Patents Assigned to Arrowhead Pharmaceuticals, Inc.
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Patent number: 11920134Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a RRM2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a RRM2 gene using said pharmaceutical composition; and methods for inhibiting the expression of RRM2 in a cell.Type: GrantFiled: April 13, 2020Date of Patent: March 5, 2024Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: John Frederick Boylan, Birgit Bramlage, Markus Hossbach, John Reidhaar-Olson
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Patent number: 11912997Abstract: Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Superoxide Dismutase 1 (SOD1) gene. The SOD1 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an SOD1 gene. Pharmaceutical compositions that include one or more SOD1 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described SOD1 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of SOD1 gene expression and a reduction in SOD1 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including amyotrophic lateral sclerosis (ALS.).Type: GrantFiled: June 14, 2023Date of Patent: February 27, 2024Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Christine Esau, Ji Young Suk, Tao Pei, Anthony Nicholas, Xiaokai Li, Jeffrey Carlson
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Patent number: 11884920Abstract: 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.Type: GrantFiled: November 10, 2021Date of Patent: January 30, 2024Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen Li, Rui Zhu, Christine I. Wooddell, Tao Pei
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Patent number: 11845937Abstract: Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a double homeobox 4 (DUX4) gene. The DUX4 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a DUX4 gene. Pharmaceutical compositions that include one or more DUX4 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described DUX4 RNAi agents to skeletal muscle cells in vivo, provides for inhibition of DUX4 gene expression and a reduction in DUX4 levels, which can provide a therapeutic benefit to subjects, including human subjects, suffering from certain skeletal muscle-related diseases or disorders including Facioscapulohumeral Muscular Dystrophy (FSHD).Type: GrantFiled: March 9, 2023Date of Patent: December 19, 2023Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Zhi-Ming Ding, Jonathan Van Dyke, Xiaokai Li, Anthony Nicholas, Casi M. Schienebeck, Tao Pei, Zhao Xu, Teng Ai, Susan Phan, Susan Ramos-Hunter
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Patent number: 11840688Abstract: RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hif2? (EPAS1) gene are described. Pharmaceutical compositions comprising one or more Hif2? RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described Hif2? RNAi triggers to tumor cells in vivo provides for inhibition of Hif2? gene expression and treatment of cancer.Type: GrantFiled: February 19, 2021Date of Patent: December 12, 2023Assignee: Arrowhead Pharmaceuticals, inc.Inventors: So Wong, David L. Lewis, David B. Rozema, Darren H. Wakefield, Steven B. Kanner, Weijun Cheng, Lauren J. Almeida, Andrei V. Blokhin, Jeffrey C. Carlson, Anthony L. Nicholas, Aaron Almeida, Jonathan D. Benson, Justin Woods
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Publication number: 20230355651Abstract: 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.Type: ApplicationFiled: December 2, 2022Publication date: November 9, 2023Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen LI, Rui ZHU, Christine I. WOODDELL, Bruce D. GIVEN, Tao PEI, David L. LEWIS, Lauren J. ALMEIDA, David B. ROZEMA, Darren H. WAKEFIELD
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Publication number: 20230355654Abstract: 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.Type: ApplicationFiled: December 21, 2022Publication date: November 9, 2023Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Christine I. WOODDELL, David B. Rozema, David L. Lewis, Darren H. Wakefield, Lauren J. Almeida
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Publication number: 20230348905Abstract: Described are methods of reducing liver Z-AAT protein levels in a human subject with a PiZZ genotype of alpha-1 antitrypsin (AAT) by using pharmaceutical compositions that include AAT RNAi agents. The pharmaceutical compositions disclosed herein that include AAT RNAi agents, when administered to a human subject with a PiZZ mutation, lead to a reduction in liver Z-AAT protein levels, including both soluble and insoluble Z-AAT protein. Such reductions can lead to the treatment of liver diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, fulminant hepatic failure, and other liver-related diseases.Type: ApplicationFiled: September 14, 2021Publication date: November 2, 2023Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Dawn Christianson, James C. Hamilton, Bruce Given
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Patent number: 11629349Abstract: The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit xanthine dehydrogenase (XDH) gene expression. Also disclosed are pharmaceutical compositions that include XDH RNAi agents and methods of use thereof. The XDH RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Delivery of the XDH RNAi agents in vivo provides for inhibition of XDH gene expression. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by XDH gene expression, such as gout and hyperuricemia.Type: GrantFiled: May 19, 2022Date of Patent: April 18, 2023Assignee: ARROWHEAD PHARMACEUTICALS, INC.Inventors: Anthony Nicholas, Tao Pei, Zhao Xu, Daniel Braas, Zhi-Ming Ding
