Last update 27 Sep 2025

Rett Syndrome (ACADIA/Stoke Therapeutics)

Overview

Basic Info

Drug Type
ASO
Synonyms
Antisense Oligonucleotide (ASO) Programs, Antisense Oligonucleotide Programs (ACADIA), MECP2 ASO (ACADIA/Stoke Therapeutics)
Target
MECP2
Action
modulators
Mechanism
MECP2 modulators(Methyl-CpG-binding protein 2 modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersOther Diseases
Active Indication-
Inactive Indication
Rett Syndrome
Originator Organization
ACADIA Pharmaceuticals, Inc.ACADIA Pharmaceuticals, Inc.Stoke Therapeutics, Inc.Stoke Therapeutics, Inc.
Active Organization-
Inactive Organization
ACADIA Pharmaceuticals, Inc.ACADIA Pharmaceuticals, Inc.Stoke Therapeutics, Inc.Stoke Therapeutics, Inc.
License Organization
Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals, Inc.Roche Holding AGRoche Holding AG
Drug Highest PhaseDiscontinuedPreclinical
First Approval Date-
Regulation-

Structure/Sequence

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Related

100 Clinical Results associated with Rett Syndrome (ACADIA/Stoke Therapeutics)
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100 Translational Medicine associated with Rett Syndrome (ACADIA/Stoke Therapeutics)
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100 Patents (Medical) associated with Rett Syndrome (ACADIA/Stoke Therapeutics)
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100 Deals associated with Rett Syndrome (ACADIA/Stoke Therapeutics)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Rett SyndromePreclinical
United States
Stoke Therapeutics, Inc.Stoke Therapeutics, Inc.
30 Oct 2023
Rett SyndromePreclinical
United States
ACADIA Pharmaceuticals, Inc.ACADIA Pharmaceuticals, Inc.
30 Oct 2023
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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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