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ION 356

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Drug Profile

ION 356

Alternative Names: ION-356

Latest Information Update: 25 Apr 2024

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Gene silencing; PLP1 protein expression inhibitors
  • Orphan Drug Status

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

    Yes - Pelizaeus-Merzbacher disease
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Pelizaeus-Merzbacher disease

Most Recent Events

  • 10 Apr 2024 Phase-I clinical trials in Pelizaeus-Merzbacher disease (In children, In adolescents) in France (Intrathecal) (NCT06150716)
  • 10 Apr 2024 Phase-I clinical trials in Pelizaeus-Merzbacher disease (In children, In adolescents) in Netherlands (Intrathecal) (NCT06150716)
  • 04 Oct 2023 Ionis Pharmaceuticals plans a first-in-patient trial for Pelizaeus-Merzbacher disease in 2024

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