JP2022530678A - 短縮センス鎖を有する二本鎖核酸阻害剤分子 - Google Patents
短縮センス鎖を有する二本鎖核酸阻害剤分子 Download PDFInfo
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- JP2022530678A JP2022530678A JP2021564975A JP2021564975A JP2022530678A JP 2022530678 A JP2022530678 A JP 2022530678A JP 2021564975 A JP2021564975 A JP 2021564975A JP 2021564975 A JP2021564975 A JP 2021564975A JP 2022530678 A JP2022530678 A JP 2022530678A
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- C—CHEMISTRY; METALLURGY
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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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- 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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- 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]
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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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- C12N2310/00—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
- C12N2310/35—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
- C12N2310/531—Stem-loop; Hairpin
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- 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
- C12N2310/50—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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- Health & Medical Sciences (AREA)
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- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962842908P | 2019-05-03 | 2019-05-03 | |
| US62/842,908 | 2019-05-03 | ||
| PCT/US2020/030398 WO2020226960A1 (en) | 2019-05-03 | 2020-04-29 | Double-stranded nucleic acid inhibitor molecules with shortened sense strands |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022530678A true JP2022530678A (ja) | 2022-06-30 |
| JP2022530678A5 JP2022530678A5 (enExample) | 2023-05-10 |
Family
ID=70802925
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564975A Withdrawn JP2022530678A (ja) | 2019-05-03 | 2020-04-29 | 短縮センス鎖を有する二本鎖核酸阻害剤分子 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US12529053B2 (enExample) |
| EP (1) | EP3963072A1 (enExample) |
| JP (1) | JP2022530678A (enExample) |
| KR (1) | KR20220004675A (enExample) |
| CN (1) | CN113795581A (enExample) |
| AU (1) | AU2020268798A1 (enExample) |
| BR (1) | BR112021021686A2 (enExample) |
| CA (1) | CA3136676A1 (enExample) |
| CL (1) | CL2021002842A1 (enExample) |
| IL (1) | IL287354A (enExample) |
| MX (1) | MX2021013418A (enExample) |
| SG (1) | SG11202111077RA (enExample) |
| WO (1) | WO2020226960A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HRP20240586T1 (hr) | 2018-10-09 | 2024-07-19 | The University Of British Columbia | Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima |
| WO2023081500A2 (en) * | 2021-11-08 | 2023-05-11 | Dicerna Pharmaceuticals, Inc. | RNAi OLIGONUCLEOTIDE CONJUGATES |
| WO2023207615A1 (zh) * | 2022-04-26 | 2023-11-02 | 南京明德新药研发有限公司 | 一类含由天然核苷酸组成突出端的双链RNAi化合物 |
Citations (7)
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| JP2010532872A (ja) * | 2007-07-06 | 2010-10-14 | プロメディオール, インコーポレイテッド | 線維化関連障害のための治療および診断方法 |
| JP2012500389A (ja) * | 2008-08-12 | 2012-01-05 | ジ・オハイオ・ステイト・ユニバーシティ・リサーチ・ファウンデイション | 多発性脊髄腫の診断、予後および治療のためのマイクロrnaに基づく組成物および方法 |
| JP2012504389A (ja) * | 2008-09-22 | 2012-02-23 | ダイセルナ ファーマシューティカルズ, インコーポレイテッド | 修飾を有するdsRNAによる遺伝子発現の特異的な阻害のための組成物および方法 |
| WO2018045317A1 (en) * | 2016-09-02 | 2018-03-08 | Dicerna Pharmaceuticals, Inc. | 4'-phosphate analogs and oligonucleotides comprising the same |
| WO2019136157A2 (en) * | 2018-01-05 | 2019-07-11 | Dicerna Pharmaceuticals, Inc. | Reducing beta-catenin and ido expression to potentiate immunotherapy |
| WO2019200124A1 (en) * | 2018-04-13 | 2019-10-17 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides |
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- 2020-04-29 JP JP2021564975A patent/JP2022530678A/ja not_active Withdrawn
- 2020-04-29 EP EP20727756.7A patent/EP3963072A1/en active Pending
- 2020-04-29 CN CN202080032477.1A patent/CN113795581A/zh not_active Withdrawn
- 2020-04-29 WO PCT/US2020/030398 patent/WO2020226960A1/en not_active Ceased
- 2020-04-29 KR KR1020217037233A patent/KR20220004675A/ko not_active Ceased
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| SG11202111077RA (en) | 2021-11-29 |
| IL287354A (en) | 2021-12-01 |
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| KR20220004675A (ko) | 2022-01-11 |
| CA3136676A1 (en) | 2020-11-12 |
| MX2021013418A (es) | 2021-12-10 |
| US12529053B2 (en) | 2026-01-20 |
| US20220177880A1 (en) | 2022-06-09 |
| CL2021002842A1 (es) | 2022-06-10 |
| EP3963072A1 (en) | 2022-03-09 |
| WO2020226960A1 (en) | 2020-11-12 |
| BR112021021686A2 (pt) | 2022-03-22 |
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