CN112424352A - 包括7′-5′-α-异头双环糖核苷的寡核苷酸缀合物 - Google Patents
包括7′-5′-α-异头双环糖核苷的寡核苷酸缀合物 Download PDFInfo
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- CN112424352A CN112424352A CN201980046260.3A CN201980046260A CN112424352A CN 112424352 A CN112424352 A CN 112424352A CN 201980046260 A CN201980046260 A CN 201980046260A CN 112424352 A CN112424352 A CN 112424352A
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- 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
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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
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- 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
- A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
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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
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/337—Chemical structure of the base in alpha-anomeric form
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862670319P | 2018-05-11 | 2018-05-11 | |
| US62/670,319 | 2018-05-11 | ||
| PCT/EP2019/062064 WO2019215333A1 (en) | 2018-05-11 | 2019-05-10 | Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112424352A true CN112424352A (zh) | 2021-02-26 |
Family
ID=66530048
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980046260.3A Pending CN112424352A (zh) | 2018-05-11 | 2019-05-10 | 包括7′-5′-α-异头双环糖核苷的寡核苷酸缀合物 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US20230132377A9 (https=) |
| EP (1) | EP3790968A1 (https=) |
| JP (1) | JP7665509B2 (https=) |
| KR (1) | KR20210008369A (https=) |
| CN (1) | CN112424352A (https=) |
| AU (1) | AU2019266550A1 (https=) |
| BR (1) | BR112020022620A2 (https=) |
| CA (1) | CA3098266A1 (https=) |
| SG (1) | SG11202010841QA (https=) |
| WO (1) | WO2019215333A1 (https=) |
| ZA (1) | ZA202007564B (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3330276A1 (en) * | 2016-11-30 | 2018-06-06 | Universität Bern | Novel bicyclic nucleosides and oligomers prepared therefrom |
| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US20220370491A1 (en) * | 2019-09-18 | 2022-11-24 | National University Corporation Tokyo Medical And Dental University | Nucleic acid complex |
| EP4213883A1 (en) * | 2020-09-16 | 2023-07-26 | Astrazeneca AB | Oligonucleotides conjugated to fatty acids |
| WO2022106695A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| IL309936A (en) | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CN120225676A (zh) | 2022-12-02 | 2025-06-27 | 上海舶望制药有限公司 | 双环脱碱基核酸类似物以及由它们制备的寡聚化合物 |
| WO2025217304A1 (en) * | 2024-04-09 | 2025-10-16 | Bio-Rad Laboratories, Inc. | Compositions and methods for detection of contaminant enzymes |
Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20010056077A1 (en) * | 1999-05-21 | 2001-12-27 | Jcr Pharmaceuticals Co., Ltd | Pharmaceutical composition for treatment of duchenne muscular dystrophy |
| US6653467B1 (en) * | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| WO2010036698A1 (en) * | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| CN102851291A (zh) * | 2006-04-03 | 2013-01-02 | 桑塔里斯制药公司 | 包含抗微小rna 反义寡核苷酸的药物组合物 |
| CN106581043A (zh) * | 2016-10-24 | 2017-04-26 | 佳木斯大学 | 一种适用于杜氏肌营养不良症的药物制剂及其制备方法 |
| CN110023322A (zh) * | 2016-11-30 | 2019-07-16 | 伯尔尼大学 | 新的双环核苷和由其制备的寡聚体 |
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| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
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| ATE162219T1 (de) | 1989-11-16 | 1998-01-15 | Univ Duke | Transformation von tierischen hautzellen mit hilfe von partikeln |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| JP3257675B2 (ja) | 1990-10-12 | 2002-02-18 | マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. | 修飾リボザイム |
