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ION 440

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ION 440

Alternative Names: ION-440

Latest Information Update: 28 Oct 2024

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Gene silencing; Methyl-CpG-binding protein 2 expression 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

  • Phase I/II Lubs X-linked mental retardation syndrome

Most Recent Events

  • 22 Oct 2024 Phase-I/II clinical trials in Lubs X-linked mental retardation syndrome (In adults, In children, In the elderly, In adolescents) in USA (Intrathecal) (NCT06430385)
  • 28 May 2024 Ionis Pharmaceuticals plans a phase I/II ATTUNE trial for Lubs-X-linked-mental-retardation-syndrome (In children, In adolescent, In adults, In elderly) in August 2024 (Intrathecal) (NCT06430385, EudraCT2023-507192-22, ION440CS1)
  • 04 Oct 2023 Ionis Pharmaceuticals has patent protection for Methods for modulating mecp2 expression for ION 440

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