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Last update 09 Dec 2025
ION-440
Last update 09 Dec 2025
Overview
Basic Info
Drug Type ASO |
Synonyms 2'-O-(2-Methoxyethyl) modified antisense oligonucleotide against MECP2 pre-mRNA, ION 440 |
Target |
Action inhibitors |
Mechanism MECP2 inhibitors(Methyl-CpG-binding protein 2 inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhasePhase 1/2 |
First Approval Date- |
RegulationOrphan Drug (United States), Orphan Drug (European Union) |
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Structure/Sequence
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Related
1
Clinical Trials associated with ION-440NCT06430385
A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome
100 Clinical Results associated with ION-440
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100 Translational Medicine associated with ION-440
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100 Patents (Medical) associated with ION-440
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100 Deals associated with ION-440
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R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Lubs X-Linked Mental Retardation Syndrome | Phase 2 | United States | 21 Oct 2024 | |
| Lubs X-Linked Mental Retardation Syndrome | Phase 2 | Austria | 21 Oct 2024 | |
| Lubs X-Linked Mental Retardation Syndrome | Phase 2 | France | 21 Oct 2024 |
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Clinical Result
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Core Patent
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Clinical Trial
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Approval
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Regulation
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