Last update 06 Nov 2025

ION-283

Overview

Basic Info

Drug Type
ASO
Synonyms
2¿-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 (GYS1) pre mRNA, ION 283, ION283
Target
GYS1
Action
inhibitors
Mechanism
GYS1 gene inhibitors, RNA interference
Therapeutic Areas
Nervous System DiseasesCongenital DisordersOther Diseases
Active Indication
Lafora Disease
Inactive Indication-
Originator Organization
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
Active Organization
Elpida TherapeuticsElpida Therapeutics
Inactive Organization
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
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-283
NCT06609889
/ RecruitingPhase 1/2IIT
A Phase 1/2 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
100 Clinical Results associated with ION-283
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100 Translational Medicine associated with ION-283
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100 Patents (Medical) associated with ION-283
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100 Deals associated with ION-283
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Lafora DiseasePhase 2
United States
Elpida TherapeuticsElpida Therapeutics
03 Dec 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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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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