Drug intelligence / Profile preview

ION547

Development stage: Phase 1
Lead developer: Ionis Pharmaceuticals
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Subcutaneous

Overview

ION547 is an investigational ligand-conjugated antisense oligonucleotide (LICA ASO) developed by Ionis Pharmaceuticals. It is designed to inhibit the expression of coagulation factor XII (FXII, also known as F12) through RNA interference mechanisms. The drug acts as a Factor XII inhibitor and is administered subcutaneously. Its primary indication under investigation has been coronary thrombosis, with a completed Phase 1 clinical trial evaluating its safety and tolerability in healthy subjects[1][2][3]. As of the latest updates, it has not received regulatory approval for any indication.

Targets

F12 (Coagulation Factor XII)

Beyond the preview

Go deeper on ION547.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on ION547.

Explore the full profile