Arrowhead Pharmaceuticals, Inc.

Erik W Bush

Zhen Li

Anthony Nicholas

Tao Pei

Rui Zhu

Matthew Fowler-Watters

Jeffrey Carlson

Agnieszka Glebocka

Aaron Almeida

Jonathan D Benson

Xiaokai Li

Casi M Schienebeck

Patrick Shao

Dongxu Shu

Angieszka Glebocka

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Mucin 5AC (MUC5AC) gene. The MUC5AC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an MUC5AC gene. Pharmaceutical compositions that include one or more MUC5AC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MUC5AC RNAi agents to pulmonary epithelial cells, in vivo, provides for inhibition of MUC5AC gene expression and a reduction in MUC5AC production, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including mucoobstructive lung disease such as severe asthma and various cancers.

RNAi agents for inhibiting expression of mucin 5AC (MUC5AC), compositions thereof, and methods of use

Integrin ligands having serum stability and affinity for αvβ6 integrins are described. Compositions comprising αvβ6 integrin ligands having serum stability and having affinity for αvβ6 integrins and methods of using them are also described.

Alpha-v beta-6 integrin ligands and uses thereof

Synthetic &#x3b1;v&#x3b2;6 integrin ligands of Formula I having serum stability and affinity for integrin &#x3b1;v&#x3b2;6, which is a receptor expressed in a variety of cell types, are described. The described ligands are useful for delivering cargo molecules, such as RNAi agents or other oligonucleotide-based compounds, to cells that express integrin &#x3b1;v&#x3b2;6, and thereby facilitating the uptake of the cargo molecules into these cells. Compositions that include &#x3b1;v&#x3b2;6 integrin ligands and methods of use are also described. 

Integrin ligands and uses thereof

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.

RNAi agents for inhibiting expression of alpha-ENaC and methods of use

Integrin ligands having serum stability and affinity for &#x3b1;v&#x3b2;6 integrins are described. Compositions comprising &#x3b1;v&#x3b2;6 integrin ligands having serum stability and having affinity for &#x3b1;v&#x3b2;6 integrins and methods of using them are also described.

Growing community of inventors

Verona, WI, United States of America

Erik W Bush