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ION 440

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Drug Profile

ION 440

Alternative Names: ION-440

Latest Information Update: 06 Jun 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

  • Preclinical X-linked genetic disorders

Most Recent Events

  • 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
  • 04 Oct 2023 Preclinical trials in X-linked genetic disorders in USA (unspecified route)

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