Drug intelligence / Profile preview

ASO-Fsp27

Development stage
Preclinical
Lead developer
Washington University School of Medicine
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
01

Overview

ASO-Fsp27 is an antisense oligonucleotide designed to silence the expression of fat-specific protein 27 (FSP27, also known as CIDEC), a lipid droplet-associated protein involved in triglyceride storage and adipocyte metabolism. FSP27 promotes the formation of large unilocular lipid droplets in white adipocytes, thereby facilitating efficient energy storage and restricting lipolysis. Inhibition or silencing of FSP27 leads to increased lipolysis, reduced triglyceride accumulation, and improved metabolic parameters such as glycemic control in preclinical models. ASO-Fsp27 has been studied primarily for its potential therapeutic effects on obesity-related metabolic disorders, including nonalcoholic steatohepatitis (NASH) and insulin resistance. Preclinical studies have shown that treatment with ASO-Fsp27 can ameliorate diet-induced steatohepatitis and improve glycemic control in mouse models of obesity[4][2]. The drug is typically investigated in combination with other agents such as fenofibrate for enhanced efficacy[4].

02

Targets

CIDEC (Fat-specific Protein 27)

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