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
less
oligomeric compound
administering
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JP2023519501A (ja
JP2023519501A5 (https=
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
    • 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
    • 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
    • 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
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • C12N2310/34—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
    • 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
    • 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)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (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)

Publication Number Publication Date
JP2023519501A JP2023519501A (ja) 2023-05-11
JP2023519501A5 JP2023519501A5 (https=) 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 (9)

Country Link
US (1) US20230149438A1 (https=)
EP (1) EP4118208A4 (https=)
JP (2) JP7795467B2 (https=)
KR (1) KR20220152569A (https=)
CN (1) CN115244179A (https=)
BR (1) BR112022016167A2 (https=)
IL (1) IL295631A (https=)
MX (1) MX2022011205A (https=)
WO (1) WO2021183857A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102365486B1 (ko) 2013-08-28 2022-02-18 아이오니스 파마수티컬즈, 인코포레이티드 프리칼리크레인 (pkk) 발현의 조절

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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102365486B1 (ko) * 2013-08-28 2022-02-18 아이오니스 파마수티컬즈, 인코포레이티드 프리칼리크레인 (pkk) 발현의 조절
ES2982752T3 (es) * 2015-10-22 2024-10-17 Iltoo Pharma Composiciones farmacéuticas de il-2
PL4119569T3 (pl) * 2015-11-06 2024-11-18 Ionis Pharmaceuticals, Inc. Sprzężone związki antysensowne do zastosowania w terapii
TW201828952A (zh) * 2017-02-08 2018-08-16 美商Ionis製藥公司 調節血管舒緩素b1(klkb1)用於治療頭痛

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

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