brogidirsen

Brogidirsen is an investigational antisense oligonucleotide designed as a dual-targeting phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 44 skipping. It contains two linked sequences that bind to separate sites within exon 44 of the DMD gene, promoting exon skipping and enabling production of partially functional dystrophin protein. Brogidirsen is administered intravenously and has demonstrated dose-dependent increases in dystrophin expression and stabilization or improvement in motor function in early-phase clinical trials. The drug has received FDA Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations[2][3][5][8].

Other names

brogidirsenNS-089/NCNP-02