AU2011244335B2 - Organic compositions to treat Beta-ENaC-related diseases - Google Patents

Organic compositions to treat Beta-ENaC-related diseases Download PDF

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Publication number
AU2011244335B2
AU2011244335B2 AU2011244335A AU2011244335A AU2011244335B2 AU 2011244335 B2 AU2011244335 B2 AU 2011244335B2 AU 2011244335 A AU2011244335 A AU 2011244335A AU 2011244335 A AU2011244335 A AU 2011244335A AU 2011244335 B2 AU2011244335 B2 AU 2011244335B2
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AU
Australia
Prior art keywords
strand
antisense strand
rnai agent
enac
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
AU2011244335A
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English (en)
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AU2011244335A1 (en
Inventor
Antonin De Fougerolles
John L. Diener
Emma Hickman
Gregory Hinkle
Stuart Milstein
Anne-Marie Pulichino
Andrew Sprague
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Arrowhead Pharmaceuticals Inc
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Arrowhead Pharmaceuticals Inc
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of AU2011244335A1 publication Critical patent/AU2011244335A1/en
Assigned to ARROWHEAD RESEARCH CORPORATION reassignment ARROWHEAD RESEARCH CORPORATION Request for Assignment Assignors: NOVARTIS AG
Application granted granted Critical
Publication of AU2011244335B2 publication Critical patent/AU2011244335B2/en
Priority to AU2015221515A priority Critical patent/AU2015221515B2/en
Priority to AU2017201838A priority patent/AU2017201838A1/en
Assigned to Arrowhead Pharmaceuticals, Inc. reassignment Arrowhead Pharmaceuticals, Inc. Request to Amend Deed and Register Assignors: ARROWHEAD RESEARCH CORPORATION
Priority to AU2019203596A priority patent/AU2019203596A1/en
Priority to AU2021240326A priority patent/AU2021240326A1/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/02—Antidotes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P41/00—Drugs used in surgical methods, e.g. surgery adjuvants for preventing adhesion or for vitreum substitution
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/38—Drugs for disorders of the endocrine system of the suprarenal hormones
    • A61P5/40—Mineralocorticosteroids, e.g. aldosterone; Drugs increasing or potentiating the activity of mineralocorticosteroids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/38—Drugs for disorders of the endocrine system of the suprarenal hormones
    • A61P5/42—Drugs for disorders of the endocrine system of the suprarenal hormones for decreasing, blocking or antagonising the activity of mineralocorticosteroids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3513—Protein; Peptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/30—Chemical structure
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    • C12N2310/3517—Marker; Tag
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
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  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Endocrinology (AREA)
  • Cardiology (AREA)
  • Immunology (AREA)
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  • Child & Adolescent Psychology (AREA)
  • Pulmonology (AREA)
  • Urology & Nephrology (AREA)
AU2011244335A 2010-04-23 2011-04-20 Organic compositions to treat Beta-ENaC-related diseases Ceased AU2011244335B2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2015221515A AU2015221515B2 (en) 2010-04-23 2015-09-03 Organic compositions to treat Beta-ENaC-related diseases
AU2017201838A AU2017201838A1 (en) 2010-04-23 2017-03-17 Organic compositions to treat Beta-ENac-related diseases
AU2019203596A AU2019203596A1 (en) 2010-04-23 2019-05-22 Organic compositions to treat Beta-ENac-related diseases
AU2021240326A AU2021240326A1 (en) 2010-04-23 2021-10-04 Organic compositions to treat Beta-ENac-related diseases

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US32737910P 2010-04-23 2010-04-23
US61/327,379 2010-04-23
US33339810P 2010-05-11 2010-05-11
US61/333,398 2010-05-11
PCT/EP2011/056299 WO2011131707A1 (en) 2010-04-23 2011-04-20 ORGANIC COMPOSITIONS TO TREAT BETA-ENaC-RELATED DISEASES

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2015221515A Division AU2015221515B2 (en) 2010-04-23 2015-09-03 Organic compositions to treat Beta-ENaC-related diseases

Publications (2)

Publication Number Publication Date
AU2011244335A1 AU2011244335A1 (en) 2012-11-22
AU2011244335B2 true AU2011244335B2 (en) 2015-06-04

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AU2011244335A Ceased AU2011244335B2 (en) 2010-04-23 2011-04-20 Organic compositions to treat Beta-ENaC-related diseases

Country Status (15)

Country Link
US (10) US8344127B2 (cg-RX-API-DMAC10.html)
EP (3) EP2561077B1 (cg-RX-API-DMAC10.html)
JP (6) JP5857378B2 (cg-RX-API-DMAC10.html)
KR (4) KR20220008383A (cg-RX-API-DMAC10.html)
CN (2) CN102985544A (cg-RX-API-DMAC10.html)
AR (1) AR081082A1 (cg-RX-API-DMAC10.html)
AU (1) AU2011244335B2 (cg-RX-API-DMAC10.html)
BR (1) BR112012027080A2 (cg-RX-API-DMAC10.html)
CA (2) CA3098080A1 (cg-RX-API-DMAC10.html)
DK (1) DK2561077T3 (cg-RX-API-DMAC10.html)
EA (1) EA034363B1 (cg-RX-API-DMAC10.html)
ES (1) ES2574204T3 (cg-RX-API-DMAC10.html)
MX (2) MX383583B (cg-RX-API-DMAC10.html)
TW (1) TWI434692B (cg-RX-API-DMAC10.html)
WO (1) WO2011131707A1 (cg-RX-API-DMAC10.html)

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Publication number Priority date Publication date Assignee Title
CA3098080A1 (en) 2010-04-23 2011-10-27 Arrowhead Research Corporation Organic compositions to treat beta-enac-related diseases
CN102397279B (zh) * 2011-11-18 2012-10-31 新乡医学院 无羁萜-3β-醇在制备抗血管性痴呆的药物中的应用
KR20150037968A (ko) * 2012-07-12 2015-04-08 프로큐알 테라퓨틱스 엔.브이. 살아있는 세포 내에 존재하는 목적 rna 분자의 서열을 변화시키기 위한 올리고뉴클레오타이드
CN106232073B (zh) 2014-02-28 2020-11-03 阿坦斯医疗保健产品公司 具有多层折叠吸收芯的吸收性用品
CN109475552B (zh) * 2016-08-09 2022-04-05 上海药苑生物科技有限公司 用于食欲控制和体重管理的方法和组合物
MX2019007004A (es) 2016-12-14 2019-08-22 Attends Healthcare Products Inc Laminados absorbentes, nucleos absorbentes y articulos desechables que utilizan los laminados y metodos relacionados.
EP3801424B1 (en) 2018-05-28 2025-01-22 Attends Healthcare Products, Inc. Dryness layer laminate for absorbent articles
CN109364248B (zh) * 2018-10-16 2021-05-18 哈尔滨医科大学 ENaC及其抑制剂在预防、缓解和/或治疗动脉粥样硬化中的应用
TW202130809A (zh) * 2019-10-29 2021-08-16 美商愛羅海德製藥公司 用於抑制β-ENaC表現之RNAi藥劑、其組合物及使用方法
US20230136960A1 (en) * 2020-03-19 2023-05-04 Nature's Toolbox, Inc. Novel MRNA-Based COVID-19 Multi-Valent Vaccine and Methods of Scaled Production of the Same
CN114149492A (zh) * 2020-08-21 2022-03-08 中国人民解放军军事科学院军事医学研究院 一种用于预防甲型流感病毒感染的HA-mRNA疫苗

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EP1752536A1 (en) * 2004-05-11 2007-02-14 RNAi Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same

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