Drug intelligence / Profile preview

androgen receptor siRNA

Development stage
Preclinical
Lead developer
Alnylam Pharmaceuticals
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

Androgen receptor siRNA is a therapeutic agent designed to silence the expression of the androgen receptor (AR) through the RNA interference (RNAi) pathway. By specifically targeting AR mRNA for degradation, it prevents the translation of the AR protein, thereby blocking androgen-driven biological processes. In research presented at ENDO 2026, a liver-targeted version of this siRNA, developed by Alnylam Pharmaceuticals, was evaluated for its potential to treat metabolic dysfunction in polycystic ovary syndrome (PCOS). The study demonstrated that hepatic AR knockdown could improve insulin sensitivity in skeletal muscle in a mouse model, suggesting that tissue-specific AR silencing may be a viable strategy for managing PCOS-associated insulin resistance.

Other names
AR siRNAhepatic androgen receptor siRNA
02

Targets

NR (Nuclear receptors (steroid))

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