Drug intelligence / Profile preview

WVE-008

Development stage
Preclinical
Lead developer
Wave Life Sciences
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

WVE-008 is an investigational GalNAc-conjugated RNA editing oligonucleotide (AIMer) developed by Wave Life Sciences for the treatment of liver disease associated with the PNPLA3-I148M genetic variant. Utilizing proprietary RNA editing technology, WVE-008 is designed to correct the single nucleotide polymorphism (SNP) in the PNPLA3 mRNA, thereby restoring the production of functional PNPLA3 protein. This restoration aims to improve lipid mobilization and reverse liver pathology, including steatosis, inflammation, and fibrosis. As of late 2025, it is a clinical candidate with a Clinical Trial Application (CTA) filing planned for 2026. The therapy specifically targets homozygous carriers of the I148M variant, who face a significantly higher risk of liver-related mortality.

02

Targets

PNPLA3 I148M (Patatin-like phospholipase domain-containing protein 3 I148M variant)

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