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AAN 2022 | AOC programs in development for DMD and myotonic dystrophy

Arthur Levin, PhD, Avidity Biosciences, La Jolla, CA, highlights ongoing programs evaluating antibody-oligonucleotide conjugates (AOCs) for the treatment of Duchenne muscular dystrophy (DMD) and myotonic dystrophy. Dr Levin first gives an overview of the role of these AOCs in the treatment of several forms of DMD and the value of using the transferrin receptor to deliver therapeutic agents. Following this, Dr Levin goes on to explain the biology behind the development of myotonic dystrophy and further discusses the potential of AOC 1001 for the treatment of this neuromuscular disorder, which is being evaluated in the Phase I/II MARINA trial (NCT05027269). This interview took place at the American Academy of Neurology 2022 Congress in Seattle, WA.

Disclosures

Dr Levin is an employee of Avidity Biosciences, a Board member at Stoke Therapeutics, and a SAB member at Atalanta Therapeutics.