CR20190572A - AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO - Google Patents
AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USOInfo
- Publication number
- CR20190572A CR20190572A CR20190572A CR20190572A CR20190572A CR 20190572 A CR20190572 A CR 20190572A CR 20190572 A CR20190572 A CR 20190572A CR 20190572 A CR20190572 A CR 20190572A CR 20190572 A CR20190572 A CR 20190572A
- Authority
- CR
- Costa Rica
- Prior art keywords
- enac
- alpha
- rnai agents
- methods
- inhibiting expression
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title abstract 7
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 7
- 230000014509 gene expression Effects 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 230000002401 inhibitory effect Effects 0.000 title 1
- 101710194189 Amiloride-sensitive sodium channel subunit alpha Proteins 0.000 abstract 6
- 102100037242 Amiloride-sensitive sodium channel subunit alpha Human genes 0.000 abstract 6
- 239000003795 chemical substances by application Substances 0.000 abstract 6
- 210000002919 epithelial cell Anatomy 0.000 abstract 2
- 230000005764 inhibitory process Effects 0.000 abstract 2
- 101000740448 Homo sapiens Amiloride-sensitive sodium channel subunit alpha Proteins 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000002685 pulmonary effect Effects 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
Classifications
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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
-
- 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
- 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
-
- 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
-
- 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
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/333—Modified A
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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
- C12N2310/34—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
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Endocrinology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762529132P | 2017-07-06 | 2017-07-06 | |
| US201862631683P | 2018-02-17 | 2018-02-17 | |
| US201862679549P | 2018-06-01 | 2018-06-01 | |
| PCT/US2018/040874 WO2019010274A1 (en) | 2017-07-06 | 2018-07-05 | RNAI AGENTS OF INHIBITION OF ALPHA-ENAC GENE EXPRESSION AND METHODS OF USE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CR20190572A true CR20190572A (es) | 2020-05-23 |
Family
ID=64904123
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CR20190572A CR20190572A (es) | 2017-07-06 | 2018-07-05 | AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO |
Country Status (22)
| Country | Link |
|---|---|
| US (3) | US10590416B2 (enExample) |
| EP (1) | EP3649240A4 (enExample) |
| JP (3) | JP2020526192A (enExample) |
| KR (1) | KR20200024793A (enExample) |
| CN (2) | CN119662650A (enExample) |
| AU (1) | AU2018297262A1 (enExample) |
| BR (1) | BR112019023650A2 (enExample) |
| CA (1) | CA3061752A1 (enExample) |
| CL (1) | CL2020000019A1 (enExample) |
| CO (1) | CO2019014671A2 (enExample) |
| CR (1) | CR20190572A (enExample) |
| EC (1) | ECSP20000655A (enExample) |
| IL (1) | IL271843A (enExample) |
| JO (1) | JOP20190263A1 (enExample) |
| MX (1) | MX2019014800A (enExample) |
| PE (1) | PE20200746A1 (enExample) |
| PH (1) | PH12019502660A1 (enExample) |
| TN (1) | TN2019000308A1 (enExample) |
| TW (1) | TW201919654A (enExample) |
| UY (1) | UY37800A (enExample) |
| WO (1) | WO2019010274A1 (enExample) |
| ZA (1) | ZA202100643B (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TN2019000308A1 (en) * | 2017-07-06 | 2021-05-07 | Arrowhead Pharmaceuticals Inc | RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE |
| CN120247964A (zh) * | 2018-02-17 | 2025-07-04 | 箭头药业股份有限公司 | 三炔连接剂和使用方法 |
| US12065458B2 (en) | 2018-02-17 | 2024-08-20 | Arrowhead Pharmaceuticals, Inc. | Trialkyne linking agents and methods of use |
| 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 |
| AU2021342155A1 (en) * | 2020-09-11 | 2023-04-13 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use |
