生产企业：Novartis
Delpacibart braxlosiran (del-brax; AOC-1020) is an investigational antibody oligonucleotide conjugate (AOC) developed by Novartis (acquired from Avidity Biosciences) for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It delivers siRNA to suppress aberrant DUX4 expression in muscle cells. The Phase I/II FORTITUDE trial met its primary biomarker endpoint, and a Phase III trial (FORTITUDE-3) is currently enrolling. Del-brax has received FDA Orphan Drug and Fast Track designations, and EMA Orphan Drug designation.
Delpacibart braxlosiran (del-brax; AOC-1020) is an investigational antibody oligonucleotide conjugate (AOC) developed by Novartis (acquired from Avidity Biosciences) for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It delivers siRNA to suppress aberrant DUX4 expression in muscle cells. The Phase I/II FORTITUDE trial met its primary biomarker endpoint, and a Phase III trial (FORTITUDE-3) is currently enrolling. Del-brax has received FDA Orphan Drug and Fast Track designations, and EMA Orphan Drug designation.
Del-brax is an antibody oligonucleotide conjugate (AOC) that combines the tissue specificity of monoclonal antibodies with the precision of oligonucleotides to enable targeted delivery of siRNA. It suppresses aberrant DUX4 expression in muscle cells, addressing the root cause of facioscapulohumeral muscular dystrophy (FSHD).
