2022 年 39 巻 4 号 p. 535-538
Antisense oligonucleotides (AON) therapy is developed to treat Duchenne muscular dystrophy (DMD) patinets. We have reported that systemic delivery of AONs targeting exon 6 and 8 of the canine DMD gene to a dystrophin–deficient canine animal model, efficiently restored functional dystrophin proteins and improved phenotypes of affected dogs. We, then, optimized AO sequences, which allow exon 53 skipping of the human DMD gene, together with Nippon Shinyaku Co. Ltd. We carried an early phase clinical trial of exon 53 skipping drug among DMD patients with highly effective exon skipping and without serious adverse events, then phase I/II trial in Japan and phase II trial in US were carried by either Nippon Shinyaku Co. Ltd. or NS Pharma, Inc. Based on favorable results in these clinical trials, exon 53 skipping drug, calles as viltolarsen, has been approved both in Japan and in US on March and August in 2020, respectively.
