AOC 1045

AOC 1045 is an investigational antibody-oligonucleotide conjugate (AOC) developed by Avidity Biosciences for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 45 skipping. The drug consists of a monoclonal antibody targeting the transferrin receptor 1 (TfR1) conjugated to a phosphorodiamidate morpholino oligomer (PMO). This design utilizes the TfR1-targeting antibody to facilitate the delivery of the PMO payload specifically into skeletal muscle and heart tissue. Once internalized, the PMO is designed to skip exon 45 of the dystrophin pre-mRNA, thereby restoring the reading frame and enabling the production of a truncated but functional dystrophin protein. AOC 1045 is part of Avidity's broader pipeline of AOCs aimed at addressing rare neuromuscular diseases by combining the tissue-targeting specificity of monoclonal antibodies with the genetic precision of oligonucleotide therapies.