Antibody-Oligonucleotide Conjugates (AOCs)

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.

Etedesiran is a component of the AOC drug Delpacibart etedesiran, formed by the reaction of an siRNA that induces cleavage of mRNA encoding myotonic dystrophy protein kinase (MTPK or DMPK ) with SMCC linker.

Braxlosiran is a component of the AOC reagent Delpacibart braxlosiran, consisting of an siRNA targeting DUX4 (double homeobox protein) and SMCC linker.

Zotadirsen is the core component of the AOC drug Delpacibart zotadirsen (AOC1044), consisting of a phosphorodiamidate morpholino oligonucleotide (PMO) targeting exon 44 of the dystrophin gene and an SMCC linker.