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OptiKira: Exploring a Novel Approach to Treating Diabetes

By: Carolyn Zheng

On July 18, 2017, the Center for Disease Control and Prevention reported that as of 2015, 30.3 million Americans were living with diabetes, and of those individuals, 7.2 million cases were undiagnosed. With the number of new cases of diabetes doubling every year and an additional 84.1 million Americans estimated to have prediabetes, a condition that can lead to type 2 diabetes within five years, it is no secret that this disease is a serious health crisis.

Type 2 diabetes, which accounts for 90-95% of all diabetes cases, is a disease ultimately characterized by irregular blood sugar levels in patients. This imbalance is caused by two factors: insulin resistance and pancreatic β cell death. The hormone insulin regulates blood glucose levels. When the body does not respond to insulin, the result is either abnormally high or low blood sugar levels, the former of which is a characteristic of diabetes. Pancreatic β cells are responsible for the production of insulin, so in the case of type 2 diabetes, not only is the body insensitive to insulin, but also there is not enough of the hormone being produced to have its intended effect.

Current treatments for diabetes target controlling the level of insulin and its efficacy in the body. Where the standard of care is lacking, however, is in preserving the functionality of β cells in order to boost levels of insulin production. That’s where OptiKira steps in.

OptiKira, LLC is a biotech company that specializes in the prevention of cell death in multiple diseases, including diabetes. Our scientific founders have identified the vital role of a mechanism known as the unfolded protein response (UPR) in managing cell responses to misfolded proteins caused by stress in the endoplasmic reticulum (ER). High levels of misfolded proteins trigger the terminal unfolded protein response (T-UPR), which results in cell death. ER stress has been identified as a catalyst in the onset of diabetes, as it both dampens insulin receptors on cell surfaces and leads to the death of β cells in charge of producing insulin.

OptiKira’s lead therapeutic, Kinase Inhibiting RNAse Attenuators (KIRAs), targets IRE1α, the life-death switch that can push a cell to cell death via the T-UPR, or direct it towards homeostasis via the adaptive UPR (A-UPR), the preferred route. By inhibiting IRE1 α from prompting cell death, KIRAs have the potential to successfully reduce the loss of β cells to the T-UPR. Our highly potent, selective, orally-active therapeutic is an incredibly innovative approach to going beyond current diabetes drugs to take on β cell dysfunction and deterioration to provide disease modifying intervention.

OptiKira’s approach to combating diabetes has already garnered grant support from the National Institute of Diabetes and Digestive and Kidney Diseases, as well as continuing support from BioMotiv and its strategic partners. With this support, OptiKira has developed highly potent and selective, KIRA compounds that will be more accurate in their targeting and stay in the body longer. These compounds are currently being tested in diabetes animal models with the ultimate goal of nominating a clinical candidate to move forward to the clinic.

To learn more about OptiKira and the work it’s doing to fight diabetes and other diseases, including pulmonary fibrosis, nonalcoholic steatohepatitis (NASH),  neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), cancer, and retinitis pigmentosa, click here.

Erin Reese