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Phase 1/2 trial found significant increases in dystrophin levels with del-zota, indicating potential for treating DMD44.
Background/Purpose: Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene. Delpacibart zotadirsen, or del-zota (AOC: 1,044), is an antibody-oligonucleotide conjugate (AOC) comprised of an anti-transferrin receptor 1 (TfR1) antibody conjugated to an exon 44-skipping phosphorodiamidate morpholino oligomer (PMO). Del-zota is designed to restore the dystrophin reading frame and produce functional, internally truncated dystrophin protein in individuals with mutations amenable to exon 44 skipping (DMD44).
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Veerapandiyan et al. (2025) studied this question.
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