Novartis Ag

Arrowhead Pharmaceuticals, Inc.

Junping Zhao

Jeremy Lee Baryza

Chandra Vargeese

Laura Ellen West

Rohan Eric John Beckwith

Thomas Zabawa

Ritesh Bhanudasji Tichkule

Gabriel Grant Gamber

Cameron Chuck-munn Lee

Tanzina Fazal

Shuangxi Wang

Crystal Byers

Jan Weiler

William Chutkow

Andrew Krueger

Andrew John Geall

Cameron Lee

Tanzina Labonte

Cameron Chuck-Munn Lee

Rohan Eric Beckwith

The present disclosure relates to compositions and methods for treating APOC3-related diseases such as: hypertriglyceridemia (e.g., Type V Hypertriglyceridemia), abnormal lipid metabolism, abnormal cholesterol metabolism, atherosclerosis, hyperlipidemia, diabetes, including Type 2 diabetes, obesity, cardiovascular disease, and coronary artery disease, among other disorders relating to abnormal metabolism or otherwise, using a therapeutically effective amount of a RNAi agent to APOC3.

Organic compositions to treat APOC3-related diseases

This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R1-R4, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

Lipids and lipid compositions for the delivery of active agents

This invention provides for a compound of formula (I): or a pharmaceutically acceptable salt thereof, wherein R-R, L and X are defined herein. The compounds of formula (I) and pharmaceutically acceptable salts thereof are cationic lipids useful in the delivery of biologically active agents to cells and tissues.

Disclosed are formulation and optimization protocols for delivery of therapeutically effective amounts of biologically active agents to liver, tumors, and/or other cells or tissues. Also provided are compositions and uses for cationic lipid compounds of formula (I). The invention also relates to compositions and uses for stealth lipids of formula (XI). Also provided are processes for making such compounds, compositions, and formulations, plus methods and uses of such compounds, compositions, and formulations to deliver biologically active agents to cells and/or tissues.

Lipids, lipid compositions, and methods of using them

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Cambridge, MA, United States of America

Junping Zhao