ORC-0001

ORC-0001 (formerly known as PROTO-1) is a small molecule compound developed by Oricula Therapeutics for the prevention of aminoglycoside-induced hearing loss (ototoxicity). Discovered through high-throughput screening of zebrafish lateral hair cells, the compound was found to protect inner ear hair cells from the toxic effects of aminoglycoside antibiotics. ORC-0001 demonstrated efficacy in both zebrafish and mammalian (rat) models, providing a foundation for the development of its more potent analog, ORC-13661. The compound is a new chemical entity discovered and patented by the University of Washington and is exclusively licensed to Oricula Therapeutics. While it served as the lead structure for structure-activity relationship (SAR) development, it has largely been superseded by ORC-13661 in clinical development.