Last update 01 Nov 2025

ION-440

Overview

Basic Info

Drug Type
ASO
Synonyms
ION 440
Target
MECP2
Action
modulators
Mechanism
MECP2 modulators(Methyl-CpG-binding protein 2 modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersOther Diseases
Active Indication
Lubs X-Linked Mental Retardation Syndrome
Inactive Indication-
Originator Organization
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
Active Organization
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
Inactive Organization-
License Organization-
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationOrphan Drug (United States)
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Structure/Sequence

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Related

1
Clinical Trials associated with ION-440
NCT06430385
/ RecruitingPhase 1/2
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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IndicationHighest PhaseCountry/LocationOrganizationDate
Lubs X-Linked Mental Retardation SyndromePhase 2
United States
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
21 Oct 2024
Lubs X-Linked Mental Retardation SyndromePhase 2
France
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
21 Oct 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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Translational Medicine

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Deal

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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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