JP7028865B2 - ５’－シクロ－ホスホン酸修飾ヌクレオチド - Google Patents

５’－シクロ－ホスホン酸修飾ヌクレオチド Download PDF

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JP7028865B2
JP7028865B2 JP2019516096A JP2019516096A JP7028865B2 JP 7028865 B2 JP7028865 B2 JP 7028865B2 JP 2019516096 A JP2019516096 A JP 2019516096A JP 2019516096 A JP2019516096 A JP 2019516096A JP 7028865 B2 JP7028865 B2 JP 7028865B2
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JP2019517588A (ja
JP2019517588A5 (enExample
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リー チェン
ペイ タオ
ローラー マイケル
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アローヘッド ファーマシューティカルズ インコーポレイテッド
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    • 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
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    • 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
    • C07H19/067—Pyrimidine radicals with ribosyl as the saccharide radical
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
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    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
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    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • C07H19/207—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids the phosphoric or polyphosphoric acids being esterified by a further hydroxylic compound, e.g. flavine adenine dinucleotide or nicotinamide-adenine dinucleotide
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    • 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
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    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • 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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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3233—Morpholino-type ring
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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
    • C12N2310/3519—Fusion with another nucleic acid

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  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2019516096A 2016-06-06 2017-06-06 ５’－シクロ－ホスホン酸修飾ヌクレオチド Active JP7028865B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022022652A JP7530927B2 (ja) 2016-06-06 2022-02-17 ５’－シクロ－ホスホン酸修飾ヌクレオチド
JP2024074835A JP2024097860A (ja) 2016-06-06 2024-05-02 ５’－シクロ－ホスホン酸修飾ヌクレオチド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662346304P 2016-06-06 2016-06-06
US62/346,304 2016-06-06
PCT/US2017/036108 WO2017214112A1 (en) 2016-06-06 2017-06-06 5'-cyclo-phosphonate modified nucleotides

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JP2022022652A Division JP7530927B2 (ja) 2016-06-06 2022-02-17 ５’－シクロ－ホスホン酸修飾ヌクレオチド

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JP2019517588A JP2019517588A (ja) 2019-06-24
JP2019517588A5 JP2019517588A5 (enExample) 2020-07-16
JP7028865B2 true JP7028865B2 (ja) 2022-03-02

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JP2019516096A Active JP7028865B2 (ja) 2016-06-06 2017-06-06 ５’－シクロ－ホスホン酸修飾ヌクレオチド
JP2022022652A Active JP7530927B2 (ja) 2016-06-06 2022-02-17 ５’－シクロ－ホスホン酸修飾ヌクレオチド
JP2024074835A Pending JP2024097860A (ja) 2016-06-06 2024-05-02 ５’－シクロ－ホスホン酸修飾ヌクレオチド

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JP2024074835A Pending JP2024097860A (ja) 2016-06-06 2024-05-02 ５’－シクロ－ホスホン酸修飾ヌクレオチド

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US (2) US11078227B2 (enExample)
EP (1) EP3464313A4 (enExample)
JP (3) JP7028865B2 (enExample)
KR (2) KR102639586B1 (enExample)
CN (2) CN114736256B (enExample)
AU (4) AU2017279512B2 (enExample)
CA (1) CA3023764A1 (enExample)
EA (1) EA201892285A1 (enExample)
IL (2) IL290633B2 (enExample)
MX (2) MX2018015109A (enExample)
TW (2) TWI912633B (enExample)
WO (1) WO2017214112A1 (enExample)

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JP2025508785A (ja) 2022-02-22 2025-04-10 セーンジーン バイオ ユーエスエー インコーポレイティド ５’修飾炭素環式リボヌクレオチド誘導体及び使用方法
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AU2023320578A1 (en) 2022-08-05 2025-02-13 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
JP2025533554A (ja) * 2022-09-21 2025-10-07 ヤンセン バイオテツク，インコーポレーテツド 新規の安定化されたヌクレオシドホスフェート及びその類似体
IL320218A (en) 2022-10-14 2025-06-01 Sanegene Bio Usa Inc Small interfering RNA targeting C3 and its uses
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US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
JP2026511018A (ja) 2023-03-21 2026-04-10 アローヘッド ファーマシューティカルズ インコーポレイテッド 補体因子Ｂ（ＣＦＢ）の発現を阻害するためのＲＮＡｉ剤、その医薬組成物、及び使用方法
WO2024208249A1 (zh) * 2023-04-06 2024-10-10 上海舶望制药有限公司 5'-膦酸酯修饰的核苷类似物及其制备的寡核苷酸
EP4731764A2 (en) 2023-06-21 2026-04-29 Sanegene Bio USA Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
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WO2025137390A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of activin receptor-like kinase 7 (alk7), compositions thereof, and methods of use
WO2025160275A1 (en) 2024-01-24 2025-07-31 Arrowhead Pharmaceuticals, Inc. Anti-transferrin receptor antibodies, conjugates thereof, and uses thereof
WO2025212467A1 (en) 2024-03-31 2025-10-09 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of microtubule associated protein tau (mapt), compositions thereof, and methods of use
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use
WO2026007905A1 (zh) * 2024-07-01 2026-01-08 成都新泽利医药科技有限公司 5'-螺环膦酸酯修饰的核苷酸
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof
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