AAN 2022 | AOC for the treatment of Duchenne muscular dystrophy in exon 44 skippers

Duchenne Muscular Dystrophy (DMD) is an X-linked muscular disease caused by predominantly out-of-frame mutations in the dystrophin gene that prevent the expression of a functional dystrophin protein. Arthur Levin, PhD, Avidity Biosciences, La Jolla, CA, describes the antibody-oligonucleotide conjugate (AOC) program targeting different mutations that are amenable to skipping, including exon 44. Data presented suggest that AOCs targeting exon 44 can skip exon 44 and produce functional dystrophin protein in vitro. The next step is to conduct clinical trials in this underserved population of boys with DMD exon 44 mutations. This interview took place at the American Academy of Neurology 2022 Congress in Seattle, WA.