AAN 2022 | AOCs in neuromuscular disorders: Duchenne muscular dystrophy

Arthur Levin, PhD, Avidity Biosciences, La Jolla, CA, discusses antibody-oligonucleotide conjugates (AOCs) and their role in the treatment of neuromuscular disorders. Dr Levin first explains the mechanism of action of AOCs and the challenges associated with delivering these agents to cells. In the case of neuromuscular disorders, the AOCs are built around a monoclonal antibody to the transferrin receptor. A study on a mouse model of Duchenne muscular dystrophy (DMD) showed that using a monoclonal antibody to the transferrin receptor can effectively deliver an oligonucleotide therapeutic. This interview took place at the American Academy of Neurology 2022 Congress in Seattle, WA.