JP7289610B2 - 修飾二本鎖rna剤 - Google Patents
修飾二本鎖rna剤 Download PDFInfo
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- JP7289610B2 JP7289610B2 JP2017509743A JP2017509743A JP7289610B2 JP 7289610 B2 JP7289610 B2 JP 7289610B2 JP 2017509743 A JP2017509743 A JP 2017509743A JP 2017509743 A JP2017509743 A JP 2017509743A JP 7289610 B2 JP7289610 B2 JP 7289610B2
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
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- 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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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
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- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
Priority Applications (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020212527A JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2020212528A JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2023178574A JP7654744B2 (ja) | 2014-08-20 | 2023-10-17 | 修飾二本鎖rna剤 |
| JP2025044214A JP2025111426A (ja) | 2014-08-20 | 2025-03-19 | 修飾二本鎖rna剤 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462039507P | 2014-08-20 | 2014-08-20 | |
| US62/039,507 | 2014-08-20 | ||
| US201462083744P | 2014-11-24 | 2014-11-24 | |
| US62/083,744 | 2014-11-24 | ||
| US201462093919P | 2014-12-18 | 2014-12-18 | |
| US62/093,919 | 2014-12-18 | ||
| PCT/US2015/045407 WO2016028649A1 (en) | 2014-08-20 | 2015-08-14 | Modified double-stranded rna agents |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020212528A Division JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2020212527A Division JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017525705A JP2017525705A (ja) | 2017-09-07 |
| JP2017525705A5 JP2017525705A5 (enExample) | 2018-09-20 |
| JP7289610B2 true JP7289610B2 (ja) | 2023-06-12 |
Family
ID=53969461
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017509743A Active JP7289610B2 (ja) | 2014-08-20 | 2015-08-14 | 修飾二本鎖rna剤 |
| JP2020212528A Active JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2020212527A Active JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2023178574A Active JP7654744B2 (ja) | 2014-08-20 | 2023-10-17 | 修飾二本鎖rna剤 |
| JP2025044214A Pending JP2025111426A (ja) | 2014-08-20 | 2025-03-19 | 修飾二本鎖rna剤 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020212528A Active JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2020212527A Active JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖rna剤 |
| JP2023178574A Active JP7654744B2 (ja) | 2014-08-20 | 2023-10-17 | 修飾二本鎖rna剤 |
| JP2025044214A Pending JP2025111426A (ja) | 2014-08-20 | 2025-03-19 | 修飾二本鎖rna剤 |
Country Status (12)
| Country | Link |
|---|---|
| US (11) | US10233448B2 (enExample) |
| EP (4) | EP3812462A1 (enExample) |
| JP (5) | JP7289610B2 (enExample) |
| KR (3) | KR102630289B1 (enExample) |
| CN (3) | CN107075516A (enExample) |
| CA (1) | CA2958758A1 (enExample) |
| EA (1) | EA201790420A1 (enExample) |
| IL (3) | IL316808A (enExample) |
| MX (3) | MX2017002144A (enExample) |
| NZ (1) | NZ730296A (enExample) |
| SG (3) | SG10201903290YA (enExample) |
| WO (1) | WO2016028649A1 (enExample) |
Families Citing this family (152)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG10201809460SA (en) | 2008-10-20 | 2018-11-29 | Alnylam Pharmaceuticals Inc | Compositions and methods for inhibiting expression of transthyretin |
| CN103890000B (zh) | 2011-06-21 | 2017-09-01 | 阿尔尼拉姆医药品有限公司 | 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法 |
