Braxlosiran

Braxlosiran is a small nucleic acid that targets DUX4 (double homeobox protein) and can be used to synthesize AOC drugs, such as Delpacibart braxlosiran (HY-177566) . Abnormal expression of the DUX4 protein leads to changes in gene expression in muscle cells, and these changes are associated with the loss of muscle function in facioscapulohumeral muscular dystrophy (FSHD) [1].