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ARO ATXN2

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Drug Profile

ARO ATXN2

Alternative Names: ARO-ATXN2

Latest Information Update: 16 Dec 2024

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At a glance

  • Originator Arrowhead Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action Ataxin-2 expression inhibitors; RNA interference
  • 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 Spinocerebellar ataxias

Most Recent Events

  • 27 Nov 2024 Arrowhead Pharmaceuticals enters into global licensing and collaboration agreement with Sarepta Therapeutics for multiple clinical and preclinical siRNA programme
  • 26 Nov 2024 Phase-I clinical trials in Spinocerebellar ataxias in New Zealand (Intrathecal) (NCT06672445) (EudraCT-2024-514763-25)
  • 07 Nov 2024 Preclinical trials in Spinocerebellar ataxias in USA (Intrathecal)

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