Drug intelligence / Profile preview

ABHD6-targeting ASO

Development stage
Preclinical
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, Intraperitoneal
01

Overview

ABHD6-targeting ASO is an antisense oligonucleotide designed to selectively knock down the expression of alpha/beta hydrolase domain 6 (ABHD6), a serine hydrolase that regulates the levels of monoacylglycerols, including the endocannabinoid 2-arachidonoylglycerol (2-AG). Developed through a collaboration between academic researchers and Ionis Pharmaceuticals (formerly Isis Pharmaceuticals), these ASOs (such as ISIS 558482) target ABHD6 mRNA for degradation, primarily in peripheral tissues like the liver and adipose tissue. Preclinical research has demonstrated that reducing ABHD6 levels protects against high-fat diet-induced obesity, hepatic steatosis, and insulin resistance. Additionally, ABHD6 knockdown has been shown to prevent the progression of metabolic dysfunction-associated steatotic liver disease (MASLD) to hepatocellular carcinoma (HCC) by altering lysosomal lipid homeostasis and autophagy.

Other names
ABHD6 ASOABHD-6 ASOABHD 6 ASOalpha/beta hydrolase domain 6 antisense oligonucleotide
02

Targets

ABHD6 (Alpha/beta-hydrolase domain-containing protein 6)

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