The myotonic dystrophy type 1 drug development pipeline: 2022 edition

Drug Discov Today. 2023 Mar;28(3):103489. doi: 10.1016/j.drudis.2023.103489. Epub 2023 Jan 9.


The beginning of the 20th decade has witnessed an increase in drug development programs for myotonic dystrophy type 1 (DM1). We have collected nearly 20 candidate drugs with accomplished preclinical and clinical phases, updating our previous drug development pipeline review with new entries and relevant milestones for pre-existing candidates. Three interventional first-in-human clinical trials got underway with distinct drug classes, namely AOC 1001 and DYNE-101 nucleic acid-based therapies, and the small molecule pitolisant, which joins the race toward market authorization with other repurposed drugs, including tideglusib, metformin, or mexiletine, already in clinical evaluation. Furthermore, newly disclosed promising preclinical data for several additional nucleic-acid therapeutic candidates and a CRISPR-based approach, as well as the advent into the pipeline of novel therapeutic programs, increase the plausibility of success in the demanding task of providing valid treatments to patients with DM1.

Keywords: CTG repeat expansion; clinical trial; drug development; drug repurposing; gene therapy; myotonic dystrophy; nucleic acids therapeutics; small molecule.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Development
  • Humans
  • Myotonic Dystrophy* / drug therapy