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Patent number: 11597701Abstract: Synthetic ?v?6 integrin ligands of Formula I having serum stability and affinity for integrin ?v?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 ?v?6, and thereby facilitating the uptake of the cargo molecules into these cells. Compositions that include ?v?6 integrin ligands and methods of use are also described.Type: GrantFiled: October 31, 2018Date of Patent: March 7, 2023Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen Li, Xiaokai Li, Erik W. Bush, Rui Zhu, Dongxu Shu, Jonathan Benson, Patrick Shao, Matthew Fowler-Watters
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Patent number: 11590156Abstract: 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.Type: GrantFiled: August 11, 2020Date of Patent: February 28, 2023Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen Li, Rui Zhu, Christine I. Wooddell, Bruce D. Given, Tao Pei, David L. Lewis, Lauren J. Almeida, David B. Rozema, Darren H. Wakefield
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Patent number: 11549112Abstract: The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit xanthine dehydrogenase (XDH) gene expression. Also disclosed are pharmaceutical compositions that include XDH RNAi agents and methods of use thereof. The XDH RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Delivery of the XDH RNAi agents in vivo provides for inhibition of XDH gene expression. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by XDH gene expression, such as gout and hyperuricemia.Type: GrantFiled: May 19, 2022Date of Patent: January 10, 2023Assignee: ARROWHEAD PHARMACEUTICALS, INC.Inventors: Anthony Nicholas, Tao Pei, Zhao Xu, Daniel Braas, Zhi-Ming Ding
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Publication number: 20230002768Abstract: 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.Type: ApplicationFiled: June 8, 2022Publication date: January 5, 2023Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Christine I. Wooddell, David L. Lewis, Darren H. Wakefield, Lauren Almeida, Steven B. Kanner
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Patent number: 11534453Abstract: 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.Type: GrantFiled: September 9, 2020Date of Patent: December 27, 2022Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Christine I. Wooddell, David B. Rozema, David L. Lewis, Darren H. Wakefield, Lauren J. Almeida
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Patent number: 11517584Abstract: 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.Type: GrantFiled: November 17, 2021Date of Patent: December 6, 2022Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen Li, Rui Zhu, Christine I. Wooddell, Bruce D. Given, Tao Pei, David L. Lewis, Lauren J. Almeida, David B. Rozema, Darren H. Wakefield
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Patent number: 11492624Abstract: 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.Type: GrantFiled: October 16, 2018Date of Patent: November 8, 2022Assignee: Arrowheads Pharmaceuticals, Inc.Inventors: Zhen Li, Tao Pei, Rui Zhu, Bruce D. Given, Stacey Melquist
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Publication number: 20220305046Abstract: Described are methods for treating alpha-1 antitrypsin deficiency (AATD) in a human patient in need of treatment, using pharmaceutical compositions that include AAT RNAi agents. The pharmaceutical compositions disclosed herein that include AAT RNAi agents, when administered to a human patient in need thereof, treat liver diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, increased risk of hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, fulminant hepatic failure, and other liver-related diseases.Type: ApplicationFiled: June 5, 2020Publication date: September 29, 2022Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Bruce Given, Dawn Christianson, James C. Hamilton, Zhen Li, Rui Zhu, Christine Wooddell, Tao Pei
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Patent number: 11384355Abstract: 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.Type: GrantFiled: May 31, 2019Date of Patent: July 12, 2022Assignee: Arrowhead Pharmaceuticals, Inc.Inventors: Christine I Wooddell, David L Lewis, Darren H Wakefield, Lauren Almeida, Steven B Kanner
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Publication number: 20220170015Abstract: 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.Type: ApplicationFiled: November 10, 2021Publication date: June 2, 2022Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen Li, Rui Zhu, Christine I. Wooddell, Tao Pei
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Publication number: 20220079973Abstract: 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.Type: ApplicationFiled: November 17, 2021Publication date: March 17, 2022Applicant: Arrowhead Pharmaceuticals, Inc.Inventors: Zhen LI, Rui ZHU, Christine I. WOODDELL, Bruce D. GIVEN, Tao PEI, David L. LEWIS, Lauren J. ALMEIDA, David B. ROZEMA, Darren H. WAKEFIELD