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Last update 15 Nov 2025
AOC-Pompe disease(Avidity Biosciences)
Last update 15 Nov 2025
Overview
Basic Info
Drug Type Antibody oligonucleotide conjugates |
Synonyms- |
Target |
Action inhibitors, antagonists |
Mechanism GYS1 gene inhibitors, TfR1 antagonists(Transferrin receptor protein 1 antagonists) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
License Organization- |
Drug Highest PhaseDiscontinuedPreclinical |
First Approval Date- |
Regulation- |
Structure/Sequence
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R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Glycogen Storage Disease Type II | Preclinical | - | - |
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Clinical Result
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