JP6715325B2 - siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用 - Google Patents
siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用 Download PDFInfo
- Publication number
- JP6715325B2 JP6715325B2 JP2018518771A JP2018518771A JP6715325B2 JP 6715325 B2 JP6715325 B2 JP 6715325B2 JP 2018518771 A JP2018518771 A JP 2018518771A JP 2018518771 A JP2018518771 A JP 2018518771A JP 6715325 B2 JP6715325 B2 JP 6715325B2
- Authority
- JP
- Japan
- Prior art keywords
- sirna
- group
- pharmaceutical composition
- pharmaceutically acceptable
- amine
- 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.)
- Active
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
-
- 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
-
- 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
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
-
- 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
- C12N15/1131—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 viruses
-
- 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
- C12N2310/35—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)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Dispersion Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN201510364229 | 2015-06-26 | ||
| CN201510364229.8 | 2015-06-26 | ||
| PCT/CN2016/087034 WO2016206626A1 (zh) | 2015-06-26 | 2016-06-24 | 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018518201A JP2018518201A (ja) | 2018-07-12 |
| JP2018518201A5 JP2018518201A5 (enExample) | 2019-06-13 |
| JP6715325B2 true JP6715325B2 (ja) | 2020-07-01 |
Family
ID=57584730
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018518771A Active JP6715325B2 (ja) | 2015-06-26 | 2016-06-24 | siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20190062749A1 (enExample) |
| EP (1) | EP3315608B1 (enExample) |
| JP (1) | JP6715325B2 (enExample) |
| CN (1) | CN107849567B (enExample) |
| WO (1) | WO2016206626A1 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3073851A1 (en) * | 2017-08-31 | 2019-03-07 | Life Technologies Corporation | Cationic lipid compositions for tissue-specific delivery |
| US12083142B2 (en) * | 2017-12-01 | 2024-09-10 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| CN111050807B (zh) * | 2017-12-01 | 2024-05-28 | 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 |
| JP7261494B2 (ja) | 2017-12-01 | 2023-04-20 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用 |
| WO2019105435A1 (zh) | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 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 |
| CN110945131B (zh) * | 2017-12-01 | 2024-05-28 | 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 |
| KR102756073B1 (ko) * | 2017-12-01 | 2025-01-20 | 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 | 이중-가닥 올리고뉴클레오티드, 조성물 및 이중-가닥 올리고뉴클레오티드를 포함하는 접합체, 그의 제조 방법 및 그의 용도 |
| WO2019105419A1 (zh) * | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 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 |
| HRP20250408T1 (hr) | 2017-12-29 | 2025-06-06 | Suzhou Ribo Life Science Co., Ltd. | Kojugati i njihova priprema i uporaba |
| US11918600B2 (en) | 2018-08-21 | 2024-03-05 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof |
| CN111655297A (zh) * | 2018-09-30 | 2020-09-11 | 苏州瑞博生物技术有限公司 | 一种siRNA缀合物及其制备方法和用途 |
| CN112423795A (zh) * | 2018-12-28 | 2021-02-26 | 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 |
| CN111377985B (zh) * | 2018-12-29 | 2023-11-10 | 苏州瑞博生物技术股份有限公司 | 化合物和缀合物及其制备方法和用途 |
| CN113330117B (zh) * | 2019-01-18 | 2024-05-28 | 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 |
| AU2020280438B2 (en) * | 2019-05-22 | 2025-03-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| CN111979237A (zh) * | 2019-05-22 | 2020-11-24 | 苏州瑞博生物技术股份有限公司 | 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
| CN111973617A (zh) * | 2019-05-23 | 2020-11-24 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与缀合物及制备方法和用途 |
| CN111973618B (zh) * | 2019-05-23 | 2024-02-02 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与siRNA缀合物及制备方法和用途 |
| TW202122093A (zh) * | 2019-08-29 | 2021-06-16 | 大陸商蘇州瑞博生物技術股份有限公司 | 化合物、藥物綴合物、試劑盒及其用途 |
| WO2021110148A1 (zh) * | 2019-12-06 | 2021-06-10 | 南京明德新药研发有限公司 | siRNA缀合物、双链siRNA缀合物及其盐和应用 |
| CN111110866B (zh) * | 2019-12-30 | 2023-04-11 | 福州大学 | 还原性聚谷氨酸/聚乙烯亚胺/siRNA复合纳米粒及制备与应用 |
| US20240033278A9 (en) * | 2020-06-10 | 2024-02-01 | Medshine Discovery Inc. | Conjugate group and conjugate |
