The myotonic dystrophy type 1 drug development pipeline: 2022 edition.
Drug Discov Today
; 28(3): 103489, 2023 03.
Article
de En
| MEDLINE
| ID: mdl-36634841
ABSTRACT
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.
Mots clés
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Sujet principal:
Dystrophie myotonique
Limites:
Humans
Langue:
En
Journal:
Drug Discov Today
Sujet du journal:
FARMACOLOGIA
/
TERAPIA POR MEDICAMENTOS
Année:
2023
Type de document:
Article
Pays d'affiliation:
Espagne