JP7795467B2 - プレカリクレイン関連の状態を治療及び予防するための組成物及び方法 - Google Patents

プレカリクレイン関連の状態を治療及び予防するための組成物及び方法

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Publication number
JP7795467B2
JP7795467B2 JP2022552307A JP2022552307A JP7795467B2 JP 7795467 B2 JP7795467 B2 JP 7795467B2 JP 2022552307 A JP2022552307 A JP 2022552307A JP 2022552307 A JP2022552307 A JP 2022552307A JP 7795467 B2 JP7795467 B2 JP 7795467B2
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weeks
once
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oligomeric compound
administering
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JP2023519501A5 (enExample
JP2023519501A (ja
Inventor
シュナイダー，ユージン
バイニー，ニコラス・ジェイ
アレクサンダー，ベロニカ・ジェイ
ボルドーネ，ローラ
ニューマン，ケネス
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/1137—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 enzymes
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21034—Plasma kallikrein (3.4.21.34)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (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)
  • Peptides Or Proteins (AREA)
JP2022552307A 2020-03-13 2021-03-12 プレカリクレイン関連の状態を治療及び予防するための組成物及び方法 Active JP7795467B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025266910A JP2026048915A (ja) 2020-03-13 2025-12-19 プレカリクレイン関連の状態を治療及び予防するための組成物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062989427P 2020-03-13 2020-03-13
US62/989,427 2020-03-13
PCT/US2021/022069 WO2021183857A1 (en) 2020-03-13 2021-03-12 Compositions and methods for treating and preventing prekallikrein-associated conditions

Related Child Applications (1)

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JP2025266910A Division JP2026048915A (ja) 2020-03-13 2025-12-19 プレカリクレイン関連の状態を治療及び予防するための組成物及び方法

Publications (3)

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JP2023519501A JP2023519501A (ja) 2023-05-11
JP2023519501A5 JP2023519501A5 (enExample) 2024-03-26
JP7795467B2 true JP7795467B2 (ja) 2026-01-07

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JP2022552307A Active JP7795467B2 (ja) 2020-03-13 2021-03-12 プレカリクレイン関連の状態を治療及び予防するための組成物及び方法
JP2025266910A Pending JP2026048915A (ja) 2020-03-13 2025-12-19 プレカリクレイン関連の状態を治療及び予防するための組成物及び方法

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JP2025266910A Pending JP2026048915A (ja) 2020-03-13 2025-12-19 プレカリクレイン関連の状態を治療及び予防するための組成物及び方法

Country Status (10)

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US (2) US20230149438A1 (enExample)
EP (1) EP4118208A4 (enExample)
JP (2) JP7795467B2 (enExample)
KR (1) KR20220152569A (enExample)
CN (1) CN115244179A (enExample)
AU (2) AU2021233914B2 (enExample)
BR (1) BR112022016167A2 (enExample)
IL (1) IL295631B1 (enExample)
MX (1) MX2022011205A (enExample)
WO (1) WO2021183857A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9670492B2 (en) 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017518045A (ja) 2014-05-01 2017-07-06 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｐｋｋ発現を調節するための組成物及び方法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9670492B2 (en) * 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
ES2982752T3 (es) * 2015-10-22 2024-10-17 Iltoo Pharma Composiciones farmacéuticas de il-2
LT4119569T (lt) * 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje
US11286307B2 (en) * 2015-12-11 2022-03-29 Takeda Pharmaceutical Company Limited Plasma kallikrein inhibitors and uses thereof for treating hereditary angioedema attack
WO2018148449A1 (en) * 2017-02-08 2018-08-16 Ionis Pharmaceuticals, Inc. Modulation of kallikrein b1 (klkb1) for treatment of headache

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017518045A (ja) 2014-05-01 2017-07-06 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｐｋｋ発現を調節するための組成物及び方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
A Study to Assess the Clinical Efficacy of Donidalorsen in Participants With Hereditary Angioedema，ClinicalTrials.gov，2020年02月18日
Jason D. Ferrone et al.，Nucleic Acid Therapeutics，2019年，Vol.29, No.2，p.82-91

Also Published As

Publication number Publication date
BR112022016167A2 (pt) 2022-10-04
IL295631B1 (en) 2026-08-01
AU2026203664A1 (en) 2026-05-28
WO2021183857A1 (en) 2021-09-16
EP4118208A1 (en) 2023-01-18
AU2021233914A1 (en) 2022-09-08
JP2026048915A (ja) 2026-03-17
MX2022011205A (es) 2022-09-19
US20230149438A1 (en) 2023-05-18
IL295631A (en) 2022-10-01
AU2021233914B2 (en) 2026-04-30
US20260158051A1 (en) 2026-06-11
EP4118208A4 (en) 2025-03-12
KR20220152569A (ko) 2022-11-16
JP2023519501A (ja) 2023-05-11
CN115244179A (zh) 2022-10-25

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