iTOL-102

A Potential Stem Cell-Derived Cure for Type 1 Diabetes

Our lead commercial program, iTOL-102, leverages significant advancements in stem cells to derive human pancreatic islets which allows an inexhaustible supply of insulin-producing cells and is 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.

We have entered into a research collaboration with Kadimastem as a potential stem cell source, which is funded by a $1 million grant from the Israel-U.S. Binational Industrial Research and Development (BIRD) Foundation, to evaluate and advance iTOL-102 towards first-in-human studies.

Compelling Results Demonstrated in Non-Human Primate (NHP) Study Published in Science Advances with iTOL-100

iTOL-100 combined with NHP cadaveric donor islets implanted into an NHP model of Type 1 Diabetes has shown long-term graft function with control of blood glucose levels and restoration of insulin secretion without the use of chronic immune suppression in a preclinical study.

How iTOL-100 is Designed to Work

In a preclinical NHP 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.

1.6 million

adult patients over the age of 20 in U.S.³

$800K – $1.7M

average costs of patient treatment
for the payer⁵