For patients with diabetes, every treatment option comes with serious drawbacks: daily injections, constant changes in dosing, ongoing risk of severe side effects, or lifelong immunosuppressant drugs that are difficult on the body.
While the science of management has advanced, insulin shots have been the enduring standard of care since the 1920s — almost a full century.
Dr. Sollinger and the ENDSULIN team have been on a steadfast mission to free patients from the daily management of their disease. Through 25+ years of innovative and focused research at the University of Wisconsin Hospitals and Clinics in Madison, they finally unlocked an elegant answer, using the latest biotechnology.
PATIENT-DRIVEN SCIENCE
GENE THERAPY
BECAUSE IT’S MINIMALLY INVASIVE AND DURABLE ► Gene therapy is one of the most promising emerging biotechnologies, because it can cure patients for a prolonged period of time. For instance, patients receiving gene therapy treatments for hemophilia have been cured for 10 years with one treatment.
GIRE
BECAUSE IT IS PRECISE ► ENDSULIN has developed a patented DNA sequence called glucose-inducible regulatory element (GIRE). It responds to glucose concentrations in the blood, then provides regulated release of insulin to accomplish blood-glucose control.
Animal experiments have achieved this type of control for the life of the animal.
AAV8
BECAUSE IT’S EFFICIENT ► GIRE is packaged inside adeno-associated virus (AAV), which is not known to cause disease, but can still transfer the gene to the liver cells. AAV8 has a long-term safety record, and the FDA has approved more than 100 AAV trials.
HEPATOCYTES
BECAUSE THEY CAN ALREADY REGULATE GLUCOSE ► A major advantage of hepatocytes is that they can effectively produce peptides, including insulin.
The liver already has regulatory elements that, with the addition of our unique GIRE, assist in maintaining normal glucose levels. The genetically altered hepatocytes mimic the action of a normal pancreas.
Because hepatocytes don’t carry proteins on their surface (which are the target of the autoimmune disease that causes diabetes), there is no risk the autoimmune system will destroy the genetically altered hepatocytes.
TARGETED
TO MITIGATE RISK ► The therapy exclusively targets a small portion of cells so it doesn’t interrupt the liver’s regular functioning. For the treatment to work, only one in 50 liver cells need to be transduced.
REALISTIC APPROACH
RESEARCH INFO
First company to publish data demonstrating that glucose control, and insulin disappearance after glucose stimuli, follow a near-perfect physiological pattern.
Among one of the first labs to pursue hepatocyte-based insulin gene therapy
The only researchers in the world testing gene therapy in large mammals for diabetes with autoimmune disease. All experts accept that for any T1DM therapy to be meaningful, it must be evaluated in an autoimmune large-animal model.