DYNE-245

DYNE-245 is an investigational antibody-oligonucleotide conjugate (AOC) being developed by Dyne Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 45 skipping. Utilizing Dyne's proprietary FORCE platform, the drug consists of a phosphorodiamidate morpholino oligomer (PMO) payload conjugated to a fragment antibody (Fab) that targets the transferrin receptor 1 (TfR1). TfR1 is highly expressed on muscle cells, and this targeting mechanism is designed to overcome the delivery challenges of traditional antisense oligonucleotides by enhancing uptake into skeletal, cardiac, and smooth muscle. Once internalized, the PMO promotes the skipping of exon 45 during pre-mRNA splicing, which restores the reading frame of the dystrophin gene and allows for the production of a truncated but functional dystrophin protein.