JP2023546369A - Ｎ－アセチルガラクトサミン（ＧａｌＮＡｃ）から誘導された化合物およびオリゴヌクレオチド - Google Patents

Ｎ－アセチルガラクトサミン（ＧａｌＮＡｃ）から誘導された化合物およびオリゴヌクレオチド Download PDF

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JP2023546369A
JP2023546369A JP2023521602A JP2023521602A JP2023546369A JP 2023546369 A JP2023546369 A JP 2023546369A JP 2023521602 A JP2023521602 A JP 2023521602A JP 2023521602 A JP2023521602 A JP 2023521602A JP 2023546369 A JP2023546369 A JP 2023546369A
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alkyl
optionally substituted
compound
galnac
compound according
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JP2023546369A5 (https=
Inventor
ジェン リー，
ルイ ジュー，
メフディ ミシェル ジャメル ヌマ，
ボー チェン，
チェイス ロバート オルソン，
チャンドラモウリ チルタ，
インドラセナ レディ クメタ，
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エーダーエックス 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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Publication of JP2023546369A publication Critical patent/JP2023546369A/ja
Publication of JP2023546369A5 publication Critical patent/JP2023546369A5/ja
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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
    • C07H19/067—Pyrimidine radicals with ribosyl as the saccharide radical
    • 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
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • 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
    • C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/55—Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023521602A 2020-10-09 2021-10-08 Ｎ－アセチルガラクトサミン（ＧａｌＮＡｃ）から誘導された化合物およびオリゴヌクレオチド Pending JP2023546369A (ja)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202063089936P 2020-10-09 2020-10-09
US63/089,936 2020-10-09
US202063113801P 2020-11-13 2020-11-13
US63/113,801 2020-11-13
US202063131317P 2020-12-29 2020-12-29
US63/131,317 2020-12-29
US202163141300P 2021-01-25 2021-01-25
US63/141,300 2021-01-25
US202163146276P 2021-02-05 2021-02-05
US63/146,276 2021-02-05
US202163251580P 2021-10-01 2021-10-01
US63/251,580 2021-10-01
PCT/US2021/054318 WO2022076922A1 (en) 2020-10-09 2021-10-08 N-ACETYLGALACTOSAMINE(GAlNAc)-DERIVED COMPOUNDS AND OLIGONUCLEOTIDES

Publications (2)

Publication Number Publication Date
JP2023546369A true JP2023546369A (ja) 2023-11-02
JP2023546369A5 JP2023546369A5 (https=) 2024-10-16

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JP2023521602A Pending JP2023546369A (ja) 2020-10-09 2021-10-08 Ｎ－アセチルガラクトサミン（ＧａｌＮＡｃ）から誘導された化合物およびオリゴヌクレオチド

Country Status (10)

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US (1) US20240083934A1 (https=)
EP (1) EP4225322A4 (https=)
JP (1) JP2023546369A (https=)
KR (1) KR20230113285A (https=)
CN (2) CN121108223A (https=)
AU (1) AU2021359017A1 (https=)
CA (1) CA3198406A1 (https=)
IL (1) IL302000A (https=)
MX (1) MX2023004170A (https=)
WO (1) WO2022076922A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7751773B1 (ja) 2024-08-15 2025-10-08 ベイジン ユーカークチュアン ファーマシューティカル テクノロジー カンパニー，リミテッド トリアゾール構造を含有するＧａｌＮＡｃ化合物、およびそのオリゴヌクレオチドコンジュゲート

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11649260B2 (en) 2021-06-18 2023-05-16 Hongene Biotech Corporation Functionalized N-acetylgalactosamine nucleosides
EP4396193A4 (en) 2021-08-30 2025-07-02 Hongene Biotech Corp FUNCTIONALIZED N-ACETYLGALACTOSAMINE ANALOGUES
JP2024546585A (ja) 2021-12-15 2024-12-26 ホンジーン バイオテック コーポレイション 官能基化ｎ－アセチルガラクトサミンアナログ
JP2025517196A (ja) * 2022-05-13 2025-06-03 エーダーエックス ファーマシューティカルズ， インコーポレイテッド 合成骨格を有するオリゴヌクレオチド及びその合成
WO2024118503A1 (en) 2022-11-28 2024-06-06 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
CN119019354B (zh) * 2023-05-24 2025-07-22 北京炫景瑞医药科技有限公司 化合物及其制备方法和用途
WO2024240101A1 (zh) * 2023-05-24 2024-11-28 北京炫景瑞医药科技有限公司 GalNAc化合物、缀合物、组合物以及它们的用途
CN119019475B (zh) * 2023-05-24 2026-02-06 北京炫景瑞医药科技有限公司 GalNAc化合物、缀合物、组合物以及它们的用途
WO2025140434A1 (zh) * 2023-12-26 2025-07-03 成都倍特药业股份有限公司 一种肝靶向化合物及其在药物缀合物中的用途
CN121263426A (zh) * 2024-05-09 2026-01-02 石药集团中奇制药技术（石家庄）有限公司 GalNAc衍生物及其寡聚核苷酸缀合物

Citations (1)

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JP2012513953A (ja) * 2008-09-23 2012-06-21 アルニラム ファーマスーティカルズ インコーポレイテッド 付加環化を用いたモノマーおよびオリゴヌクレオチドの化学修飾

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US6472378B2 (en) * 1998-08-31 2002-10-29 Pro-Neuron, Inc. Compositions and methods for treatment of mitochondrial diseases
US6852485B1 (en) * 2002-01-22 2005-02-08 California State University Fullerton Foundation Method for identifying a compound for the treatment of microorganism infections by inhibiting energy storage and utilization in pathogens
CN111808084A (zh) * 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
GB201408623D0 (en) * 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
KR20180031025A (ko) * 2015-07-31 2018-03-27 아크투루스 쎄라퓨틱스, 인크. 약물 전달을 위한 다중 리간드 제제
CA3020585A1 (en) * 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions

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JP2012513953A (ja) * 2008-09-23 2012-06-21 アルニラム ファーマスーティカルズ インコーポレイテッド 付加環化を用いたモノマーおよびオリゴヌクレオチドの化学修飾

Non-Patent Citations (3)

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7751773B1 (ja) 2024-08-15 2025-10-08 ベイジン ユーカークチュアン ファーマシューティカル テクノロジー カンパニー，リミテッド トリアゾール構造を含有するＧａｌＮＡｃ化合物、およびそのオリゴヌクレオチドコンジュゲート
JP2026034359A (ja) * 2024-08-15 2026-02-27 ベイジン ユーカークチュアン ファーマシューティカル テクノロジー カンパニー，リミテッド トリアゾール構造を含有するＧａｌＮＡｃ化合物、およびそのオリゴヌクレオチドコンジュゲート

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EP4225322A4 (en) 2024-12-11
IL302000A (en) 2023-06-01
WO2022076922A1 (en) 2022-04-14
CA3198406A1 (en) 2022-04-14
CN121108223A (zh) 2025-12-12
AU2021359017A9 (en) 2024-09-26
CN121108222A (zh) 2025-12-12
MX2023004170A (es) 2023-06-27
KR20230113285A (ko) 2023-07-28
AU2021359017A1 (en) 2023-05-25
US20240083934A1 (en) 2024-03-14
EP4225322A1 (en) 2023-08-16

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