JP2024538646A - ＡＮＧＰＴＬ７を標的化するためのｉＲＮＡ組成物および方法 - Google Patents

ＡＮＧＰＴＬ７を標的化するためのｉＲＮＡ組成物および方法 Download PDF

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Publication number
JP2024538646A
JP2024538646A JP2024519765A JP2024519765A JP2024538646A JP 2024538646 A JP2024538646 A JP 2024538646A JP 2024519765 A JP2024519765 A JP 2024519765A JP 2024519765 A JP2024519765 A JP 2024519765A JP 2024538646 A JP2024538646 A JP 2024538646A
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Prior art keywords
nucleotides
angptl7
nucleotide
dsrna
strand
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JP2024519765A
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Japanese (ja)
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JP2024538646A5 (https=
Inventor
ディー マキニンチ，ジェイムズ
アール ジャダブ，バサント
エイ パンディア，バウミク
カステヤノス－リサルドス，エレナ
カストレノ，アダム
ロマノ，カーメロ
パテル，ガウラング
ホゥ，イン
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Regeneron Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc
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Publication of JP2024538646A publication Critical patent/JP2024538646A/ja
Publication of JP2024538646A5 publication Critical patent/JP2024538646A5/ja
Pending legal-status Critical Current

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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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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    • C12N2310/312—Phosphonates
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    • C12N2310/3125—Methylphosphonates
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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
    • C12N2310/30—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)
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  • Engineering & Computer Science (AREA)
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  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024519765A 2021-10-01 2022-10-01 ＡＮＧＰＴＬ７を標的化するためのｉＲＮＡ組成物および方法 Pending JP2024538646A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163251203P 2021-10-01 2021-10-01
US63/251,203 2021-10-01
US202163287414P 2021-12-08 2021-12-08
US63/287,414 2021-12-08
PCT/US2022/077447 WO2023056478A1 (en) 2021-10-01 2022-10-01 iRNA COMPOSITIONS AND METHODS FOR TARGETING ANGPTL7

Publications (2)

Publication Number Publication Date
JP2024538646A true JP2024538646A (ja) 2024-10-23
JP2024538646A5 JP2024538646A5 (https=) 2025-12-05

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ID=85783703

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JP2024519765A Pending JP2024538646A (ja) 2021-10-01 2022-10-01 ＡＮＧＰＴＬ７を標的化するためのｉＲＮＡ組成物および方法

Country Status (4)

Country Link
US (1) US20240409943A1 (https=)
EP (1) EP4408438A4 (https=)
JP (1) JP2024538646A (https=)
WO (1) WO2023056478A1 (https=)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004070062A2 (en) * 2003-02-04 2004-08-19 Wyeth Compositions and methods for diagnosing and treating cancers
EP2397559A1 (en) * 2010-06-17 2011-12-21 Max-Delbrück-Centrum Für Molekulare Medizin Stage-specific biomarkers for the diagnosis of acute kidney injury
MX2021008797A (es) * 2019-01-23 2022-01-31 Regeneron Pharma Tratamiento de afecciones oftalmicas con inhibidores de proteinas relacionadas a angiopoyetina 7 (angptl7).
JP2022535717A (ja) * 2019-05-24 2022-08-10 エンピリコ インク． アンジオポエチン様７（ａｎｇｐｔｌ７）関連疾患の処置
US11865134B2 (en) * 2021-02-26 2024-01-09 Regeneron Pharmaceuticals, Inc. Treatment of inflammation with glucocorticoids and angiopoietin-like 7 (ANGPTL7) inhibitors
WO2023031359A1 (en) * 2021-09-02 2023-03-09 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell

Also Published As

Publication number Publication date
US20240409943A1 (en) 2024-12-12
EP4408438A4 (en) 2026-04-01
EP4408438A1 (en) 2024-08-07
WO2023056478A1 (en) 2023-04-06
WO2023056478A8 (en) 2024-04-04

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