Drug intelligence / Profile preview

SCD1 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
Intraperitoneal
01

Overview

SCD1 ASO is an antisense oligonucleotide drug that targets and inhibits the mRNA of stearoyl-CoA desaturase 1 (SCD1), a key enzyme catalyzing the synthesis of monounsaturated fatty acids from saturated fatty acids. By reducing SCD1 expression, SCD1 ASO lowers SCD1 activity in liver and adipose tissues, leading to decreased hepatic steatosis, reduced triglycerides, and prevention of diet-induced obesity and insulin resistance in rodent models. The drug works through gene silencing, specifically reducing SCD1 mRNA levels and enzymatic activity in a dose- and time-dependent manner. However, SCD1 inhibition using ASO can also lead to adverse effects such as increased proinflammatory signaling and accelerated atherosclerosis under certain dietary conditions. This drug is primarily investigated for the treatment and prevention of metabolic syndrome, obesity, hepatic insulin resistance, and related metabolic disorders[2][3][4][5][7].

02

Targets

SCD1 (Stearoyl-CoA desaturase 1)

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