Our Mechanism 

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) in response to various forms of ER stress.

Cells may respond to low levels of misfolded proteins by increasing expression of chaperones, increasing ER-associated degradation, inhibiting RNA translation, and other adaptive processes. High levels of unfolded proteins trigger apoptosis -or, cell death, the terminal unfolded protein response, and activate a variety of inflammatory and profibrotic pathways. IRE1α is the master regulator of the UPR, directing the cellular response toward the adaptive or terminal pathways.

OptiKira’s academic founders made the seminal discovery that proprietary small molecule IRE1α 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 pulmonary fibrosis, diabetes, and retinitis pigmentosa.

OptiKira is built upon the work of our founders and others in the field who have demonstrated that the UPR and its master regulator, IRE1α, are centrally involved in the pathology of multiple diseases.  

OptiKira has developed highly potent, selective, and orally-active KIRAs as systemic drug candidates for fibrosis, diabetes, neurodegenerative, and other immunological diseases.  

Our collaborative efforts on targeting premature cell death have produced this unique opportunity to address an unmet clinical need.
— Feroz Papa, M.D., Ph.D., Scientific Founder

Intellectual Property

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.