Last update 21 Nov 2024

ION-283

Overview

Basic Info

Drug Type
ASO
Synonyms-
Target
GYS1
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.
Drug Highest PhasePhase 1/2
First Approval Date-
Regulation-
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Structure

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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
US
Elpida TherapeuticsElpida Therapeutics
01 Dec 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

No Data

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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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