JP2024520522A - ムチン5AC(MUC5AC)の発現を阻害するためのRNAi剤、その組成物、及び使用方法 - Google Patents
ムチン5AC(MUC5AC)の発現を阻害するためのRNAi剤、その組成物、及び使用方法 Download PDFInfo
- Publication number
- JP2024520522A JP2024520522A JP2023573300A JP2023573300A JP2024520522A JP 2024520522 A JP2024520522 A JP 2024520522A JP 2023573300 A JP2023573300 A JP 2023573300A JP 2023573300 A JP2023573300 A JP 2023573300A JP 2024520522 A JP2024520522 A JP 2024520522A
- Authority
- JP
- Japan
- Prior art keywords
- rnai agent
- nucleotides
- muc5ac
- sense strand
- antisense strand
- 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.)
- Pending
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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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
- 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
- C12N2310/10—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
- 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
- C12N2310/317—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. ==--==--==--
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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
- 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
Landscapes
- 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)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163194370P | 2021-05-28 | 2021-05-28 | |
| US63/194,370 | 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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2024520522A true JP2024520522A (ja) | 2024-05-24 |
| JP2024520522A5 JP2024520522A5 (enExample) | 2025-06-02 |
Family
ID=84229166
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023573300A Pending JP2024520522A (ja) | 2021-05-28 | 2022-05-25 | ムチン5AC(MUC5AC)の発現を阻害するためのRNAi剤、その組成物、及び使用方法 |
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)
| 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 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
| DE60040274D1 (de) | 1999-03-10 | 2008-10-30 | Phogen Ltd | Verabreichung von nukleinsäuren und proteinen an zellen |
| AU2003295600A1 (en) * | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| US7820380B2 (en) | 2004-05-07 | 2010-10-26 | Celera Corporation | Genetic polymorphisms associated with liver fibrosis |
| CN101500548A (zh) | 2006-08-18 | 2009-08-05 | 弗·哈夫曼-拉罗切有限公司 | 用于体内递送多核苷酸的多缀合物 |
| WO2010067882A1 (ja) * | 2008-12-12 | 2010-06-17 | 株式会社クレハ | 癌及び喘息治療のための医薬組成物 |
| SI2539451T1 (sl) | 2010-02-24 | 2016-04-29 | Arrowhead Research Corporation | Sestavki za ciljano dostavo sirna |
| ES2719624T3 (es) | 2010-09-23 | 2019-07-11 | Prec Biologics Inc | Peptidomiméticos de cáncer de colon y de páncreas |
| 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 |
| WO2013032829A1 (en) | 2011-08-26 | 2013-03-07 | Arrowhead Research Corporation | Poly(vinyl ester) polymers for in vivo nucleic acid delivery |
| CA2842041A1 (en) | 2012-04-18 | 2013-10-24 | 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) |
| CA2948013A1 (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) |
| MX2018015109A (es) | 2016-06-06 | 2019-04-22 | Arrowhead Pharmaceuticals Inc | Nucleotidos modificados con 5'-ciclo-fosfonato. |
| JOP20190015A1 (ar) * | 2016-08-04 | 2019-02-04 | Arrowhead Pharmaceuticals Inc | عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب |
| SI3535397T1 (sl) | 2016-11-01 | 2022-04-29 | Arrowhead Pharmaceuticals, Inc. | Ligandi alfa-V beta-6 integrina in njihove uporabe |
| WO2019089765A1 (en) * | 2017-11-01 | 2019-05-09 | Arrowhead Pharmcaceuticals, Inc. | Integrin ligands and uses thereof |
| JP7420727B2 (ja) | 2018-02-17 | 2024-01-23 | アローヘッド ファーマシューティカルズ インコーポレイテッド | トリアルキン結合剤及び使用方法 |
| US11040056B2 (en) * | 2018-05-11 | 2021-06-22 | The University Of North Carolina At Chapel Hill | Benzimidazoles that enhance the activity of oligonucleotides |
-
2022
- 2022-05-25 MX MX2023014026A patent/MX2023014026A/es unknown
- 2022-05-25 US US17/824,841 patent/US12365897B2/en active Active
- 2022-05-25 EP EP22812096.0A patent/EP4352229A4/en active Pending
- 2022-05-25 WO PCT/US2022/030969 patent/WO2022251394A1/en not_active Ceased
- 2022-05-25 IL IL308883A patent/IL308883A/en unknown
- 2022-05-25 CA CA3218815A patent/CA3218815A1/en active Pending
- 2022-05-25 PE PE2023003126A patent/PE20250265A1/es unknown
- 2022-05-25 AU AU2022280863A patent/AU2022280863A1/en active Pending
- 2022-05-25 CN CN202280052248.5A patent/CN117858949A/zh active Pending
- 2022-05-25 BR BR112023024615A patent/BR112023024615A2/pt not_active Application Discontinuation
- 2022-05-25 JP JP2023573300A patent/JP2024520522A/ja active Pending
- 2022-05-25 KR KR1020237044563A patent/KR20240014067A/ko active Pending
- 2022-05-27 TW TW111119839A patent/TW202313068A/zh unknown
-
2023
- 2023-11-23 CO CONC2023/0016058A patent/CO2023016058A2/es unknown
- 2023-11-24 CL CL2023003505A patent/CL2023003505A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022251394A8 (en) | 2023-06-22 |
| CN117858949A (zh) | 2024-04-09 |
| CA3218815A1 (en) | 2022-12-01 |
| KR20240014067A (ko) | 2024-01-31 |
| US12365897B2 (en) | 2025-07-22 |
| TW202313068A (zh) | 2023-04-01 |
| CO2023016058A2 (es) | 2023-12-11 |
| WO2022251394A1 (en) | 2022-12-01 |
| BR112023024615A2 (pt) | 2024-02-20 |
| IL308883A (en) | 2024-01-01 |
| MX2023014026A (es) | 2023-12-12 |
| AU2022280863A9 (en) | 2023-12-07 |
| CL2023003505A1 (es) | 2024-10-04 |
| PE20250265A1 (es) | 2025-01-29 |
| US20230002767A1 (en) | 2023-01-05 |
| EP4352229A4 (en) | 2025-06-25 |
| EP4352229A1 (en) | 2024-04-17 |
| AU2022280863A1 (en) | 2023-11-30 |
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