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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.
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