Wave Life Sciences Ltd. announced on its ClinicalTrials.gov website that a phase 2 clinical trial of WVE-N531 (Exon 53 Skipping Therapy) will begin in November 2025 with 175 participants in Jordan and the United Kingdom.
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What is WVE-N531 Exon Skipping Therapy
WVE-N531 is an investigational exon skipping therapy being developed as a potential therapy for patients with DMD amenable to exon 53 skipping. WVE-N531 was designed using Wave’s best-in-class oligonucleotide chemistry modifications, including PN backbone chemistry. WVE-N531 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration.
Read More: Mutations and Deletions Amenable to Exon 53 Skipping Therapies for Duchenne Muscular Dystrophy
WVE-N531 Exon Skipping Therapy Phase 2 – NCT07209332
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531.
Study Start (Estimated): 2025-11
Primary Completion (Estimated): 2029-03
Study Completion (Estimated): 2029-03
Number of Participants (Estimated): 175
Study Type: Interventional
Location: Jordan, United Kingdom
Ages Eligible for Study: Child, Adult, Older Adult
Principal inclusion and exclusion criteria. – NCT07209332 –
How to Participate in the WVE-N531 Clinical Trial
To participate in the WVE-N531 exon skipping therapy phase 2 clinical trial, you can use the contact information below. Learn More: How to Participate in Clinical Trials for Duchenne (DMD)?
Name: Clinical Operations
Phone Number: 855-215-4687
Email: [email protected]
Read More: Clinical Trials for Duchenne (List of All Researches)
