ARNI agents to inhibit androgen receptor (AR) expression, their compositions and methods of use

Abstract

RNAi agents, compositions including RNAi agents, and methods for inhibiting an androgen receptor (AR) gene are described. The AR RNAi agents and RNAi agent conjugates disclosed herein inhibit AR gene expression. Pharmaceutical compositions including one or more AR RNAi agents, optionally with one or more additional drugs, are also described. In vivo administration of the described AR RNAi agents to central nervous system (CNS) and / or skeletal muscle tissue enables inhibition of AR gene expression and a reduction in AR activity, which may provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases, including spinal and bulbar muscular atrophy (SBMA).