CA3034566A1 - Compositions and methods for modulating factor ix function - Google Patents

Compositions and methods for modulating factor ix function Download PDF

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Publication number
CA3034566A1
CA3034566A1 CA3034566A CA3034566A CA3034566A1 CA 3034566 A1 CA3034566 A1 CA 3034566A1 CA 3034566 A CA3034566 A CA 3034566A CA 3034566 A CA3034566 A CA 3034566A CA 3034566 A1 CA3034566 A1 CA 3034566A1
Authority
CA
Canada
Prior art keywords
factor
variant
substitution
nucleic acid
substituted
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3034566A
Other languages
English (en)
French (fr)
Inventor
Valder R. Arruda
Rodney M. Camire
Benjamin SAMELSON-JONES
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Children's Hospital of Philadelphia
Original Assignee
Children's Hospital of Philadelphia
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Children's Hospital of Philadelphia filed Critical Children's Hospital of Philadelphia
Publication of CA3034566A1 publication Critical patent/CA3034566A1/en
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

Landscapes

  • 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)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • 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)
CA3034566A 2016-07-27 2017-07-27 Compositions and methods for modulating factor ix function Pending CA3034566A1 (en)

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
CA3034566A1 true CA3034566A1 (en) 2018-02-01

Family

ID=61016739

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3034566A Pending CA3034566A1 (en) 2016-07-27 2017-07-27 Compositions and methods for modulating factor ix function

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
ES3062513T3 (en) * 2019-03-19 2026-04-13 CSL Innovation Pty Ltd Factor ix variants and uses thereof in therapy

Family Cites Families (8)

* 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
EP2423307A1 (en) 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IV polypeptides and use thereof for treatment
US7968683B1 (en) * 2008-05-07 2011-06-28 Schering Corporation Factor IXa crystals, related complexes and methods
EA201290069A1 (ru) 2009-07-31 2012-07-30 Байер Хелскер Ллс Модифицированные полипептиды фактора ix и их применения
TWI595004B (zh) * 2010-11-03 2017-08-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
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

Also Published As

Publication number Publication date
EP4566666A3 (en) 2025-09-17
EP4566666A2 (en) 2025-06-11
JP2019525764A (ja) 2019-09-12
EP3490586B1 (en) 2025-03-26
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
JP7181855B2 (ja) 2022-12-01
US20200248161A1 (en) 2020-08-06
EP3490586A4 (en) 2020-05-20

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