DYNE-244

DYNE-244 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 44 skipping. The therapeutic is engineered using Dyne's proprietary FORCE platform, which consists of a phosphorodiamidate morpholino oligomer (PMO) payload conjugated to a fragment antibody (Fab) that specifically binds to the transferrin receptor 1 (TfR1). This targeting strategy is designed to overcome the delivery challenges of traditional antisense oligonucleotides by facilitating robust uptake into skeletal, cardiac, and smooth muscle tissues. Once delivered to the nucleus of muscle cells, the PMO promotes exon skipping during pre-mRNA splicing, thereby restoring the genetic reading frame and enabling the production of a truncated but functional dystrophin protein.