Braxlosiran

Braxlosiran is a small nucleic acid targeting DUX4 (double homeobox protein), utilized in the synthesis of AOC drugs such as Delpacibart braxlosiran. Abnormal expression of the DUX4 protein leads to altered gene expression in muscle cells, which is associated with muscle function loss in facioscapulohumeral muscular dystrophy (FSHD) .