| US5858784A (en) | 1991-12-17 | 1999-01-12 | The Regents Of The University Of California | Expression of cloned genes in the lung by aerosol- and liposome-based delivery |
| US6020317A (en) | 1993-02-19 | 2000-02-01 | Nippon Shinyaku Co. Ltd. | Glycerol derivative, device and pharmaceutical composition |
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| US5994316A (en) | 1996-02-21 | 1999-11-30 | The Immune Response Corporation | Method of preparing polynucleotide-carrier complexes for delivery to cells |
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| US6042820A (en) | 1996-12-20 | 2000-03-28 | Connaught Laboratories Limited | Biodegradable copolymer containing α-hydroxy acid and α-amino acid units |
| US6248878B1 (en) | 1996-12-24 | 2001-06-19 | Ribozyme Pharmaceuticals, Inc. | Nucleoside analogs |
| US6472375B1 (en) | 1998-04-16 | 2002-10-29 | John Wayne Cancer Institute | DNA vaccine and methods for its use |
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| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US10077443B2 (en) * | 2012-11-15 | 2018-09-18 | Roche Innovation Center Copenhagen A/S | Oligonucleotide conjugates |
| CN104955952A (zh) * | 2013-01-30 | 2015-09-30 | 弗·哈夫曼-拉罗切有限公司 | Lna寡核苷酸碳水化合物缀合物 |
-
2019
- 2019-05-10 CA CA3098266A patent/CA3098266A1/en active Pending
- 2019-05-10 US US17/054,724 patent/US20230132377A9/en not_active Abandoned
- 2019-05-10 JP JP2021513004A patent/JP7665509B2/ja active Active
- 2019-05-10 WO PCT/EP2019/062064 patent/WO2019215333A1/en not_active Ceased
- 2019-05-10 CN CN201980046260.3A patent/CN112424352A/zh active Pending
- 2019-05-10 KR KR1020207034385A patent/KR20210008369A/ko not_active Ceased
- 2019-05-10 EP EP19723781.1A patent/EP3790968A1/en active Pending
- 2019-05-10 AU AU2019266550A patent/AU2019266550A1/en not_active Abandoned
- 2019-05-10 SG SG11202010841QA patent/SG11202010841QA/en unknown
- 2019-05-10 BR BR112020022620-3A patent/BR112020022620A2/pt not_active IP Right Cessation
-
2020
- 2020-12-04 ZA ZA2020/07564A patent/ZA202007564B/en unknown
-
2024
- 2024-09-10 US US18/830,182 patent/US20250250565A1/en active Pending
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20010056077A1 (en) * | 1999-05-21 | 2001-12-27 | Jcr Pharmaceuticals Co., Ltd | Pharmaceutical composition for treatment of duchenne muscular dystrophy |
| US6653467B1 (en) * | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| CN102851291A (zh) * | 2006-04-03 | 2013-01-02 | 桑塔里斯制药公司 | 包含抗微小rna 反义寡核苷酸的药物组合物 |
| WO2010036698A1 (en) * | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| CN106581043A (zh) * | 2016-10-24 | 2017-04-26 | 佳木斯大学 | 一种适用于杜氏肌营养不良症的药物制剂及其制备方法 |
| CN110023322A (zh) * | 2016-11-30 | 2019-07-16 | 伯尔尼大学 | 新的双环核苷和由其制备的寡聚体 |
Non-Patent Citations (2)
| Title |
|---|
| NICOLAS WEIN 等: "Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach", 《J NEUROMUSCUL DIS》, vol. 4, no. 3, pages 199 - 207 * |
| 黄世杰: "外显子53跳跃药物戈洛地生治疗杜氏肌营养不良症试验呈阳性结果", 《国际药学研究杂志》, vol. 44, no. 10, pages 979 * |
Also Published As
| Publication number | Publication date |
|---|---|
| US20220017898A1 (en) | 2022-01-20 |
| JP7665509B2 (ja) | 2025-04-21 |
| US20230132377A9 (en) | 2023-04-27 |
| BR112020022620A2 (pt) | 2021-02-17 |
| CA3098266A1 (en) | 2019-11-14 |
| KR20210008369A (ko) | 2021-01-21 |
| US20250250565A1 (en) | 2025-08-07 |
| EP3790968A1 (en) | 2021-03-17 |
| SG11202010841QA (en) | 2020-11-27 |
| ZA202007564B (en) | 2021-10-27 |
| AU2019266550A1 (en) | 2020-11-26 |
| JP2021522862A (ja) | 2021-09-02 |
| WO2019215333A1 (en) | 2019-11-14 |
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