Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy

Harry Wilton-Clark; Toshifumi Yokota

doi:10.3390/pharmaceutics15030778

Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy

Pharmaceutics. 2023 Feb 26;15(3):778. doi: 10.3390/pharmaceutics15030778.

Authors

Harry Wilton-Clark¹, Toshifumi Yokota¹

Affiliation

¹ Department of Medical Genetics, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Abstract

Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting 1/5000 boys globally, characterized by progressive muscle breakdown and eventual death, with an average lifespan in the mid-late twenties. While no cure yet exists for DMD, gene and antisense therapies have been heavily explored in recent years to better treat this disease. Four antisense therapies have received conditional FDA approval, and many more exist in varying stages of clinical trials. These upcoming therapies often utilize novel drug chemistries to address limitations of existing therapies, and their development could herald the next generation of antisense therapy. This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown.

Keywords: ATL1102; DS-5141B; NS-089/NCNP-02; SCAAV9.U7.ACCA; SRP-5051; WVE-N531; casimersen; eteplirsen; golodirsen; viltolarsen.

Publication types

Review

Grants and funding

H.W.C. is grateful for funding provided by the H Jean McDiarmaid Scholarship, endowed by the family of H Jean McDiarmaid and distributed by the University of Alberta. T.Y is grateful for the support provided by the Women and Children’s Health Research Institute (WCHRI), Canadian Institute of Health Research (CIHR), Heart and Stroke Foundation Canada, Defeat Duchenne Canada, and the United States Department of Defense.