JP7181855B2 - 第ｉｘ因子の機能を調節するための組成物及び方法 - Google Patents

第ｉｘ因子の機能を調節するための組成物及び方法 Download PDF

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JP7181855B2
JP7181855B2 JP2019504782A JP2019504782A JP7181855B2 JP 7181855 B2 JP7181855 B2 JP 7181855B2 JP 2019504782 A JP2019504782 A JP 2019504782A JP 2019504782 A JP2019504782 A JP 2019504782A JP 7181855 B2 JP7181855 B2 JP 7181855B2
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factor
variant
nucleic acid
vector
certain embodiments
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JP2019525764A (ja
JP2019525764A5 (enExample
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ヴァルディア アール アルーダ
ロドニー エム カミーレ
ジョーンズ ベンジャミン サメルソン
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Children's Hospital of Philadelphia
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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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • 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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
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  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2019504782A 2016-07-27 2017-07-27 第ｉｘ因子の機能を調節するための組成物及び方法 Active JP7181855B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662367321P 2016-07-27 2016-07-27
US62/367,321 2016-07-27
PCT/US2017/044104 WO2018022844A2 (en) 2016-07-27 2017-07-27 Compositions and methods for modulating factor ix function

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JP2019525764A JP2019525764A (ja) 2019-09-12
JP2019525764A5 JP2019525764A5 (enExample) 2020-09-03
JP7181855B2 true JP7181855B2 (ja) 2022-12-01

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US (1) US11708570B2 (enExample)
EP (2) EP3490586B1 (enExample)
JP (1) JP7181855B2 (enExample)
CN (1) CN109789181A (enExample)
CA (1) CA3034566A1 (enExample)
WO (1) WO2018022844A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
ES3062513T3 (en) * 2019-03-19 2026-04-13 CSL Innovation Pty Ltd Factor ix variants and uses thereof in therapy

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013500726A (ja) 2009-07-31 2013-01-10 バイエル・ヘルスケア・エルエルシー 改変第ｉｘ因子ポリペプチドおよびその使用
US20130243855A1 (en) 2006-06-19 2013-09-19 Jorge Oyhenart Modified coagulation factor IX polypeptides and use thereof for treatment
US20160201047A1 (en) 2010-11-03 2016-07-14 Catalyst Biosciences, Inc. Modified factor ix polypeptides and uses thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX336958B (es) 2005-11-15 2016-02-05 Philadelphia Children Hospital Metodos y composiciones para modular la hemostasia.
AU2007253264A1 (en) * 2006-05-24 2007-11-29 Novo Nordisk Health Care Ag Factor IX analogues having prolonged in vivo half life
US7968683B1 (en) * 2008-05-07 2011-06-28 Schering Corporation Factor IXa crystals, related complexes and methods
JP6514893B2 (ja) 2011-09-30 2019-05-15 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 止血を調節するための組成物および方法
WO2016028872A2 (en) * 2014-08-19 2016-02-25 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor ix function

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130243855A1 (en) 2006-06-19 2013-09-19 Jorge Oyhenart Modified coagulation factor IX polypeptides and use thereof for treatment
JP2013500726A (ja) 2009-07-31 2013-01-10 バイエル・ヘルスケア・エルエルシー 改変第ｉｘ因子ポリペプチドおよびその使用
US20160201047A1 (en) 2010-11-03 2016-07-14 Catalyst Biosciences, Inc. Modified factor ix polypeptides and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Johnathan, D. et.al.，The efficacy and the risk of immunogenicity of FIX Pauda (R338L) in hemophilia B dogs treated with by AAV muscle gene therapy，Blood，2012年，Vol.120, No.23，pp.4521-4523

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Publication number Publication date
EP4566666A3 (en) 2025-09-17
EP4566666A2 (en) 2025-06-11
JP2019525764A (ja) 2019-09-12
EP3490586B1 (en) 2025-03-26
CA3034566A1 (en) 2018-02-01
US11708570B2 (en) 2023-07-25
WO2018022844A2 (en) 2018-02-01
WO2018022844A3 (en) 2019-04-18
EP3490586A2 (en) 2019-06-05
CN109789181A (zh) 2019-05-21
US20200248161A1 (en) 2020-08-06
EP3490586A4 (en) 2020-05-20

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