BF844

BF844 is a first-in-class small molecule drug developed as a stabilizer of the CLRN1 (Clarin 1) protein, specifically targeting the N48K mutation associated with Usher syndrome type III (USH3). USH3 is a rare genetic disorder characterized by progressive hearing and vision loss due to mutations in the CLRN1 gene. BF844 works by modulating and stabilizing mutant CLRN1 protein, thereby mitigating its dysfunction. Preclinical studies have shown that BF844 can attenuate progressive hearing loss and prevent deafness in animal models of USH3. The drug is currently being evaluated in Phase 1 clinical trials for safety, tolerability, pharmacokinetics, and pharmacodynamics when administered orally to healthy volunteers[1][3][5][6][7][9].