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zeleciment rostudirsen
- Development stage
- Phase 3
- Lead developer
- Dyne Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
Zeleciment rostudirsen (DYNE-251) is an investigational phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that targets the transferrin receptor 1 (TfR1). Developed by Dyne Therapeutics using their proprietary FORCE platform, it is designed to treat Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 51 skipping. By binding to TfR1, the Fab moiety facilitates the delivery of the PMO into muscle cells, where the PMO promotes the skipping of exon 51 during dystrophin pre-mRNA splicing. This process aims to restore the reading frame and allow for the production of a truncated but functional dystrophin protein, potentially improving muscle function and slowing disease progression.
- Brand names
- zeleciment rostudirsen
- Other names
- zeleciment rostudirsenDYNE-251DYNE251DYNE 251
02
Targets
Dystrophin pre-messenger RNA exon 51TFRC (Transferrin Receptor)
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