
iTOL-102
A Potential Stem Cell-Derived Cure for Type 1 Diabetes
iTOL-102 is our second lead program being developed as a potential cure for Type 1 Diabetes. Using our proprietary biotin-PEG microgel platform, iTOL-100, which acts to generate localized immune tolerance, insulin producing stem cell-derived pancreatic islets will be implanted in the body. These stem cell-derived pancreatic islets 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 provide a potentially inexhaustible supply of insulin-producing cells.
In a pre-clinical non-human primate study of our lead program using allogenic pancreatic islet cells and iTOL-100, the implanted pancreatic islets were shown to act as native islets, secreting insulin in response to sugar intake. Additionally, study animals did not require long-term immunosuppression therapy.

Potential to facilitate
functional engraftment while reducing cell rejection

Potential to eliminate the need for sustained immunosuppressants and antiviral, antifungal and antibacterial drugs

Multiple partnering opportunities for
stem cell-derived islets
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.
26 million
patients worldwide
1.6 million
patients in the U.S.
70K – 100K
very severe uncontrolled
type 1 diabetes; “brittle”²
Poor glucose control has long term consequences¹
%
Retinopathy at 18 years
(45% are severe)
%
Neuropathy
at 18 years
%
Nephropathy
at 18 years
1: Insulin independence rate from 2007-2010 in 677 islet transplants from Schuetz C, Markmann JF. Islet cell transplant: Update on current clinical trials. Curr Transplant Rep. 2016;3(3):254-263: 2: Vanstone, M. Patient Perspectives on Quality of Life With Uncontrolled Type 1 Diabetes Mellitus: A Systematic Review and Qualitative Meta-synthesis. Ont Health Technol Assess Ser., 15(17), 1–29.
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