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ARO-ATXN2

Oligonucleotide (siRNA) · Gene silencing: ATXN2 · developed by ARROWHEAD PHARMACEUTICALS

Phase 1 Most advanced trial

ARWR Developer

1 Registered trials

What is ARO-ATXN2?

ARO-ATXN2 is a small interfering RNA (siRNA) targeting ATXN2 mRNA, administered intrathecally. It is being studied in spinocerebellar ataxia type 2.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Oligonucleotide (siRNA)
Targets: ATXN2
Mechanism: Gene silencing: ATXN2
Therapy area: Neurology
Developer: ARROWHEAD PHARMACEUTICALS (ARWR)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT06672445	Phase 1	Spinocerebellar Ataxia Type 2	Active not recruiting	ARWR

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Spinocerebellar Ataxia Type 2

More from ARROWHEAD PHARMACEUTICALS

Plozasiran FDA approved ARC-521 Phase 1 ARC-AAT Phase 2 ARO-033 Phase 1 ARO-ALK7 Phase 1/2 ARO-ANG 3 Phase 2 ARO-APOC3 Phase 2 ARO-C3 Phase 1/2 ARO-CFB Phase 1 ARO-DIMERPA Phase 1/2 ARO-ENaC Phase 1/2 ARO-HBV Phase 1/2

Sources

ClinicalTrials.gov NCT06672445

Updated Oct 3, 2026. Something wrong or missing? Report it on the ARWR page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z