JP2024506351A - Ｐｌｎの発現を低減するための化合物及び方法 - Google Patents

Ｐｌｎの発現を低減するための化合物及び方法 Download PDF

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Publication number
JP2024506351A
JP2024506351A JP2023548623A JP2023548623A JP2024506351A JP 2024506351 A JP2024506351 A JP 2024506351A JP 2023548623 A JP2023548623 A JP 2023548623A JP 2023548623 A JP2023548623 A JP 2023548623A JP 2024506351 A JP2024506351 A JP 2024506351A
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modified
modified oligonucleotide
oligomeric
nucleobase
sugar moiety
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JP2023548623A
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Japanese (ja)
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JP2024506351A5 (enExample
Inventor
ムリック，アダム
ブイ，フィン・ホア
フレアー，スーザン・エム
イエ，ティン・ユアン
クブリ，ディーター
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of JP2024506351A publication Critical patent/JP2024506351A/ja
Publication of JP2024506351A5 publication Critical patent/JP2024506351A5/ja
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
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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
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70582—CD71
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    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
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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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
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    • A01K2207/00—Modified animals
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
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    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
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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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12Y301/26—Endoribonucleases producing 5'-phosphomonoesters (3.1.26)
    • C12Y301/26004—Ribonuclease H (3.1.26.4)

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Environmental Sciences (AREA)
  • Immunology (AREA)
  • Heart & Thoracic Surgery (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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JP2023548623A 2021-02-11 2022-02-10 Ｐｌｎの発現を低減するための化合物及び方法 Pending JP2024506351A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163148579P 2021-02-11 2021-02-11
US63/148,579 2021-02-11
PCT/US2022/016015 WO2022173976A1 (en) 2021-02-11 2022-02-10 Compounds and methods for reducing pln expression

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JP2024506351A true JP2024506351A (ja) 2024-02-13
JP2024506351A5 JP2024506351A5 (enExample) 2025-02-13

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US (1) US20240191232A1 (enExample)
EP (1) EP4291651A4 (enExample)
JP (1) JP2024506351A (enExample)
KR (1) KR20230145133A (enExample)
CN (1) CN117597442A (enExample)
AU (1) AU2022219976A1 (enExample)
BR (1) BR112023015160A2 (enExample)
CA (1) CA3210172A1 (enExample)
IL (1) IL304775A (enExample)
MX (1) MX2023009429A (enExample)
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Publication number Priority date Publication date Assignee Title
EP4689119A1 (en) * 2023-04-05 2026-02-11 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing pln expression
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
TW202528539A (zh) * 2023-09-20 2025-07-16 日商第一三共股份有限公司 抑制受磷蛋白之表現的siRNA

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002528512A (ja) * 1998-11-02 2002-09-03 ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア 心臓病及び心不全の治療のためのホスホランバン活性の阻害方法
JP2004512810A (ja) * 1999-08-31 2004-04-30 サーナ・セラピューティクス・インコーポレイテッド 核酸に基づく遺伝子発現の調節剤
WO2010135322A1 (en) * 2009-05-19 2010-11-25 Medtronic, Inc. Methods and devices for improved efficiency of rna delivery to cells
WO2018154439A1 (en) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030050259A1 (en) * 1999-12-06 2003-03-13 Lawrence Blatt Method and reagent for the treatment of cardiac disease
AU2005323437B2 (en) * 2004-04-30 2011-10-06 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a C5-modified pyrimidine
US7491813B2 (en) * 2005-12-07 2009-02-17 Monsanto Technology Llc Promoter polynucleotides identified from Zea mays for use in plants
US20100298697A1 (en) * 2009-05-19 2010-11-25 Medtronic, Inc. Method and devices for improved efficiency of rna delivery to cells
CA2954791C (en) * 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002528512A (ja) * 1998-11-02 2002-09-03 ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア 心臓病及び心不全の治療のためのホスホランバン活性の阻害方法
JP2004512810A (ja) * 1999-08-31 2004-04-30 サーナ・セラピューティクス・インコーポレイテッド 核酸に基づく遺伝子発現の調節剤
WO2010135322A1 (en) * 2009-05-19 2010-11-25 Medtronic, Inc. Methods and devices for improved efficiency of rna delivery to cells
WO2018154439A1 (en) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
KAIROUZ VICTOR ET AL.: "Molecular targets in heart failure gene therapy: current controversies and translational perspective", ANN. N.Y. ACAD. SCI., vol. 1254, JPN6025050087, 2012, pages 42 - 50, ISSN: 0005746911 *
MORIHARA HIROFUMI ET AL.: "Phospholamban Inhibition by a Single Dose of Locked Nucleic Acid Antisense Oligonucleotide Improves", JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS, vol. 22, no. 3, JPN6025050086, 2017, pages 273 - 282, ISSN: 0005746910 *

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IL304775A (en) 2023-09-01
KR20230145133A (ko) 2023-10-17
BR112023015160A2 (pt) 2023-10-03
EP4291651A4 (en) 2025-07-02
CA3210172A1 (en) 2022-08-18
WO2022173976A1 (en) 2022-08-18
EP4291651A1 (en) 2023-12-20
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US20240191232A1 (en) 2024-06-13
AU2022219976A1 (en) 2023-08-10
MX2023009429A (es) 2023-09-27

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