DYNE-253

DYNE-253 is a preclinical development candidate being advanced by Dyne Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping. Built on Dyne's proprietary FORCE platform, the drug is a targeted oligonucleotide conjugate consisting of a phosphorodiamidate morpholino oligomer (PMO) payload linked to an antigen-binding fragment (Fab). The Fab moiety specifically targets the transferrin receptor 1 (TfR1), which is highly expressed on the surface of muscle cells, facilitating the delivery of the PMO into skeletal, cardiac, and smooth muscle tissues. Once internalized, the PMO is designed to promote the skipping of exon 53 during the splicing of the dystrophin pre-mRNA, thereby restoring the reading frame and allowing for the production of a truncated but functional dystrophin protein. DYNE-253 is currently undergoing IND-enabling studies as part of a broader DMD franchise.