Vesleteplirsen

Vesleteplirsen is an investigational antisense oligonucleotide drug developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. It utilizes a next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) chemistry, which enhances cell penetration and increases exon skipping efficiency compared to earlier PMO-based therapies. The mechanism of action involves binding to pre-mRNA and altering splicing to skip exon 51, thereby restoring the open reading frame and enabling production of functional dystrophin protein. Vesleteplirsen was administered intravenously in clinical trials and demonstrated increased dystrophin expression but was associated with adverse events such as hypomagnesemia and kidney function decline. Development was discontinued in late 2024 following risk-benefit analysis, FDA feedback, and changes in the DMD therapeutic landscape[1][2][5][6][7][8].

Other names

vesleteplirsen