The Founding academic team including Scott Oakes, Feroz Papa, and Brad Backes at UCSF and Dustin Maly at the University of Washington has defined the critical role of inositol-requiring enzyme-1α (IRE1α) in directing the cellular unfolded protein response (UPR). The UPR allows cells to manage dysfunctional, misfolded proteins produced in the endoplasmic reticulum (ER). Cells respond to low levels of misfolded proteins by, for example, increasing expression of chaperones, increasing ER-associated degradation, inhibiting RNA translation, and other adaptive processes. High levels of unfolded proteins trigger apoptosis, the terminal unfolded protein response. IRE1α is the master regulator of the UPR, directing the cellular response towards the adaptive or terminal pathways. Our academic Founders made the seminal discovery that certain small molecule kinase inhibitors can effectively block the terminal pathway. They have demonstrated that these compounds, which they have named kinase-inhibiting RNase attenuators (KIRAs), can protect cells from degeneration in preclinical models of retinitis pigmentosa (RP), pulmonary fibrosis, and diabetes.
OptiKira has built upon the work of our Founders, developing highly potent and selective KIRAs as drug candidates. The company has developed drug candidates with application for back-of-the-eye diseases including RP with a goal to considerably slow or halt photoreceptor cell death and preserve vision. In addition, the company has developed highly potent and selective, systemic drug candidates with application for diabetes, neurological, and other immunological diseases.
Our technology is based on intellectual property exclusively licensed from University of California, San Francisco, and University of Washington. The company has also developed new intellectual property related to the improved compounds.