Arrowhead Pharmaceuticals, Inc.

Mirus Bio Corporation

Roche Madison Inc.

Christine I Wooddell

Darren H Wakefield

David L Lewis

Lauren Almeida

David B Rozema

Tao Pei

Zhen Li

Rui Zhu

Steven B Kanner

Bruce D Given

Hans Herweijer

Jon Asher Wolff

Lauren J Almeida

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

RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.

Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use

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

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

RNAi Therapy for Hepatitis B Virus Infection

The present invention relates to compositions and methods for increasing long term expression in vitro and in vivo, comprising sequences and gene expression cassettes that increase expression of genes to which they are operably linked.

Methods for expression of transgenes

We disclose compositions and processes for enhancing transposon mediated integration of a nucleic acid molecule into another target nucleic acid molecule. Integration by an integrator complex is enhanced by cationic reagents.

Enhancing intermolecular integration of nucleic acids using integrator complexes

Growing community of inventors

Madison, WI, United States of America

Christine I Wooddell