WVE-N531
Duchenne Muscular Dystrophy (Exon 53)
Phase 1Active
Key Facts
About WaVe Life Sciences
Wave Life Sciences is a clinical-stage biotech focused on developing stereopure oligonucleotide therapeutics via its proprietary PRISM® platform. The company has built a diversified pipeline targeting both rare genetic disorders and common diseases, with key programs in obesity (WVE-007), alpha-1 antitrypsin deficiency (WVE-006), Duchenne muscular dystrophy, and Huntington's disease. Its strategy combines validated human genetics, a multi-modal RNA-targeting approach, and stereochemistry control to aim for best-in-class efficacy and tolerability. Recent positive interim Phase 1 data in obesity and strategic moves to accelerate its RNA editing portfolio underscore its momentum.
View full company profileOther Duchenne Muscular Dystrophy (Exon 53) Drugs
| Drug | Company | Phase |
|---|---|---|
| Viltolarsen (Viltepso) | Nippon Shinyaku | Approved |