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STOKE ASO 1

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Drug Profile

STOKE ASO 1

Alternative Names: STOKE-ASO-1

Latest Information Update: 14 May 2026

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At a glance

  • Originator Stoke Therapeutics
  • Class Antisense oligonucleotides; Eye disorder therapies
  • Mechanism of Action Methyl-CpG-binding protein 2 expression stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Research Rett syndrome

Most Recent Events

  • 03 Sep 2025 Stoke Therapeutics terminates its licensing agreement with Acadia Pharmaceuticals for MECP2 and the undisclosed neurodevelopmental programs
  • 26 Feb 2025 Early research in Rett syndrome in USA (unspecified route) before February 2025 (ACADIA Pharmaceuticals pipeline, February 2025)

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