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ION 581

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Drug Profile

ION 581

Alternative Names: ION 581; UBE3A gene expression stimulant; UBE3A-ATS targeting oligoneucleotides

Latest Information Update: 28 Jun 2022

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At a glance

  • Originator Baylor College of Medicine; Isis Pharmaceuticals
  • Developer Baylor College of Medicine; Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Genetic transcription inhibitors
  • 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

  • No development reported Angelman syndrome

Most Recent Events

  • 28 Jun 2022 No recent reports of development identified for preclinical development in Angelman-syndrome in USA
  • 24 Jan 2020 Preclinical development is ongoing in Angelman-syndrome in USA
  • 07 Jan 2020 Ionis plans a clinical trial for Angelman Syndrome in 2020

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