Delpacibart zotadirsen (Del-zota), an antibody oligonucleotide conjugate (AOC), consists of a monoclonal antibody (Delpacibart) that binds to the transferrin receptor 1 (TfR1) conjugated to a phosphorodiamidate morpholino conjugate (PMO), Delpacibart zotadirsen is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. Delpacibart zotadirsen is used for the study of myotonic dystrophy type 1 (DM1) [1].
In Vitro, Delpacibart zotadirsen is primarily used in the study of Duchenne muscular dystrophy with exon 44 skipping mutations (DMD44), and can cover both ambulatory and non-ambulatory patients aged 7-27 years[1].
References:
[1]. Compounds, pharmaceutical compositions, and methods of preparing compounds and of their use. WO2025177244A1
[2]. Etxaniz U, Marks I, Albin T, et al. AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy. Nucleic Acids Res. 2025;53(6):gkaf241.
In Vitro, Delpacibart zotadirsen is primarily used in the study of Duchenne muscular dystrophy with exon 44 skipping mutations (DMD44), and can cover both ambulatory and non-ambulatory patients aged 7-27 years[1].
References:
[1]. Compounds, pharmaceutical compositions, and methods of preparing compounds and of their use. WO2025177244A1
[2]. Etxaniz U, Marks I, Albin T, et al. AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy. Nucleic Acids Res. 2025;53(6):gkaf241.