Arrowhead Pharmaceuticals, Inc.

Stacey Melquist

Tao Pei

Zhen Li

David B Rozema

Darren H Wakefield

David L Lewis

Steven B Kanner

Vladimir S Trubetskoy

Lauren Almeida

Aaron Almeida

Rui Zhu

Bruce D Given

Lauren J Almeida

Described herein are compositions and methods for inhibition of Asialoglycoprotein receptor 1 (ASGR1) gene expression. RNA interference (RNAi) agents, e.g., double stranded RNAi agents, and RNAi agent-targeting ligand conjugates for inhibiting the expression of an ASGR1 gene are described. Pharmaceutical compositions comprising one or more ASGR1 RNAi agents, optionally with one or more additional therapeutics, are also described. The ASGR1 RNAi agents can be used in methods of treatment of various diseases and conditions, such as cardiometabolic diseases related to elevated non-HDL cholesterol (non-HDL-C) levels, elevated LDL cholesterol (LDL-C) levels, elevated total cholesterol levels, and/or elevated triglyceride (TG) levels.

RNAi agents and compositions for inhibiting expression of Asialoglycoprotein receptor 1

RNA interference (RNAi) agents and RNAi agent conjugates for inhibiting the expression of the LPA (apo(a)) gene are described. Pharmaceutical compositions comprising one or more LPA RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described LPA RNAi agents to liver cells in vivo provides for inhibition of LPA gene expression and treatment of cardiovascular and cardiovascular-related diseases.

Compositions and methods for inhibiting gene expression of LPA

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Madison, WI, United States of America

Stacey Melquist