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Drug Profile

ARO ATXN1

Alternative Names: ARO-ATXN1

Latest Information Update: 09 Dec 2024

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At a glance

Originator Arrowhead Pharmaceuticals
Class Small interfering RNA
Mechanism of Action Ataxin-1 expression modulators; 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

Preclinical 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
27 Nov 2024 Preclinical trials in Spinocerebellar ataxias in USA (Parenteral), before November 2024

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