JPWO2021020412A1 - - Google Patents
Info
- Publication number
- JPWO2021020412A1 JPWO2021020412A1 JP2021535370A JP2021535370A JPWO2021020412A1 JP WO2021020412 A1 JPWO2021020412 A1 JP WO2021020412A1 JP 2021535370 A JP2021535370 A JP 2021535370A JP 2021535370 A JP2021535370 A JP 2021535370A JP WO2021020412 A1 JPWO2021020412 A1 JP WO2021020412A1
- Authority
- JP
- Japan
- 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.)
- Granted
Links
Classifications
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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
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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
- C12N15/1137—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 enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y203/00—Acyltransferases (2.3)
- C12Y203/02—Aminoacyltransferases (2.3.2)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019140142 | 2019-07-30 | ||
| JP2019140142 | 2019-07-30 | ||
| PCT/JP2020/028960 WO2021020412A1 (ja) | 2019-07-30 | 2020-07-29 | Ｍｕｒｆ１を標的とする核酸医薬 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JPWO2021020412A1 true JPWO2021020412A1 (https=) | 2021-02-04 |
| JPWO2021020412A5 JPWO2021020412A5 (https=) | 2023-07-25 |
| JP7692829B2 JP7692829B2 (ja) | 2025-06-16 |
Family
ID=74229918
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021535370A Active JP7692829B2 (ja) | 2019-07-30 | 2020-07-29 | Ｍｕｒｆ１を標的とする核酸医薬 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US12584132B2 (https=) |
| EP (1) | EP4005602A4 (https=) |
| JP (1) | JP7692829B2 (https=) |
| WO (1) | WO2021020412A1 (https=) |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009518022A (ja) * | 2005-12-12 | 2009-05-07 | ピエール・ファーブル・デルモ−コスメティク | 抗ミオシンＶａｓｉＲＮＡおよび皮膚の脱色 |
| JP2015502365A (ja) * | 2011-12-12 | 2015-01-22 | オンコイミューニン，インコーポレイティド | オリゴヌクレオチドのイン−ビボ送達 |
| JP2018513668A (ja) * | 2014-12-26 | 2018-05-31 | 日東電工株式会社 | Ｐ２１遺伝子調節のためのｒｎａ剤 |
| WO2019113393A1 (en) * | 2017-12-06 | 2019-06-13 | Avidity Biosciences Llc | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2692265B1 (fr) | 1992-05-25 | 1996-11-08 | Centre Nat Rech Scient | Composes biologiquement actifs de type phosphotriesters. |
| FR2705099B1 (fr) | 1993-05-12 | 1995-08-04 | Centre Nat Rech Scient | Oligonucléotides phosphorothioates triesters et procédé de préparation. |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ／エス | オリゴヌクレオチド類似体 |
| KR20020013513A (ko) | 1999-03-18 | 2002-02-20 | 추후제출 | Ｘｙｌｏ-ｌｎａ 유사체 |
| US7098192B2 (en) | 1999-04-08 | 2006-08-29 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of STAT3 expression |
| EP1481983A4 (en) | 2002-02-13 | 2008-04-02 | Takeshi Imanishi | NUCLEOSIDE ANALOGUE AND OLIGONUCLEOTIDE DERIVATIVES CONTAINING A NUCLEOTIDE ANALOGON THEREOF |
| EP1572713A4 (en) | 2002-08-14 | 2006-05-24 | Pharmacia Corp | ANTISENSE MODULATION OF ACYL-COA SYNTHETASE 1 EXPRESSION |
| AU2003295600A1 (en) | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| WO2004063331A2 (en) | 2003-01-03 | 2004-07-29 | Gencia Corporation | SiRNA MEDIATED POST-TRANSRIPTIONAL GENE SILENCING OF GENES INVOLVED IN ALOPECIA |
| EP1661905B9 (en) | 2003-08-28 | 2012-12-19 | IMANISHI, Takeshi | Novel artificial nucleic acids of n-o bond crosslinkage type |
| JP2009536222A (ja) | 2006-05-05 | 2009-10-08 | アイシス ファーマシューティカルズ， インコーポレーテッド | Ｐｃｓｋ９の発現を調節するための化合物および方法 |
| CA2927045A1 (en) | 2006-10-03 | 2008-04-10 | Muthiah Manoharan | Lipid containing formulations |
| EP2215229B1 (en) | 2007-11-28 | 2015-07-08 | National University Corporation Tokyo Medical and Dental University | System for delivering nucleic acids for suppressing target gene expression by utilizing endogenous chylomicron |
| EP2238249A2 (en) | 2007-12-07 | 2010-10-13 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of mcl-1 |
| EP2264167B1 (en) | 2008-03-31 | 2016-10-12 | National Institute of Advanced Industrial Science and Technology | Double-stranded lipid-modified rna having high rna interference effect |
| CA2721183C (en) | 2008-04-11 | 2019-07-16 | Alnylam Pharmaceuticals, Inc. | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| CA2721333C (en) | 2008-04-15 | 2020-12-01 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for nucleic acid delivery |
