Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study

Antisense oligonucleotide-induced exon skipping, which is being studied for the treatment of Duchenne muscular dystrophy (DMD), allows synthesis of partially functional dystrophin. Patients amenable to exon 53 skipping form the second-largest population after patients amenable to exon 51 skipping. Therefore, in 2009, the National Center of Neurology and Psychiatry and Nippon Shinyaku Company collaborated to jointly develop an exon 53-skipping drug; an investigator-initiated clinical trial was started in June 2013 (NCT02081625) to examine the efficacy of NS-065/NCNP-01, a morpholino-based antisense oligonucleotide that facilitates skipping of exon 53 of the dystrophin gene.