Wave Life Sciences reports positive interim Phase 2 data for WVE-N531: adjusted dystrophin 9.0% in DMD exon 53

Wave Life Sciences Ltd.

Mean muscle content-adjusted dystrophin expression 9.0% (range 4.6-13.9%); unadjusted 5.5% (range 3.3-8.3%) in ambulatory boys.
Dystrophin isoforms consistent with milder Becker DMD; 89% of ambulatory participants achieved ≥5% adjusted dystrophin.
WVE-N531 safe and well tolerated; all treatment-related AEs mild, no serious AEs or discontinuations.
Muscle concentration ~41,000 ng/g; 61-day half-life supports monthly dosing; preclinical data suggest higher concentration in heart/diaphragm.
Wave expects regulatory feedback and complete 48-week FORWARD-53 data in 1Q 2025.

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