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SRP 1004

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Drug Profile

SRP 1004

Alternative Names: ARO-ATXN2; SRP-1004

Latest Information Update: 16 Feb 2025

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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/II Spinocerebellar ataxias

Most Recent Events

  • 10 Feb 2025 Arrowhead Pharmaceuticals completes global licensing and collaboration agreement with Sarepta Therapeutics for multiple clinical and preclinical siRNA programme
  • 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/II clinical trials in Spinocerebellar ataxias in New Zealand (Intrathecal) (NCT06672445) (EudraCT-2024-514763-25),

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