Last update 06 Nov 2025

Pelizaeus-Merzbacher Disease Therapeutics (Ionis Pharmaceuticals)

Overview

Basic Info

Drug Type
ASO
Synonyms
2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA
Target
PLP1
Action
inhibitors
Mechanism
PLP1 inhibitors(proteolipid protein 1 inhibitors)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Pelizaeus-Merzbacher Disease
Inactive Indication-
Originator Organization
Case Western Reserve UniversityCase Western Reserve University
Active Organization
Case Western Reserve UniversityCase Western Reserve University
Inactive Organization
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States), Orphan Drug (European Union)

Structure/Sequence

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Related

100 Clinical Results associated with Pelizaeus-Merzbacher Disease Therapeutics (Ionis Pharmaceuticals)
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100 Translational Medicine associated with Pelizaeus-Merzbacher Disease Therapeutics (Ionis Pharmaceuticals)
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100 Patents (Medical) associated with Pelizaeus-Merzbacher Disease Therapeutics (Ionis Pharmaceuticals)
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100 Deals associated with Pelizaeus-Merzbacher Disease Therapeutics (Ionis Pharmaceuticals)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Pelizaeus-Merzbacher DiseasePreclinical
United States
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.
01 Jul 2020
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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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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