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)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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 (Direct) | 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 (Direct) |
| EP (1) | EP2451461A4 (Direct) |
| JP (2) | JP5998326B2 (Direct) |
| KR (1) | KR101885383B1 (Direct) |
| CN (1) | CN102596204B (Direct) |
| AU (1) | AU2010270714B2 (Direct) |
| BR (1) | BR112012000828A8 (Direct) |
| CA (1) | CA2767253A1 (Direct) |
| CL (1) | CL2012000021A1 (Direct) |
| IL (1) | IL217370A (Direct) |
| IN (1) | IN2012DN00720A (Direct) |
| MX (1) | MX342945B (Direct) |
| RU (1) | RU2612521C2 (Direct) |
| SG (2) | SG177564A1 (Direct) |
| WO (1) | WO2011005761A1 (Direct) |
Families Citing this family (94)
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|---|---|---|---|---|
| CN103816174B (zh) * | 2008-04-03 | 2017-12-05 | 春堤制药公司 | 用于治疗病毒感染的化合物和方法 |
| JP5645840B2 (ja) | 2008-12-02 | 2014-12-24 | 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ | リン原子修飾核酸の合成方法 |
| IN2012DN00720A (Direct) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| RU2014105311A (ru) * | 2011-07-19 | 2015-08-27 | Уэйв Лайф Сайенсес Пте. Лтд. | Способы синтеза функционализованных нуклеиновых кислот |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| SG11201500232UA (en) * | 2012-07-13 | 2015-04-29 | Wave Life Sciences Pte Ltd | Chiral control |
| US9950001B2 (en) | 2012-08-20 | 2018-04-24 | The Regents Of The University Of California | Polynucleotides having bioreversible groups |
| SG11201506727RA (en) * | 2013-03-01 | 2015-09-29 | Astex Pharmaceuticals Inc | Drug combinations |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| 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 |
| US10149905B2 (en) | 2014-01-15 | 2018-12-11 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| 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 |
| CA2968336C (en) | 2014-12-17 | 2021-11-23 | Proqr Therapeutics Ii B.V. | Construct for site directed editing of an adenosine nucleotide in target rna |
| 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 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MX2018003992A (es) | 2015-10-09 | 2018-08-01 | Wave Life Sciences Ltd | Composiciones oligonucleotidicas y sus metodos. |
| 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 |
| CN108779139A (zh) * | 2016-03-18 | 2018-11-09 | 罗氏创新中心哥本哈根有限公司 | 酰基保护的l-lna-鸟嘌呤核苷单体 |
| JP7033547B2 (ja) | 2016-04-18 | 2022-03-10 | サレプタ セラピューティクス， インコーポレイテッド | 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法 |
| EA201892467A1 (ru) | 2016-04-29 | 2019-05-31 | Сарепта Терапьютикс, Инк. | Олигонуклеотидные аналоги, нацеленные на lmna человека |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MA45188A (fr) | 2016-06-03 | 2019-04-10 | Wave Life Sciences Ltd | Oligonucléotides, compositions et méthodes associées |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| EP3478697A1 (en) | 2016-06-30 | 2019-05-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| CR20190071A (es) * | 2016-07-15 | 2019-10-29 | Sperovie Biosciences Inc | Compuestos, composiciones y métodos para el tratamiento de enfermedades |
| US11981703B2 (en) | 2016-08-17 | 2024-05-14 | Sirius Therapeutics, Inc. | Polynucleotide constructs |
| IL264685B (en) | 2016-08-23 | 2022-09-01 | Dicerna Pharmaceuticals Inc | Compositions comprising reversibly modified oligonucleotides and uses thereof |
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| MA46905A (fr) | 2016-11-23 | 2019-10-02 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
| JOP20170192A1 (ar) | 2016-12-01 | 2019-01-30 | Takeda Pharmaceuticals Co | داي نوكليوتيد حلقي |
| EP4115908B1 (en) | 2016-12-19 | 2025-08-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| JP7118063B2 (ja) | 2016-12-19 | 2022-08-15 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| IL304284B2 (en) | 2016-12-19 | 2025-03-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer fusions for muscular dystrophy |
| CN110475784B (zh) * | 2017-03-29 | 2024-07-16 | 罗氏创新中心哥本哈根有限公司 | 用于制备立体限定硫代磷酸酯寡核苷酸的正交保护基 |
| US11638717B2 (en) | 2017-03-29 | 2023-05-02 | Shionogi & Co., Ltd. | Complex of nucleic acid medicine and multibranched lipid |
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| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
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| AU2018333065B2 (en) | 2017-09-18 | 2024-04-18 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| 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 |
| JP2020536057A (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 |
| WO2019067975A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| 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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| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
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| US9744183B2 (en) | 2017-08-29 |
| AU2010270714B2 (en) | 2015-08-13 |
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| EP2451461A4 (en) | 2013-05-29 |
| BR112012000828A2 (pt) | 2016-02-23 |
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| AU2010270714A1 (en) | 2012-02-16 |
| WO2011005761A1 (en) | 2011-01-13 |
| RU2612521C2 (ru) | 2017-03-09 |
| US20120316224A1 (en) | 2012-12-13 |
| BR112012000828A8 (pt) | 2017-10-10 |
| JP2012532199A (ja) | 2012-12-13 |
| RU2012102480A (ru) | 2013-08-20 |
| IL217370A (en) | 2017-09-28 |
| IL217370A0 (en) | 2012-02-29 |
| KR20120046238A (ko) | 2012-05-09 |
| EP2451461A1 (en) | 2012-05-16 |
| CL2012000021A1 (es) | 2012-07-20 |
| MX342945B (es) | 2016-10-18 |
| US20160347784A1 (en) | 2016-12-01 |
| KR101885383B1 (ko) | 2018-08-03 |
| JP2015205910A (ja) | 2015-11-19 |
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