JP7667775B2 - ヌクレオチド類似体を含有するオリゴヌクレオチド - Google Patents

ヌクレオチド類似体を含有するオリゴヌクレオチド Download PDF

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JP7667775B2
JP7667775B2 JP2022514677A JP2022514677A JP7667775B2 JP 7667775 B2 JP7667775 B2 JP 7667775B2 JP 2022514677 A JP2022514677 A JP 2022514677A JP 2022514677 A JP2022514677 A JP 2022514677A JP 7667775 B2 JP7667775 B2 JP 7667775B2
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formula
nucleotide
group
oligonucleotide
double
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JP2022547888A5 (https=
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JP2022547888A (ja
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ボードー・ブルナー
アルミン・ホフマイスター
ケルスティーン・ヤーン－ホフマン
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Sanofi SA
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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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C07H21/04—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 deoxyribosyl as saccharide radical
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
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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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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022514677A 2019-09-05 2020-09-04 ヌクレオチド類似体を含有するオリゴヌクレオチド Active JP7667775B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19195696 2019-09-05
EP19195696.0 2019-09-05
PCT/EP2020/074800 WO2021044004A1 (en) 2019-09-05 2020-09-04 Oligonucleotides containing nucleotide analogs

Publications (4)

Publication Number Publication Date
JP2022547888A JP2022547888A (ja) 2022-11-16
JP2022547888A5 JP2022547888A5 (https=) 2025-01-08
JPWO2021044004A5 JPWO2021044004A5 (https=) 2025-01-08
JP7667775B2 true JP7667775B2 (ja) 2025-04-23

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US (1) US20220372063A1 (https=)
EP (1) EP4025582A1 (https=)
JP (1) JP7667775B2 (https=)
CN (1) CN114728999A (https=)
WO (1) WO2021044004A1 (https=)

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Publication number Priority date Publication date Assignee Title
EP3762395A1 (en) 2018-03-07 2021-01-13 Sanofi Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same
EP3884051A2 (en) 2018-11-23 2021-09-29 Sanofi Novel rna compositions and methods for inhibiting angptl8
CA3226457A1 (en) * 2021-07-30 2023-02-02 Amy WALKER Nanoparticles and peptides for the delivery of cargos to muscle cells
WO2024002006A1 (zh) * 2022-06-27 2024-01-04 大睿生物医药科技(上海)有限公司 具有增强的稳定性的核苷酸替代物
JP2025532985A (ja) 2022-09-30 2025-10-03 アルナイラム ファーマシューティカルズ， インコーポレイテッド 修飾二本鎖ｒｎａ剤
WO2025031479A1 (zh) * 2023-08-10 2025-02-13 大睿生物 含有核苷酸类似物的双链rna
WO2025077949A1 (zh) * 2023-11-06 2025-04-17 杭州天龙药业有限公司 提升双链寡核苷酸靶基因抑制效果的修饰模板及其组合与应用
CN118994133A (zh) * 2023-12-08 2024-11-22 浙江大学 基于无保护扩环策略的吗啉代核苷单体全流程合成工艺
WO2025240414A1 (en) * 2024-05-14 2025-11-20 Sri International Cardiomyocyte-specific molecular guidance system peptides and uses thereof
WO2026002117A1 (zh) * 2024-06-26 2026-01-02 上海拓界生物医药科技有限公司 双环结构及包含其的寡核苷酸
WO2026064467A1 (en) * 2024-09-18 2026-03-26 Eli Lilly And Company Improved chemical methods for manufacturing a phosphoramidite used in rna synthesis

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JP2010528041A (ja) 2007-05-22 2010-08-19 エムディーアールエヌエー，インコーポレイテッド ヒドロキシメチル置換ｒｎａオリゴヌクレオチドおよびｒｎａ複合体
US20150315582A1 (en) 2013-01-16 2015-11-05 Iowa State University Research Foundation, Inc. Deep intronic target for splicing correction on spinal muscular atrophy gene
JP2018518148A (ja) 2015-03-18 2018-07-12 サレプタ セラピューティクス，インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
JP2021517126A (ja) 2018-03-07 2021-07-15 サノフイＳａｎｏｆｉ ヌクレオチド前駆体、ヌクレオチド類似体およびこれを含むオリゴマー化合物
JP2022513111A (ja) 2018-11-23 2022-02-07 サノフイ Ａｎｇｐｔｌ８を阻害するための新規のｒｎａ組成物および方法
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Publication number Priority date Publication date Assignee Title
JP2006109846A (ja) 1994-08-09 2006-04-27 Novartis Ag 抗腫瘍性アンチセンスオリゴヌクレオチド
JP2010528041A (ja) 2007-05-22 2010-08-19 エムディーアールエヌエー，インコーポレイテッド ヒドロキシメチル置換ｒｎａオリゴヌクレオチドおよびｒｎａ複合体
US20150315582A1 (en) 2013-01-16 2015-11-05 Iowa State University Research Foundation, Inc. Deep intronic target for splicing correction on spinal muscular atrophy gene
JP2018518148A (ja) 2015-03-18 2018-07-12 サレプタ セラピューティクス，インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
JP2021517126A (ja) 2018-03-07 2021-07-15 サノフイＳａｎｏｆｉ ヌクレオチド前駆体、ヌクレオチド類似体およびこれを含むオリゴマー化合物
JP2022513111A (ja) 2018-11-23 2022-02-07 サノフイ Ａｎｇｐｔｌ８を阻害するための新規のｒｎａ組成物および方法
JP2022546040A (ja) 2019-08-27 2022-11-02 サノフイ Ｐｃｓｋ９を阻害するための組成物および方法

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CN114728999A (zh) 2022-07-08
US20220372063A1 (en) 2022-11-24
EP4025582A1 (en) 2022-07-13
WO2021044004A1 (en) 2021-03-11
JP2022547888A (ja) 2022-11-16

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