JP2025507372A - 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法 - Google Patents

微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法 Download PDF

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Publication number
JP2025507372A
JP2025507372A JP2024547599A JP2024547599A JP2025507372A JP 2025507372 A JP2025507372 A JP 2025507372A JP 2024547599 A JP2024547599 A JP 2024547599A JP 2024547599 A JP2024547599 A JP 2024547599A JP 2025507372 A JP2025507372 A JP 2025507372A
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Japan
Prior art keywords
nucleotides
dsrna agent
tau
nucleotide
antisense strand
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Pending
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JP2024547599A
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English (en)
Japanese (ja)
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JP2025507372A5 (https=
JPWO2023154900A5 (https=
Inventor
ズーバー，ジェフリー
エドワード ファーリー，ジョナサン
カストレノ，アダム
ヘインズ，ジェファリー
ギャノン，ショーン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regeneron Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc
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Publication of JP2025507372A publication Critical patent/JP2025507372A/ja
Publication of JP2025507372A5 publication Critical patent/JP2025507372A5/ja
Publication of JPWO2023154900A5 publication Critical patent/JPWO2023154900A5/ja
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024547599A 2022-02-11 2023-02-10 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法 Pending JP2025507372A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263309399P 2022-02-11 2022-02-11
US63/309,399 2022-02-11
US202263322618P 2022-03-22 2022-03-22
US63/322,618 2022-03-22
PCT/US2023/062440 WO2023154900A2 (en) 2022-02-11 2023-02-10 Microtubule associated protein tau (mapt) irna agent compositions and methods of use thereof

Publications (3)

Publication Number Publication Date
JP2025507372A true JP2025507372A (ja) 2025-03-18
JP2025507372A5 JP2025507372A5 (https=) 2026-02-17
JPWO2023154900A5 JPWO2023154900A5 (https=) 2026-02-17

Family

ID=87565165

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024547599A Pending JP2025507372A (ja) 2022-02-11 2023-02-10 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法

Country Status (4)

Country Link
US (1) US20250171777A1 (https=)
EP (1) EP4476338A2 (https=)
JP (1) JP2025507372A (https=)
WO (1) WO2023154900A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2026048822A1 (ja) * 2024-08-30 2026-03-05 東亞合成株式会社 Ｍａｐｔ ｒｎａ配列に基づく新規二本鎖ｒｎａ及びその利用

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008124066A1 (en) 2007-04-05 2008-10-16 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
EP3274456B1 (en) 2015-03-25 2020-06-24 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
AU2021246024A1 (en) * 2020-03-30 2022-10-27 Alnylam Pharmaceuticals, Inc. Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof

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Publication number Publication date
WO2023154900A2 (en) 2023-08-17
US20250171777A1 (en) 2025-05-29
WO2023154900A3 (en) 2023-09-28
EP4476338A2 (en) 2024-12-18

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