body.

In addition to its work with diabetes, Sana also develops medicines for diseases like cancer and other autoimmune disorders. Sana operates in Seattle, WA, Cambridge, MA, San Francisco, CA; and Bothell, WA.

After the positive trial data, Sana’s stock price surged 152% in one week.

“Today’s data, when combined with progress elsewhere in the field, provides real hope that a scalable, curative treatment for patients with type 1 diabetes, meaning normal blood glucose with no insulin injections or immunosuppression, is possible,” said Carlsson. “We look forward to longer follow-up and plan to submit study results for publication as well as for presentation at an upcoming scientific forum.”

Giant leap toward a T1D cure

Sana Biotech’s positive clinical results bring us closer to the cure for type 1 diabetes, potentially transforming the treatment for T1Ds to live full lives sans pumps and shots and managing blood sugar levels without the dangerous risks of immune-suppressive drugs. The early data from this transformative cell therapy is a first and breeds hope.

However, there needs to be a supply accessible to the whole T1D community, as cadaver-sourced cells are in limited supply, and mass production of pluripotent stem cells is required.

T1D Strong will continue to watch this innovative study for the safety and function of these transplanted cells.