delpacibart braxlosiran

Delpacibart braxlosiran (also known as AOC 1020 or del-brax) is an investigational antibody-oligonucleotide conjugate developed by Avidity Biosciences for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It is designed to address the underlying cause of FSHD, which is the abnormal expression of the DUX4 gene. Delpacibart braxlosiran consists of a proprietary monoclonal antibody that targets transferrin receptor 1 (TfR1) on muscle cells and is conjugated with a small interfering RNA (siRNA) that specifically targets DUX4 mRNA. This dual mechanism enables targeted delivery and silencing of DUX4 in muscle tissue, reducing both DUX4 mRNA and protein levels. Clinical trials have shown significant reductions in DUX4-regulated genes, improvements in muscle function, and favorable safety/tolerability profiles. The drug has received Orphan Drug designation from both FDA and EMA as well as Fast Track status from FDA for FSHD[2][3][5][6][7].

Brand names

del-brax

Other names

del-braxdelpacibart braxlosiranAOC 1020AOC1020AOC-1020