| HK1200191A1 (en) | 2011-11-18 | 2015-07-31 | Alnylam Pharmaceuticals, Inc. | Modified rnai agents |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| EP3412774A1 (en) | 2013-05-22 | 2018-12-12 | Alnylam Pharmaceuticals, Inc. | Serpina1 irna compositions and methods of use thereof |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| KR20250005546A (ko) * | 2015-04-13 | 2025-01-09 | 알닐람 파마슈티칼스 인코포레이티드 | 안지오포이에틴-유사 3(angptl3) irna 조성물 및 이의 이용 방법 |
| PL3329002T3 (pl) | 2015-07-31 | 2021-04-19 | Alnylam Pharmaceuticals, Inc. | Kompozycje irna transtyretyny (ttr) i sposoby ich zastosowania do leczenia lub zapobiegania chorobom związanym z ttr |
| EP3334499A4 (en) | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CN108778345B (zh) * | 2016-02-01 | 2022-11-29 | 阿拉基斯医疗公司 | 治疗rna介导的疾病的化合物和方法 |
| WO2017173301A1 (en) | 2016-04-01 | 2017-10-05 | Avidity Biosciences Llc | Egfr nucleic acids and uses thereof |
| MA45470A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques kras et leurs utilisations |
| KR102775461B1 (ko) | 2016-04-01 | 2025-02-28 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩타이드 조성물 및 이의 용도 |
| EP3436582B1 (en) | 2016-04-01 | 2021-10-13 | Avidity Biosciences, Inc. | Beta-catenin nucleic acids and uses thereof |
| CA3023764A1 (en) | 2016-06-06 | 2017-12-14 | Arrowhead Pharmaceuticals, Inc. | 5'-cyclo-phosphonate modified nucleotides |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| EP3500581A4 (en) * | 2016-08-17 | 2021-10-06 | Solstice Biologics, Ltd. | POLYNUCLEOTIDE CONSTRUCTS |
| WO2018085752A2 (en) * | 2016-11-07 | 2018-05-11 | nanoSUR LLC | Post-transcriptionally chemically modified double strand rnas |
| WO2018089582A1 (en) | 2016-11-10 | 2018-05-17 | San Diego State University Research Foundation | Compounds for fluorescence sensing of duplex formation |
| TW202313978A (zh) | 2016-11-23 | 2023-04-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法 |
| CN117757791A (zh) * | 2016-11-23 | 2024-03-26 | 阿尔尼拉姆医药品有限公司 | 具有降低的脱靶效应的修饰的rna试剂 |
| EP3554561B1 (en) | 2016-12-14 | 2023-06-28 | Janssen Biotech, Inc. | Cd137 binding fibronectin type iii domains |
| AU2017378226A1 (en) | 2016-12-14 | 2019-06-20 | Janssen Biotech, Inc. | CD8A-binding fibronectin type III domains |
| SG10201913552UA (en) * | 2016-12-16 | 2020-03-30 | Alnylam Pharmaceuticals Inc | Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions |
| EP3550022A1 (en) * | 2018-04-05 | 2019-10-09 | Silence Therapeutics GmbH | Products and compositions |
| SI3607068T1 (sl) * | 2017-04-05 | 2023-02-28 | Silence Therapeutics Gmbh | Z RNA interferenco posredovano inhibiranje TMPRSS6 |
| RS63836B1 (sr) | 2017-04-05 | 2023-01-31 | Silence Therapeutics Gmbh | Proizvodi i sastavi |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| EP3645546A4 (en) | 2017-06-30 | 2021-12-01 | Solstice Biologics, Ltd. | CHIRAL PHOSPHORAMIDITIS AUXILIARIES AND THEIR METHODS OF USE |
| AU2018331383B2 (en) | 2017-09-14 | 2024-12-05 | Arrowhead Pharmaceuticals, Inc. | RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use |
| BR112020005230A2 (pt) | 2017-09-19 | 2020-09-24 | Alnylam Pharmaceuticals, Inc. | composições e métodos para o tratamento da amiloidose mediada por transtiretina (ttr) |
| CN111655268B (zh) * | 2017-10-04 | 2024-03-26 | 艾维迪提生物科学公司 | 核酸-多肽组合物及其用途 |
| CN111527206A (zh) * | 2017-11-13 | 2020-08-11 | 赛伦斯治疗有限责任公司 | 用于抑制靶标基因表达的包含二硫代磷酸酯键的核酸 |
| WO2019109046A1 (en) | 2017-11-30 | 2019-06-06 | Arrakis Therapeutics, Inc. | Nucleic acid-binding photoprobes and uses thereof |
| 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 |
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