JP5998326B2 - 新規核酸プロドラッグおよびその使用方法 - Google Patents
新規核酸プロドラッグおよびその使用方法 Download PDFInfo
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- JP5998326B2 JP5998326B2 JP2012519668A JP2012519668A JP5998326B2 JP 5998326 B2 JP5998326 B2 JP 5998326B2 JP 2012519668 A JP2012519668 A JP 2012519668A JP 2012519668 A JP2012519668 A JP 2012519668A JP 5998326 B2 JP5998326 B2 JP 5998326B2
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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
-
- 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
-
- 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
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- 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
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/02—Nutrients, e.g. vitamins, minerals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/655—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms
- C07F9/65515—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms the oxygen atom being part of a five-membered ring
-
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
- A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
- A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
- A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
- A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Nutrition Science (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US22336909P | 2009-07-06 | 2009-07-06 | |
| US61/223,369 | 2009-07-06 | ||
| US24272209P | 2009-09-15 | 2009-09-15 | |
| US61/242,722 | 2009-09-15 | ||
| PCT/US2010/041068 WO2011005761A1 (en) | 2009-07-06 | 2010-07-06 | Novel nucleic acid prodrugs and methods use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015133754A Division JP2015205910A (ja) | 2009-07-06 | 2015-07-02 | 新規核酸プロドラッグおよびその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012532199A JP2012532199A (ja) | 2012-12-13 |
| JP2012532199A5 JP2012532199A5 (enExample) | 2013-08-29 |
| JP5998326B2 true JP5998326B2 (ja) | 2016-09-28 |
Family
ID=43429503
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012519668A Active JP5998326B2 (ja) | 2009-07-06 | 2010-07-06 | 新規核酸プロドラッグおよびその使用方法 |
| JP2015133754A Pending JP2015205910A (ja) | 2009-07-06 | 2015-07-02 | 新規核酸プロドラッグおよびその使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015133754A Pending JP2015205910A (ja) | 2009-07-06 | 2015-07-02 | 新規核酸プロドラッグおよびその使用方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US9744183B2 (enExample) |
| EP (1) | EP2451461A4 (enExample) |
| JP (2) | JP5998326B2 (enExample) |
| KR (1) | KR101885383B1 (enExample) |
| CN (1) | CN102596204B (enExample) |
| AU (1) | AU2010270714B2 (enExample) |
| BR (1) | BR112012000828A8 (enExample) |
| CA (1) | CA2767253A1 (enExample) |
| CL (1) | CL2012000021A1 (enExample) |
| IL (1) | IL217370A (enExample) |
| IN (1) | IN2012DN00720A (enExample) |
| MX (1) | MX342945B (enExample) |
| RU (1) | RU2612521C2 (enExample) |
| SG (2) | SG10201403841QA (enExample) |
| WO (1) | WO2011005761A1 (enExample) |
Families Citing this family (102)
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| KR101806314B1 (ko) * | 2008-04-03 | 2017-12-07 | 스프링 뱅크 파마슈티칼스, 인크. | 바이러스 감염증을 치료하기 위한 조성물 및 방법 |
| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CN107365339A (zh) * | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| DK2872147T3 (da) * | 2012-07-13 | 2023-02-20 | Wave Life Sciences Ltd | Fremgangsmåde til fremstilling af kirale oligonukleotider |
| JP2015529073A (ja) * | 2012-08-20 | 2015-10-05 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物可逆性基を有するポリヌクレオチド |
| CN105025884B (zh) * | 2013-03-01 | 2019-08-20 | 阿斯泰克斯制药公司 | 药物组合 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| WO2015108046A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| DK3234131T3 (da) | 2014-12-16 | 2020-06-29 | Roche Innovation Ct Copenhagen As | Fremgangsmåde til screening af kiral toksicitet |
| AU2015367378A1 (en) | 2014-12-17 | 2017-06-15 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| WO2016208773A1 (ja) * | 2015-06-26 | 2016-12-29 | 協和発酵キリン株式会社 | オリゴヌクレオチド誘導体 |
| CN117965536A (zh) | 2015-07-22 | 2024-05-03 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US20190070212A1 (en) * | 2016-03-11 | 2019-03-07 | Spring Bank Pharmaceuticals, Inc. | Compounds and compositions for the treatment of infections |
| MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| WO2017157672A1 (en) * | 2016-03-18 | 2017-09-21 | Roche Innovation Center Copenhagen A/S | Acyl-protected l-lna-guanosine monomers |
| US11060089B2 (en) | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
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| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| SG11201900154VA (en) * | 2016-07-15 | 2019-02-27 | Sperovie Biosciences Inc | Compounds, compositions, and methods for the treatment of disease |
| WO2018035380A1 (en) | 2016-08-17 | 2018-02-22 | Solstice Biologics, Ltd. | Polynucleotide constructs |
| MX2019002075A (es) | 2016-08-23 | 2019-07-01 | Dicerna Pharmaceuticals Inc | Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos. |
| WO2018041973A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| WO2018098264A1 (en) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| WO2018100558A2 (en) | 2016-12-01 | 2018-06-07 | Takeda Pharmaceutical Company Limited | Cyclic dinucleotide |
| ES3051834T3 (en) | 2016-12-19 | 2025-12-30 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| PT4122497T (pt) | 2016-12-19 | 2024-06-12 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
| NZ755416A (en) | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
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| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536058A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
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| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| CN107892710B (zh) * | 2017-10-10 | 2021-04-13 | 河北大学 | 一种易于回收循环使用的负载型铂配合物氧化剂及其制备方法和应用 |
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| TW202241454A (zh) * | 2021-02-01 | 2022-11-01 | 日商第一三共股份有限公司 | 抗體-免疫賦活化劑共軛物之新穎製造方法 |
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| EP4555085A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
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| JP2012532199A (ja) | 2012-12-13 |
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| RU2612521C2 (ru) | 2017-03-09 |
| IN2012DN00720A (enExample) | 2015-06-19 |
| KR20120046238A (ko) | 2012-05-09 |
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| EP2451461A4 (en) | 2013-05-29 |
| AU2010270714B2 (en) | 2015-08-13 |
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| AU2010270714A1 (en) | 2012-02-16 |
| SG10201403841QA (en) | 2014-09-26 |
| US9744183B2 (en) | 2017-08-29 |
| EP2451461A1 (en) | 2012-05-16 |
| WO2011005761A1 (en) | 2011-01-13 |
| SG177564A1 (en) | 2012-02-28 |
| MX342945B (es) | 2016-10-18 |
| US20160347784A1 (en) | 2016-12-01 |
| RU2012102480A (ru) | 2013-08-20 |
| CA2767253A1 (en) | 2011-01-13 |
| KR101885383B1 (ko) | 2018-08-03 |
| JP2015205910A (ja) | 2015-11-19 |
| BR112012000828A2 (pt) | 2016-02-23 |
| US10307434B2 (en) | 2019-06-04 |
| IL217370A (en) | 2017-09-28 |
| CN102596204B (zh) | 2016-11-23 |
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