| WO2023116607A1 (zh) | 2021-12-21 | 2023-06-29 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
| KR20250043472A (ko) * | 2022-07-27 | 2025-03-28 | 이-테라퓨틱스 피엘씨 | 핵산 화합물 |
| WO2024023256A1 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
| WO2024188174A1 (zh) | 2023-03-10 | 2024-09-19 | 苏州瑞博生物技术股份有限公司 | 一种药物组合物及其用途 |
| MX2025014897A (es) * | 2023-06-13 | 2026-02-03 | Tuojie Biotech Shanghai Co Ltd | Arnip dirigido a mmp7, conjugado de arnip y su uso medico |
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| TN2019000308A1 (en) * | 2017-07-06 | 2021-05-07 | Arrowhead Pharmaceuticals Inc | RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE |
| SG11202002967SA (en) * | 2017-11-01 | 2020-05-28 | Arrowhead Pharmaceuticals Inc | Integrin ligands and uses thereof |
| CN120247964A (zh) * | 2018-02-17 | 2025-07-04 | 箭头药业股份有限公司 | 三炔连接剂和使用方法 |
-
2018
- 2018-07-05 TN TNP/2019/000308A patent/TN2019000308A1/en unknown
- 2018-07-05 KR KR1020197038728A patent/KR20200024793A/ko not_active Ceased
- 2018-07-05 CN CN202411877733.3A patent/CN119662650A/zh active Pending
- 2018-07-05 PE PE2019002542A patent/PE20200746A1/es unknown
- 2018-07-05 JP JP2019572571A patent/JP2020526192A/ja not_active Withdrawn
- 2018-07-05 MX MX2019014800A patent/MX2019014800A/es unknown
- 2018-07-05 CR CR20190572A patent/CR20190572A/es unknown
- 2018-07-05 EP EP18828259.4A patent/EP3649240A4/en active Pending
- 2018-07-05 BR BR112019023650-3A patent/BR112019023650A2/pt not_active Application Discontinuation
- 2018-07-05 US US16/028,006 patent/US10590416B2/en active Active
- 2018-07-05 CA CA3061752A patent/CA3061752A1/en active Pending
- 2018-07-05 CN CN201880045293.1A patent/CN110832077B/zh active Active
- 2018-07-05 AU AU2018297262A patent/AU2018297262A1/en not_active Abandoned
- 2018-07-05 WO PCT/US2018/040874 patent/WO2019010274A1/en not_active Ceased
- 2018-07-06 UY UY0001037800A patent/UY37800A/es not_active Application Discontinuation
- 2018-07-06 TW TW107123550A patent/TW201919654A/zh unknown
-
2019
- 2019-01-06 JO JOP/2019/0263A patent/JOP20190263A1/ar unknown
- 2019-11-25 PH PH12019502660A patent/PH12019502660A1/en unknown
- 2019-12-24 CO CONC2019/0014671A patent/CO2019014671A2/es unknown
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2020
- 2020-01-03 CL CL2020000019A patent/CL2020000019A1/es unknown
- 2020-01-06 IL IL271843A patent/IL271843A/en unknown
- 2020-01-06 EC ECSENADI2020655A patent/ECSP20000655A/es unknown
- 2020-01-31 US US16/778,582 patent/US11214802B2/en active Active
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2021
- 2021-01-29 ZA ZA2021/00643A patent/ZA202100643B/en unknown
- 2021-11-29 US US17/536,754 patent/US20220090077A1/en not_active Abandoned
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2023
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-
2025
- 2025-10-02 JP JP2025166543A patent/JP2025185070A/ja active Pending
Also Published As
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|---|---|
| AU2018297262A1 (en) | 2020-02-27 |
| JP2025185070A (ja) | 2025-12-18 |
| JP2023158192A (ja) | 2023-10-26 |
| US20190010494A1 (en) | 2019-01-10 |
| EP3649240A1 (en) | 2020-05-13 |
| EP3649240A4 (en) | 2021-07-07 |
| CN119662650A (zh) | 2025-03-21 |
| PH12019502660A1 (en) | 2020-06-08 |
| US20220090077A1 (en) | 2022-03-24 |
| KR20200024793A (ko) | 2020-03-09 |
| ZA202100643B (en) | 2023-03-29 |
| US10590416B2 (en) | 2020-03-17 |
| WO2019010274A1 (en) | 2019-01-10 |
| MX2019014800A (es) | 2020-02-10 |
| US11214802B2 (en) | 2022-01-04 |
| IL271843A (en) | 2020-02-27 |
| CL2020000019A1 (es) | 2020-06-12 |
| CO2019014671A2 (es) | 2020-01-17 |
| CA3061752A1 (en) | 2019-01-10 |
| TN2019000308A1 (en) | 2021-05-07 |
| JOP20190263A1 (ar) | 2019-11-12 |
| JP2020526192A (ja) | 2020-08-31 |
| ECSP20000655A (es) | 2020-05-29 |
| TW201919654A (zh) | 2019-06-01 |
| US20200299691A1 (en) | 2020-09-24 |
| PE20200746A1 (es) | 2020-07-24 |
| CN110832077B (zh) | 2025-01-03 |
| CN110832077A (zh) | 2020-02-21 |
| BR112019023650A2 (pt) | 2020-06-02 |
| UY37800A (es) | 2019-01-02 |
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