New biotech start-up OptiKira created by BioMotiv

USA-based BioMotiv, a drug development accelerator associated with The Harrington Project, the University of California San Francisco (UCSF), and University of Washington, Seattle, have announced the formation of OptiKira, a platform company that will develop small-molecule therapeutics that prevent cell death in pathologies caused by misfolded or unfolded proteins.

Extensive research by the Founders has helped define the biological pathway leading to progressive cell death which characterizes diseases such as retinitis pigmentosa, diabetes, and amyotrophic lateral sclerosis (ALS). They have found that the Unfolded Protein Response (UPR), a mechanism by which the cell deals with functionally abnormal proteins, has an important housekeeping role in cell metabolism. However when overloaded, the "terminal UPR" results in the accumulation of excessive unfolded proteins and cellular death.

The team has synthesized inhibitors of IRE1a to prevent the activation of the terminal UPR, while not disrupting the housekeeping role of the enzyme. They have demonstrated that inhibitors of IRE1a, which they have named kinase-inhibiting RNase attenuators (KIRAs), protect cells from degeneration in preclinical models of retinitis pigmentosa and diabetes.