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Research programme: antisense oligonucleotides - Stoke Therapeutics

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Drug Profile

Research programme: antisense oligonucleotides - Stoke Therapeutics

Alternative Names: TANGO ASO

Latest Information Update: 28 Mar 2024

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At a glance

  • Originator Stoke Therapeutics
  • Class Antiepileptic drugs; Antisense oligonucleotides; Eye disorder therapies; Hepatoprotectants; Neuroprotectants
  • Mechanism of Action Methyl-CpG-binding protein 2 expression stimulants; NAV1.1 voltage-gated sodium channel modulators; RNA interference; SYNGAP1 protein expression stimulants
  • Orphan Drug Status

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

    Yes - Dravet syndrome
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Rett syndrome
  • No development reported Dravet syndrome; Haematological disorders; Liver disorders; Neurological disorders; Optic atrophy

Most Recent Events

  • 28 Mar 2024 No recent reports of development identified for preclinical development in Dravet-syndrome in USA
  • 28 Mar 2024 No recent reports of development identified for preclinical development in Neurological-disorders in USA
  • 28 Feb 2023 Stoke Therapeutics delivers a notice of termination to Cold Spring Harbor Laboratory for the CSHL Agreement
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