| CN113663089B (zh) * | 2021-06-25 | 2022-10-28 | 北京理工大学 | 可电离脂质纳米颗粒组合物、制备方法及应用 |
| WO2023116764A1 (zh) * | 2021-12-23 | 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其用途 |
| CN114634984A (zh) * | 2022-02-02 | 2022-06-17 | 复旦大学 | 一种脑胶质瘤生物标记物mlkl基因及其应用 |
| CN115487308B (zh) * | 2022-09-29 | 2024-09-27 | 北京大学 | 反义核酸糖基缀合物及其制备方法和在肝癌治疗中的应用 |
| KR20250120380A (ko) * | 2022-12-08 | 2025-08-08 | 1글로브 헬스 인스티튜트 엘엘씨 | 안티센스 RNA(asRNA) 기술 및 이의 용도 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2005222902B2 (en) * | 2004-03-12 | 2010-06-10 | Alnylam Pharmaceuticals, Inc. | iRNA agents targeting VEGF |
| KR101147147B1 (ko) * | 2004-04-01 | 2012-05-25 | 머크 샤프 앤드 돔 코포레이션 | Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드 |
| KR20100060018A (ko) * | 2005-03-09 | 2010-06-04 | 재단법인 목암생명공학연구소 | 작은 간섭 rna 및 이를 포함하는 b형 간염 바이러스 치료용 약학 조성물 |
| CN101426912A (zh) * | 2005-08-17 | 2009-05-06 | 瑟纳治疗公司 | 介导rna干扰的化学修饰短干扰核酸分子 |
| CN102140458B (zh) * | 2010-01-29 | 2013-05-22 | 苏州瑞博生物技术有限公司 | 小干扰核酸和药物组合物及其制药应用 |
| CN102140461B (zh) * | 2010-01-29 | 2012-12-05 | 苏州瑞博生物技术有限公司 | 小干扰核酸和药物组合物及其制药应用 |
| ES2702428T3 (es) * | 2010-11-15 | 2019-02-28 | Life Technologies Corp | Reactivos de transfección que contienen amina y métodos para prepararlos y usarlos |
| WO2013061295A1 (en) * | 2011-10-28 | 2013-05-02 | University Of The Witwatersrand, Johannesburg | Inhibition of viral gene expression |
| IN2014CN03463A (enExample) * | 2011-11-18 | 2015-10-09 | Alnylam Pharmaceuticals Inc | |
| US10086081B2 (en) * | 2012-08-06 | 2018-10-02 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| TW201437368A (zh) * | 2012-12-14 | 2014-10-01 | Dicerna Pharmaceuticals Inc | 藉由雙股rna之ckap5之專一性抑制之方法和組成物 |
| ME03043B (me) * | 2013-03-14 | 2018-10-20 | Alnylam Pharmaceuticals Inc | Irnk sastavi komponente komplementa c5 i metode za njihovu upotrebu |
-
2016
- 2016-06-24 US US15/738,006 patent/US20190062749A1/en not_active Abandoned
- 2016-06-24 EP EP16813742.0A patent/EP3315608B1/en active Active
- 2016-06-24 JP JP2018518771A patent/JP6715325B2/ja active Active
- 2016-06-24 WO PCT/CN2016/087034 patent/WO2016206626A1/zh not_active Ceased
- 2016-06-24 CN CN201680036513.5A patent/CN107849567B/zh active Active
Also Published As
| Publication number | Publication date |
|---|---|
| EP3315608A4 (en) | 2018-12-12 |
| US20190062749A1 (en) | 2019-02-28 |
| CN107849567B (zh) | 2024-07-23 |
| JP2018518201A (ja) | 2018-07-12 |
| EP3315608B1 (en) | 2020-09-16 |
| EP3315608A1 (en) | 2018-05-02 |
| CN107849567A (zh) | 2018-03-27 |
| WO2016206626A1 (zh) | 2016-12-29 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6715325B2 (ja) | siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用 | |
| Hu et al. | Therapeutic siRNA: state of the art | |
| JP7727308B2 (ja) | 二本鎖オリゴヌクレオチド、二本鎖オリゴヌクレオチドを含む組成物および複合体ならびに調製方法と使用 | |
| CN111107853B (zh) | 用于抑制载脂蛋白C-III (APOC3)的表达的RNAi试剂和组合物 | |
| TWI880645B (zh) | 抑制lpa之基因表現之組合物及方法 | |
| ES2988612T3 (es) | Agentes de ARNi para la infección por el virus de la hepatitis B | |
| JP2024112838A (ja) | 17β-HSD13型(HSD17B13)の発現を阻害するためのRNAi剤、その組成物、および使用方法 | |
| CN103747805B (zh) | 用于治疗、延缓和/或预防人类遗传性疾病如强直性肌营养不良症1型(dm1)的化合物 | |
| JP2024528634A (ja) | 二本鎖オリゴヌクレオチド、二本鎖オリゴヌクレオチドを含む組成物及び複合体並びに調製方法と使用 | |
| WO2017027350A2 (en) | Rnai therapy for hepatitis b virus infection | |
| KR20220011656A (ko) | 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도 | |
| CN115819484A (zh) | 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 | |
| KR101685304B1 (ko) | 간 조직 특이적 유전자 발현 억제인자 전달용 나노리포좀 | |
| CN119799705B (zh) | 靶向调控HBV基因表达的siRNA及其应用 | |
| EP4455284A1 (en) | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof | |
| JP2025514180A (ja) | 強化された肝臓及び腎臓特異的標的化のための遺伝子配列-炭水化物コンジュゲート | |
| CN119799706A (zh) | 靶向调控HBV基因表达的siRNA及其应用 | |
| WO2025067544A1 (zh) | 用于抑制乙型肝炎病毒的siRNA及其用途 | |
| TW202444904A (zh) | 用於抑制粒線體醯胺肟還原組分1(MARC1)之表現之RNAi藥劑、其醫藥組合物及使用方法 | |
| CN119709734A (zh) | 用于干扰乙型肝炎病毒的siRNA及其用途 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190426 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20190426 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20200310 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20200521 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20200602 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20200608 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 6715325 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |