IL308883A - RNAi AGENTS FOR INHIBITING EXPRESSION OF MUCIN 5AC (MUC5AC), COMPOSITIONS THEREOF, AND METHODS OF USE - Google Patents

RNAi AGENTS FOR INHIBITING EXPRESSION OF MUCIN 5AC (MUC5AC), COMPOSITIONS THEREOF, AND METHODS OF USE

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Publication number
IL308883A
IL308883A IL308883A IL30888323A IL308883A IL 308883 A IL308883 A IL 308883A IL 308883 A IL308883 A IL 308883A IL 30888323 A IL30888323 A IL 30888323A IL 308883 A IL308883 A IL 308883A
Authority
IL
Israel
Prior art keywords
rnai agent
nucleotides
muc5ac
seq
sense strand
Prior art date
Application number
IL308883A
Other languages
English (en)
Hebrew (he)
Inventor
Erik W Bush
Anthony Nicholas
Casi M Schienebeck
Original Assignee
Arrowhead Pharmaceuticals Inc
Erik W Bush
Anthony Nicholas
Casi M Schienebeck
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Arrowhead Pharmaceuticals Inc, Erik W Bush, Anthony Nicholas, Casi M Schienebeck filed Critical Arrowhead Pharmaceuticals Inc
Publication of IL308883A publication Critical patent/IL308883A/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • 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
    • A61P11/06—Antiasthmatics
    • 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
    • A61P11/12—Mucolytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/30—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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pulmonology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
IL308883A 2021-05-28 2022-05-25 RNAi AGENTS FOR INHIBITING EXPRESSION OF MUCIN 5AC (MUC5AC), COMPOSITIONS THEREOF, AND METHODS OF USE IL308883A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163194370P 2021-05-28 2021-05-28
PCT/US2022/030969 WO2022251394A1 (en) 2021-05-28 2022-05-25 Rnai agents for inhibiting expression of mucin 5ac (muc5ac), compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
IL308883A true IL308883A (en) 2024-01-01

Family

ID=84229166

Family Applications (1)

Application Number Title Priority Date Filing Date
IL308883A IL308883A (en) 2021-05-28 2022-05-25 RNAi AGENTS FOR INHIBITING EXPRESSION OF MUCIN 5AC (MUC5AC), COMPOSITIONS THEREOF, AND METHODS OF USE

Country Status (15)

Country Link
US (1) US12365897B2 (enExample)
EP (1) EP4352229A4 (enExample)
JP (1) JP2024520522A (enExample)
KR (1) KR20240014067A (enExample)
CN (1) CN117858949A (enExample)
AU (1) AU2022280863A1 (enExample)
BR (1) BR112023024615A2 (enExample)
CA (1) CA3218815A1 (enExample)
CL (1) CL2023003505A1 (enExample)
CO (1) CO2023016058A2 (enExample)
IL (1) IL308883A (enExample)
MX (1) MX2023014026A (enExample)
PE (1) PE20250265A1 (enExample)
TW (1) TW202313068A (enExample)
WO (1) WO2022251394A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024158905A1 (en) * 2023-01-24 2024-08-02 Ohio State Innovation Foundation Muc5ac in pancreatic ductal adenocarcinoma
WO2025128462A1 (en) * 2023-12-12 2025-06-19 Eli Lilly And Company Novel therapeutic delivery moieties and uses thereof

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US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
JP2002537828A (ja) 1999-03-10 2002-11-12 フォゲン リミティド 細胞への物質のデリバリー
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
EP2412826B1 (en) * 2004-05-07 2014-01-08 Celera Corporation Genetic polymorphism associated with liver fibrosis methods of detection and uses thereof
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
EP2377934A4 (en) * 2008-12-12 2012-09-26 Kureha Corp PHARMACEUTICAL COMPOSITION FOR THE TREATMENT OF TUMOR AND ASTHMA
EP3023495B1 (en) 2010-02-24 2019-05-08 Arrowhead Pharmaceuticals, Inc. Compositions for targeted delivery of sirna
WO2012040617A2 (en) * 2010-09-23 2012-03-29 Neogenix Oncology, Inc. Colon and pancreas cancer peptidomimetics
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
KR20140051357A (ko) 2011-08-26 2014-04-30 애로우헤드 리서치 코오포레이션 Ｉｎ Ｖｉｖｏ 핵산 전달용 폴리(비닐 에스테르) 고분자
AU2012377385A1 (en) 2012-04-18 2014-01-23 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
US9452228B2 (en) * 2013-04-01 2016-09-27 Immunomedics, Inc. Antibodies reactive with an epitope located in the N-terminal region of MUC5AC comprising cysteine-rich subdomain 2 (Cys2)
WO2016003869A1 (en) * 2014-06-30 2016-01-07 Immunomedics, Inc. Antibodies reactive with an epitope located in the n-terminal region of muc5ac comprising cysteine-rich subdomain 2 (cys2)
CA3023764A1 (en) 2016-06-06 2017-12-14 Arrowhead Pharmaceuticals, Inc. 5'-cyclo-phosphonate modified nucleotides
JOP20190015A1 (ar) * 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
CN109952376B (zh) 2016-11-01 2023-09-05 箭头药业股份有限公司 αvβ6整联蛋白配体及其应用
KR102762143B1 (ko) 2017-11-01 2025-02-07 애로우헤드 파마슈티컬스 인코포레이티드 인테그린 리간드 및 그의 용도
CA3089276A1 (en) 2018-02-17 2019-08-22 Arrowhead Pharmaceuticals, Inc. Trialkyne linking agents and methods of use
US11040056B2 (en) * 2018-05-11 2021-06-22 The University Of North Carolina At Chapel Hill Benzimidazoles that enhance the activity of oligonucleotides

Also Published As

Publication number Publication date
MX2023014026A (es) 2023-12-12
AU2022280863A1 (en) 2023-11-30
US20230002767A1 (en) 2023-01-05
EP4352229A1 (en) 2024-04-17
JP2024520522A (ja) 2024-05-24
PE20250265A1 (es) 2025-01-29
EP4352229A4 (en) 2025-06-25
US12365897B2 (en) 2025-07-22
CA3218815A1 (en) 2022-12-01
KR20240014067A (ko) 2024-01-31
CL2023003505A1 (es) 2024-10-04
AU2022280863A9 (en) 2023-12-07
WO2022251394A8 (en) 2023-06-22
CO2023016058A2 (es) 2023-12-11
WO2022251394A1 (en) 2022-12-01
BR112023024615A2 (pt) 2024-02-20
CN117858949A (zh) 2024-04-09
TW202313068A (zh) 2023-04-01

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