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WVE-008 (Wave Life Sciences)
- Development stage
- Preclinical
- Lead developer
- Wave Life Sciences
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
- 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 diseases associated with the PNPLA3 I148M genetic variant, such as metabolic dysfunction-associated steatohepatitis (MASH) and alcohol-associated liver disease. The therapeutic utilizes endogenous adenosine deaminase acting on RNA (ADAR) enzymes to edit the single-nucleotide mutation in PNPLA3 I148M transcripts, restoring the production of functional wild-type PNPLA3 protein. By correcting the transcript rather than silencing the gene, WVE-008 aims to preserve the physiological role of PNPLA3 in hepatic lipid homeostasis and retinol metabolism, thereby reducing intracellular lipid droplet density, inflammation, and fibrosis.
- Other names
- Anti-PNPLA3 GalNAc-AIMerAnti-PNPLA-3 GalNAc-AIMerAnti-PNPLA 3 GalNAc-AIMerPNPLA3 AIMerPNPLA-3 AIMerPNPLA 3 AIMerPNPLA3 GalNAc AIMerPNPLA-3 GalNAc AIMerPNPLA 3 GalNAc AIMer
02
Targets
ADAR (Adenosine deaminases acting on RNA (ADAR) family)PNPLA3 (Patatin-like phospholipase domain-containing protein 3)
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