Zotadirsen
Zotadirsen is the core component of the AOC drug Delpacibart zotadirsen (AOC1044) (HY-177564), consisting of a phosphorodiamidate morpholino oligonucleotide (PMO) targeting exon 44 of the dystrophin gene and an SMCC linker (HY-42360). Upon conjugation with the anti-TfR1 antibody Delpacibart (HY-P990051), Zotadirsen exerts splicing regulatory activity and mediates exon skipping. Zotadirsen can be used in studies related to AOC synthesis.
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- Masse moléculaire:8467.80
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Stockage:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Voir tous les produits spécifiques à Isoform Antibody-Oligonucleotide Conjugates (AOCs)
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Activité biologique
Description
IC50 & Target
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Oligonucleotide-Linker Conjugates for AOC |
In Vivo
Zotadirsen, the active oligonucleotide component of Delpacibart zotadirsen (5–10 mg/kg), induces exon 44 skipping in a dose-dependent manner, elevates dystrophin expression in DMD44 subjects, reduces serum markers of muscle injury, and exhibits favorable safety and tolerability profiles[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Essai clinique
| NCT Number | Sponsor | Condition | Start Date |
Phase
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|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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Appearance Solid
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Masse moléculaire 8467.80
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Color White to off-white
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SMILES
[Zotadirsen]
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Livraison
Room temperature in continental US; may vary elsewhere.
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Stockage
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Pureté et documentation
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Fiche technique (274 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Instruction de manipulation (2659 KB)
Références
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)