Endsulin is taking an entirely new approach to therapeutic development in diabetes. Watch the video below to see how our gene therapy works.
Our approach
Our approach involves using gene therapy to modify cells in a patient’s liver to directly replace the insulin-producing beta cells that have been destroyed in people with Type 1 Diabetes. The foundational work for this proprietary technology was done by our founder, Dr. Hans Sollinger and his team at the University of Wisconsin-Madison.
Our gene therapy
Gene therapy has the potential to deliver results with prolonged durability, which has been observed in the treatment of several other conditions. Our goal in taking this scientific approach is to deliver durability measured in years, and potentially life-long duration, with a single outpatient infusion that could be given by any endocrinologist or clinic.
Our delivery vector
Our delivery vector, a specific adeno-associated virus (AAV) known as AAV8, has been proven to yield durable liver expression in the clinic.
This is one of the most widely studied AAVs, with a long-term safety record. In fact, the FDA has approved more than 100 AAV trials.
Our patented gene construct
Our patented gene construct exploits well-characterized glucose-sensing expression pathways in the liver cells known as hepatocytes to produce insulin. We also include a genetic factor that enables these cells to be actively regulated by the body’s own systems based on changing glucose levels in the blood. Hepatocytes lack the surface proteins that are the target of the autoimmune response that destroys beta cells, reducing the risk that the genetically treated hepatocytes will be targeted. That makes it unlikely that our potential therapy would require the addition of costly and lifestyle-inhibiting immunosuppressants.
