Drug intelligence / Profile preview

thrap3 antisense oligonucleotide

Development stage
Preclinical
Lead developer
Ionis Pharmaceuticals
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

Thrap3 antisense oligonucleotide is an experimental RNA-based therapy designed to knock down the expression of thyroid hormone receptor-associated protein 3 (Thrap3). Thrap3 is a transcriptional coregulator that interacts with PPARγ when it is phosphorylated at Ser273 by CDK5, a modification associated with insulin resistance. This interaction controls a specific diabetic gene program in adipose tissue. By reducing Thrap3 levels, the ASO restores the expression of beneficial adipokines such as adiponectin and adipsin, effectively improving hyperglycemia and insulin sensitivity in preclinical models without the weight gain typically associated with PPARγ agonists. Developed in research settings (notably the Spiegelman lab in collaboration with Ionis Pharmaceuticals), it represents a targeted approach to treating type 2 diabetes and obesity by modulating the pathological transcriptional activity of PPARγ.

Other names
Thrap3 ASOThrap-3 ASOThrap 3 ASOthyroid hormone receptor-associated protein 3 antisense oligonucleotide
02

Targets

THRAP3 (Thyroid hormone receptor-associated protein 3)

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