BMN-351

BMN-351 is a next-generation antisense oligonucleotide (ASO) therapy under investigation for the treatment of Duchenne Muscular Dystrophy (DMD) in patients with mutations amenable to exon 51 skipping. It is a fully modified 18-mer oligonucleotide with a TEG modification at its 5' end that binds to exon 51 of dystrophin pre-mRNA. By inducing skipping of exon 51 during mRNA splicing, BMN-351 enables the production of an internally deleted but functional dystrophin protein, potentially alleviating symptoms and resulting in a milder disease presentation. The drug is administered intravenously and is currently being evaluated in Phase 1/2 clinical trials for safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on dystrophin expression and physical function[1][3][6][7][8].