iTOL-102

A Potential Stem Cell-Derived Cure for Type 1 Diabetes

iTOL-102 is our lead commercial program being developed as a potential cure for Type 1 Diabetes. Using our proprietary SA-FasL biotin-PEG microgel platform, iTOL-100, which acts to generate localized immune tolerance, allogeneic stem cell-derived pancreatic islets will be implanted in the body. These stem cells are potentially capable of secreting insulin in response to sugar intake, similar to how native pancreatic islet cells behave. Additionally, the use of stem cell-derived pancreatic islets provides a potentially inexhaustible supply of insulin-producing cells.

We have initiated preclinical studies with iTOL-102, utilizing different stem cell sources, as we advance towards our first-in-human studies.

Compelling Results Demonstrated in NHP Study Published in Science Advances with iTOL-101

iTOL-101 demonstrated long-term function with control of blood glucose levels and restoration of insulin secretion without the use of chronic immune suppression.

How iTOL-100 is Designed to Work

In a pre-clinical non-human primate study of our lead program using allogeneic pancreatic islet cells and iTOL-100, the implanted pancreatic islets were shown to act similar to native islets, by secreting C-peptide and insulin in response to sugar intake.  The study included early treatment with an immunosuppressive drug, sirolimus (full dose for two weeks and then tapering off over 90 days).  The duration of the study was six months and allowed the researchers to confirm the viability of the implanted pancreatic islets through C-peptide and insulin levels at 3 and 6 months in the fasted and stimulated with glucose states.  At the conclusion of the study, the implanted tissue was removed from the animals confirming the implanted pancreatic islets were responsible for the C-peptide and insulin levels in the fasting and stimulated with glucose states.

Even with Insulin Treatment, There Remains a Significant Unmet Need in Type 1 Diabetes

Type 1 Diabetes is a chronic condition in which the pancreas produces little or no insulin. People with brittle diabetes frequently experience large swings in blood sugar that can quickly move from too high to too low or vice versa. Severely low blood sugar, called hypoglycemia, can cause sudden and unexpected seizures, coma, heart attacks, and even death.

70K – 100K

very severe uncontrolled
type 1 diabetes; “brittle”²