A new startup company developing therapeutics with potential applications to ALS has just been launched! The company, OptiKira, is developing small molecules that can prevent cell death due to misfolded or unfolded proteins. The scientific founding team, which includes Scott Oakes and Feroz Papa from University of California, San Francisco (UCSF) and collaborators from UCSF and University of Washington, have made seminal discoveries on the role of the unfolded protein response (UPR) in cellular metabolism and mechanisms by which overactivation of the UPR leads to cell death. The platform company will initially focus on inhibitors of inositol-requiring enzyme-1α (IRE1α), a key activator of the UPR. OptiKira’s platform could have direct applications to ALS, based on increasing evidence that inhibition of the UPR may be a promising therapeutic avenue in neurodegenerative diseases linked to protein misfolding (see Oct 2013 news story; June 2008 news story).
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