CN109789181A - 用于调节因子ix功能的组合物和方法 - Google Patents

用于调节因子ix功能的组合物和方法 Download PDF

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Publication number
CN109789181A
CN109789181A CN201780059441.0A CN201780059441A CN109789181A CN 109789181 A CN109789181 A CN 109789181A CN 201780059441 A CN201780059441 A CN 201780059441A CN 109789181 A CN109789181 A CN 109789181A
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CN
China
Prior art keywords
variant
factor
substitution
nucleic acid
arg
Prior art date
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Pending
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CN201780059441.0A
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English (en)
Chinese (zh)
Inventor
瓦尔德·R·阿鲁达
罗德尼·M·卡米尔
本杰明·萨梅尔森-琼斯
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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Publication date
Application filed by Childrens Hospital of Philadelphia CHOP filed Critical Childrens Hospital of Philadelphia CHOP
Publication of CN109789181A publication Critical patent/CN109789181A/zh
Pending legal-status Critical Current

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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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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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)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Diabetes (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN201780059441.0A 2016-07-27 2017-07-27 用于调节因子ix功能的组合物和方法 Pending CN109789181A (zh)

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

Publications (1)

Publication Number Publication Date
CN109789181A true CN109789181A (zh) 2019-05-21

Family

ID=61016739

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780059441.0A Pending CN109789181A (zh) 2016-07-27 2017-07-27 用于调节因子ix功能的组合物和方法

Country Status (6)

Country Link
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
CN117603949A (zh) * 2019-03-19 2024-02-27 杰特创新股份有限公司 因子ix变体及其在治疗中的用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080102115A1 (en) * 2006-06-19 2008-05-01 Jorge Oyhenart Modified coagulation factor IX polypeptides and use thereof for treatment
CN103282491A (zh) * 2010-11-03 2013-09-04 催化剂生物科学公司 修饰的因子ix多肽及其用途

Family Cites Families (6)

* 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.
EP2213733A3 (en) 2006-05-24 2010-12-29 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
AU2010278721A1 (en) 2009-07-31 2012-02-16 Bayer Healthcare Llc Modified factor IX polypeptides and uses thereof
EP2760887B1 (en) 2011-09-30 2024-10-23 The Children's Hospital of Philadelphia Compositions and methods for modulating hemostasis
US10676731B2 (en) 2014-08-19 2020-06-09 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor IX function

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080102115A1 (en) * 2006-06-19 2008-05-01 Jorge Oyhenart Modified coagulation factor IX polypeptides and use thereof for treatment
CN103282491A (zh) * 2010-11-03 2013-09-04 催化剂生物科学公司 修饰的因子ix多肽及其用途

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PAUL E. MONAHAN等: "employing a gain-of-funcition Factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy:preclinical evaluation supporting an ongoing adeno-associated virus clinical trial", 《HUMAN GENE THERAPY》 *

Also Published As

Publication number Publication date
US20200248161A1 (en) 2020-08-06
WO2018022844A2 (en) 2018-02-01
EP3490586A2 (en) 2019-06-05
CA3034566A1 (en) 2018-02-01
EP4566666A3 (en) 2025-09-17
EP3490586B1 (en) 2025-03-26
JP2019525764A (ja) 2019-09-12
JP7181855B2 (ja) 2022-12-01
EP4566666A2 (en) 2025-06-11
US11708570B2 (en) 2023-07-25
WO2018022844A3 (en) 2019-04-18
EP3490586A4 (en) 2020-05-20

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