Drug Profile

SRP 1001

Alternative Names: ARO-DUX4; SRP-1001

Latest Information Update: 20 Mar 2026

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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

02 Mar 2026 Arrowhead Pharmaceuticals has patent protection for SRP 1001 in USA
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

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