Arrowhead Pharmaceuticals, Inc.

Arrowhead Madison, Inc.

Empirico Inc.

Hoffmann-la Roche Inc.

Mirus Bio Corporation

Roche Madison Inc.

F. Hoffmann-la Roche Ag

Arrowhead Research Corporation

David L Lewis

David B Rozema

Darren H Wakefield

Lauren Almeida

Peter Mohr

Philipp Hadwiger

Steven B Kanner

Torsten Hoffmann

Eric Argirios Kitas

Jon Asher Wolff

Christine I Wooddell

Ingo Roehl

Omri Gottesman

Andrei V Blokhin

Jeffrey Carlson

Jonathan D Benson

Hans Martin Mueller

Shannon Bruse

Brian Cajes

Kerstin Jahn-Hofmann

Guenther Ott

Tao Pei

Zhen Li

So Wong

Aaron Almeida

James E Hagstrom

Linda Valis

Sean D Monahan

Vladimir S Trubetskoy

Rui Zhu

Anthony Nicholas

Hans Herweijer

Weijun Cheng

Bruce D Given

Justin Woods

Paul Buske

Jason Klein

Wilma Thuer

Stacey Melquist

Aaron G Loomis

Julia Hegge

Lori Higgs

Ingo Röhl

Günther Ott

Kerstin Jahn-Hoffmann

David Jakubosky

John Vekich

Torsten Hoffman

Sarah Kleinstein

Erik Kitas

Jeffrey C Carlson

Linda Christine Valis

Eric A Kitas

Lauren J Almeida

Andrei Blokhin

Vladimir Trubetskoy

Weijun Chen

Anthony L Nicholas

Provided herein are oligonucleotide compositions that inhibit ANGPTL7 and reduce intraocular pressure when administered to an eye. The oligonucleotide compositions contain nucleoside modifications.

Treatment of angiopoietin like 7 (ANGPTL7) related diseases

Disclosed herein are compositions comprising an oligonucleotide that targets MST1. The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating conditions associated with MST1 variants that include providing an oligonucleotide that targets MST1 to a subject.

Treatment of MST1 related diseases and disorders

Disclosed herein are compositions comprising modified oligonucleotides. In some embodiments, the oligonucleotide includes a hydrophobic moiety. In some embodiments, the oligonucleotide includes a vinyl phosphonate. In some embodiments, the oligonucleotide includes modified oligonucleotide sugars such as 2' modified ribose molecules. In some embodiments, the oligonucleotide includes modified internucleoside linkages such as phosphorothioate linkages. In some embodiments, the oligonucleotide includes an siRNA. In some embodiments, the siRNA comprises a duplex region with overhangs on either end.

Modified oligonucleotides

The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference. The invention also relates to a pharmaceutical composition comprising the AAT RNAi trigger together with an excipient capable of improving delivery of the RNAi trigger to a liver cell in vivo. Delivery of the AAT RNAi trigger to liver cells in vivo provides for inhibition of AAT gene expression and treatment of alpha 1-antitrypsin deficiency and associated diseases.

Compositions and methods for inhibiting gene expression of alpha-1 antitrypsin

RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hif2&#x3b1; (EPAS1) gene are described. Pharmaceutical compositions comprising one or more Hif2&#x3b1; RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described Hif2&#x3b1; RNAi triggers to tumor cells in vivo provides for inhibition of Hif2&#x3b1; gene expression and treatment of cancer.

Compositions and methods for inhibiting gene expression of Hif2alpha

Described herein are compounds and pharmaceutical compositions containing such compounds which are prodrugs of ALOX-15 inhibitors. Also described herein are methods for using such compounds in the treatment of disease.

Prodrugs of ALOX-15 inhibitors and methods of using the same

Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) agents for inhibiting the expression of Hepatitis B virus gene are described. The HBV RNAi agents disclosed herein may be targeted to cells, such as hepatocytes, for example, by using conjugated targeting ligands. Pharmaceutical compositions comprising one or more HBV RNAi agents optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi agents to infected liver in vivo provides for inhibition of HBV gene expression and treatment of diseases and conditions associated with HBV infection.

RNAi agents for hepatitis B virus infection

Described are compositions and methods for inhibition of Hepatitis B virus gene expression. RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hepatitis B virus gene are described. Pharmaceutical compositions comprising one or more HBV RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described HBV RNAi triggers to infected liver in vivo provides for inhibition of HBV gene expression and treatment.

RNAi therapy for hepatitis B virus infection

RNAi agents for Hepatitis B virus infection

Provided are compositions comprising an oligonucleotide that targets Thymic stromal lymphopoietin (TSLP). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating an airway disorder by providing an oligonucleotide that targets TSLP to a subject in need thereof. In some embodiments, the oligonucleotide targeting is specific for a long isoform of TSLP (1fTSLP).

Treatment of thymic stromal lymphopoietin (TSLP) related diseases by inhibition of long-form TSLP transcripts

Compositions and methods for inhibiting gene expression of alpha-1 AntiTrypsin

Provided are compositions comprising an oligonucleotide that targets Angiopoietin-like 4 (ANGPTL4). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating a metabolic or cardiovascular disorder by providing an oligonucleotide that targets ANGPTL4 to a subject in need thereof.

Oligonucleotides for treatment of angiopoietin like 4 (ANGPTL4) related diseases

Growing community of inventors

Madison, WI, United States of America

David L Lewis