| JP2010184879A (ja) | 2009-02-10 | 2010-08-26 | Univ Of Tokyo | 抗筋萎縮剤 |
| CA2778171C (en) | 2009-10-29 | 2017-07-25 | Osaka University | Bridged artificial nucleoside and nucleotide |
| EP2327783A1 (en) | 2009-11-27 | 2011-06-01 | Universitätsklinikum Freiburg | Pharmaceutical composition comprising miRNA-100 and its use in the modulation of blood vessel growth |
| AU2011235276B2 (en) | 2010-03-29 | 2015-09-03 | Alnylam Pharmaceuticals, Inc. | SiRNA therapy for transthyretin (TTR) related ocular amyloidosis |
| EP2576785A4 (en) | 2010-06-04 | 2014-12-24 | Univ Texas | METABOLIC CHANGE BY ME-378 |
| CA2812046A1 (en) | 2010-09-15 | 2012-03-22 | Alnylam Pharmaceuticals, Inc. | Modified irna agents |
| SI3366775T2 (sl) | 2011-11-18 | 2025-12-31 | Alnylam Pharmaceuticals, Inc. | Modificirana sredstva RNAI |
| EP3730618A1 (en) | 2011-11-18 | 2020-10-28 | Alnylam Pharmaceuticals, Inc. | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
| JP6112569B2 (ja) | 2011-12-16 | 2017-04-12 | 国立大学法人 東京医科歯科大学 | キメラ２重鎖核酸 |
| EA030211B1 (ru) | 2012-04-25 | 2018-07-31 | Регьюлэс Терапьютикс Инк. | Соединения на основе микрорнк и способы модулирования активности mir-21 |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| US20150337002A1 (en) | 2013-01-15 | 2015-11-26 | Shionogi & Co., Ltd. | Nucleoside and nucleotide having sulfonamide structure |
| WO2014126229A1 (ja) | 2013-02-18 | 2014-08-21 | 塩野義製薬株式会社 | 含窒素複素環構造を有するヌクレオシド及びヌクレオチド |
| JP6482475B2 (ja) | 2014-01-07 | 2019-03-13 | レナセラピューティクス株式会社 | アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド |
| US20170051286A1 (en) | 2014-05-01 | 2017-02-23 | Larry J. Smith | METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT |
| US10610564B2 (en) * | 2015-02-26 | 2020-04-07 | Stc.Unm | IRGM and precision autophagy controls for antimicrobial and inflammatory disease states and methods of detection of autophagy |
| EP3603648B1 (en) | 2017-03-29 | 2025-09-17 | Shionogi & Co., Ltd | Complex of nucleic acid medicine and multibranched lipid |
-
2020
- 2020-07-29 JP JP2021535370A patent/JP7692829B2/ja active Active
- 2020-07-29 WO PCT/JP2020/028960 patent/WO2021020412A1/ja not_active Ceased
- 2020-07-29 US US17/630,671 patent/US12584132B2/en active Active
- 2020-07-29 EP EP20847104.5A patent/EP4005602A4/en active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009518022A (ja) * | 2005-12-12 | 2009-05-07 | ピエール・ファーブル・デルモ−コスメティク | 抗ミオシンＶａｓｉＲＮＡおよび皮膚の脱色 |
| JP2015502365A (ja) * | 2011-12-12 | 2015-01-22 | オンコイミューニン，インコーポレイティド | オリゴヌクレオチドのイン−ビボ送達 |
| JP2018513668A (ja) * | 2014-12-26 | 2018-05-31 | 日東電工株式会社 | Ｐ２１遺伝子調節のためのｒｎａ剤 |
| WO2019113393A1 (en) * | 2017-12-06 | 2019-06-13 | Avidity Biosciences Llc | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
Non-Patent Citations (5)
| Title |
|---|
| ADAMS, V. ET AL.: "Myocardial expression of Murf-1 and MAFbx after induction of chronic heart failure: Effect on myocar", CARDIOVASCULAR RESEARCH, vol. 73, JPN6020036324, 2007, pages 120 - 129, ISSN: 0005412230 * |
| CASTILLERO, E. ET AL.: "Suppression of atrogin-1 and MuRF1 prevents dexamethasone-induced atrophy of cultured myotubes", METABOLISM CLINICAL AND EXPERIMENTAL, vol. 62, JPN6020036323, 2013, pages 1495 - 1502, ISSN: 0005412229 * |
| CHEN, B. ET AL.: "Activation of AMPK inhibits cardiomyocyte hypertrophy by modulating of the FOXO1/MuRF1 signaling pat", ACTA PHARMACOLOGICA SINICA, vol. 31, JPN6020036322, 2010, pages 798 - 804, ISSN: 0005412228 * |
| KIM, J. ET AL.: "p38 MAPK participates in muscle-specific RING Finger 1-mediated atrophy in cast-immobilized rat gast", THE KOREAN JOURNAL OF PHYSIOLOGY AND PHARMACOLOGY, vol. 13, JPN6020036321, 2009, pages 491 - 496, ISSN: 0005412227 * |
| POLGE, C. ET AL.: "Muscle actin is polyubiquitinylated in vitro and in vivo and targeted for breakdown by the E3 ligase", THE FASEB JOURNAL, vol. 25, JPN6020036320, 2011, pages 3790 - 3802, ISSN: 0005412226 * |
Also Published As
| Publication number | Publication date |
|---|---|
| US20220267778A1 (en) | 2022-08-25 |
| WO2021020412A1 (ja) | 2021-02-04 |
| JP7692829B2 (ja) | 2025-06-16 |
| US12584132B2 (en) | 2026-03-24 |
| EP4005602A4 (en) | 2024-06-12 |
| EP4005602A1 (en) | 2022-06-01 |
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