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SRP 1001

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Drug Profile

SRP 1001

Alternative Names: ARO-DUX4; SRP-1001

Latest Information Update: 16 Feb 2025

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At a glance

  • Originator Arrowhead Pharmaceuticals
  • Class Small interfering RNA
  • Mechanism of Action DUX4L1 protein 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 Facioscapulohumeral muscular dystrophy

Most Recent Events

  • 10 Feb 2025 Arrowhead Pharmaceuticals completes global licensing and collaboration agreement with Sarepta Therapeutics for multiple clinical and preclinical programme
  • 27 Nov 2024 Arrowhead Pharmaceuticals enters into global licensing and collaboration agreement with Sarepta Therapeutics for multiple clinical and preclinical siRNA programme
  • 04 Sep 2023 Phase-I/II clinical trials in Facioscapulohumeral muscular dystrophy in New Zealand (IV) prior to September 2023 (Arrowhead Pharmaceuticals pipeline, September 2023) (NCT06131983)

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