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WVE-008 (Wave Life Sciences, 628545)
- 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, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
WVE-008 (PNPLA3 GalNAc-AIMer) is an investigational GalNAc-conjugated, subcutaneously delivered RNA editing oligonucleotide (AIMer) developed by Wave Life Sciences. It is designed to treat liver diseases associated with the homozygous PNPLA3 I148M genetic variant, such as metabolic dysfunction-associated steatohepatitis (MASH) and hepatic steatosis. WVE-008 utilizes endogenous adenosine deaminase acting on RNA (ADAR) enzymes to perform precise A-to-I (adenosine-to-inosine) editing on the mutant PNPLA3 I148M transcripts. This editing corrects the genetic mutation, restoring the production of functional wild-type PNPLA3 protein and promoting lipid mobilization to reduce hepatic fat accumulation. Unlike silencing approaches (such as siRNAs) which can worsen liver disease by completely knocking down PNPLA3, WVE-008's RNA editing approach aims to restore physiological lipid metabolism.
- Other names
- PNPLA3 GalNAc-AIMerPNPLA-3 GalNAc-AIMerPNPLA 3 GalNAc-AIMerPNPLA3 RNA editing candidatePNPLA-3 RNA editing candidatePNPLA 3 RNA editing candidate
02
Targets
ADAR (Adenosine deaminases acting on RNA (ADAR) family)
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