Drug intelligence / Profile preview

ION839

Development stage: Unknown
Lead developer: AstraZeneca
Modality: miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Subcutaneous

Overview

ION839 (also known as AZD-2693) is an **investigational antisense oligonucleotide (ASO) therapeutic** developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH)[3][5][1]. It is specifically engineered to inhibit the expression of **patatin-like phospholipase domain-containing protein 3 (PNPLA3)**, a genetic risk factor driving disease pathogenesis in patients with the homozygous PNPLA3 I148M variant. The drug utilizes **N-acetylgalactosamine (GalNAc) conjugation** to enable targeted hepatic delivery via the asialoglycoprotein receptor (ASGPR), maximizing uptake by hepatocytes and enhancing therapeutic specificity for liver disease[3]. Mechanistically, ION839 reduces hepatic PNPLA3 mRNA and protein, leading to decreased liver fat, improved inflammatory and lipid biomarkers, and potential histological improvement in MASH. Its development represents a genetically informed approach to address a subset of patients with high unmet need due to rapid progression to advanced liver disease[3][5].

Other names: ION 839ION839ION-839AZD-2693AZD2693AZD 2693IONIS-AZ6-2.5-LRxIONIS-AZ-6-2.5-LRxIONIS-AZ 6-2.5-LRx

Targets

PNPLA3 (Patatin-like phospholipase domain-containing protein 3)

Beyond the preview

Go deeper on ION839.

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 ION839.

Explore the full profile