KR101768896B1 - 그의 보조인자의 부존재하에서 응고활성을 갖는 인자 ix 변이체 및 출혈 질환을 치료하기 위한 그의 용도 - Google Patents

그의 보조인자의 부존재하에서 응고활성을 갖는 인자 ix 변이체 및 출혈 질환을 치료하기 위한 그의 용도 Download PDF

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KR101768896B1
KR101768896B1 KR1020117001765A KR20117001765A KR101768896B1 KR 101768896 B1 KR101768896 B1 KR 101768896B1 KR 1020117001765 A KR1020117001765 A KR 1020117001765A KR 20117001765 A KR20117001765 A KR 20117001765A KR 101768896 B1 KR101768896 B1 KR 101768896B1
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bleeding
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에르하르트 세이프리이드
조르그 슈트럼프
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디알케이-블루트스펜데딘스트 바덴-뷔르템버그-헤센 지지엠비에이치
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21022Coagulation factor IXa (3.4.21.22)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/948Hydrolases (3) acting on peptide bonds (3.4)
    • G01N2333/95Proteinases, i.e. endopeptidases (3.4.21-3.4.99)
    • G01N2333/964Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue
    • G01N2333/96425Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals
    • G01N2333/96427Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general
    • G01N2333/9643Proteinases, i.e. endopeptidases (3.4.21-3.4.99) derived from animal tissue from mammals in general with EC number
    • G01N2333/96433Serine endopeptidases (3.4.21)
    • G01N2333/96441Serine endopeptidases (3.4.21) with definite EC number
    • G01N2333/9645Factor IX (3.4.21.22)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/22Haematology
    • G01N2800/224Haemostasis or coagulation

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020117001765A 2008-07-28 2009-07-28 그의 보조인자의 부존재하에서 응고활성을 갖는 인자 ix 변이체 및 출혈 질환을 치료하기 위한 그의 용도 Active KR101768896B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP08013561A EP2149603A1 (en) 2008-07-28 2008-07-28 Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders
EP08013561.9 2008-07-28
PCT/EP2009/005465 WO2010012451A1 (en) 2008-07-28 2009-07-28 Factor ix variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders

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KR1020177022245A Division KR101947424B1 (ko) 2008-07-28 2009-07-28 그의 보조인자의 부존재하에서 응고활성을 갖는 인자 ix 변이체 및 출혈 질환을 치료하기 위한 그의 용도

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KR20110036071A KR20110036071A (ko) 2011-04-06
KR101768896B1 true KR101768896B1 (ko) 2017-08-17

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KR1020177022245A Active KR101947424B1 (ko) 2008-07-28 2009-07-28 그의 보조인자의 부존재하에서 응고활성을 갖는 인자 ix 변이체 및 출혈 질환을 치료하기 위한 그의 용도

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US (2) US10125357B2 (enExample)
EP (6) EP2149603A1 (enExample)
JP (1) JP6029826B2 (enExample)
KR (2) KR101768896B1 (enExample)
CN (1) CN102112608B (enExample)
AU (1) AU2009275391B2 (enExample)
CA (2) CA2729501C (enExample)
ES (3) ES2753638T3 (enExample)
WO (1) WO2010012451A1 (enExample)

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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
CA2828769A1 (en) 2011-03-02 2012-09-07 Novo Nordisk A/S Coagulation factor-targeting to tlt-1 on activated platelets
US10656167B2 (en) 2011-07-25 2020-05-19 Bioverativ Therapeutics Inc. Assays to monitor bleeding disorders
CN103173482A (zh) * 2011-12-22 2013-06-26 武汉科技大学 野生型和突变体hFⅨ毕赤酵母表达载体及构建方法和应用
EP2666782A1 (en) * 2012-05-22 2013-11-27 Imnate Sarl Coagulation factor VIII with reduced immunogenicity.
WO2014070349A1 (en) * 2012-10-29 2014-05-08 Regents Of The University Of Minnesota Factor ix variants
CN103060366A (zh) * 2012-12-25 2013-04-24 武汉科技大学 人凝血因子ⅸ突变体毕赤酵母表达载体及构建方法和应用
DK3889173T5 (da) 2013-02-15 2024-08-05 Bioverativ Therapeutics Inc Optimeret faktor viii-gen
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EP2881463A1 (en) 2013-12-09 2015-06-10 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
EP3325010B1 (en) 2015-07-23 2023-06-21 The Regents of The University of California Antibodies to coagulation factor xia and uses thereof
EP3411478B1 (en) 2016-02-01 2022-06-08 Bioverativ Therapeutics Inc. Optimized factor viii genes
PL3652326T3 (pl) 2017-07-10 2025-02-24 Uniqure Ip B.V. Środki i sposoby terapii genowej aav u ludzi
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
GB201817810D0 (en) * 2018-10-31 2018-12-19 Ucl Business Plc Modified factor IX polypeptides
WO2020104480A1 (en) * 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
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
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
KR20230074703A (ko) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
US20230149563A1 (en) 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
US20250242056A1 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response

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EP2767587A3 (en) 2014-11-26
US10883097B2 (en) 2021-01-05
WO2010012451A1 (en) 2010-02-04
JP2011528913A (ja) 2011-12-01
EP3241899B1 (en) 2019-08-21
EP2149603A1 (en) 2010-02-03
AU2009275391A1 (en) 2010-02-04
CN102112608B (zh) 2013-11-20
CN102112608A (zh) 2011-06-29
KR20110036071A (ko) 2011-04-06
JP6029826B2 (ja) 2016-11-24
CA3033578A1 (en) 2010-02-04
EP2767587A2 (en) 2014-08-20
EP2318526A1 (en) 2011-05-11
KR101947424B1 (ko) 2019-02-13
US20190017039A1 (en) 2019-01-17
CA2729501C (en) 2019-04-02
KR20170096210A (ko) 2017-08-23
CA2729501A1 (en) 2010-02-04
CA3033578C (en) 2022-07-26
AU2009275391B2 (en) 2015-01-22
EP3623470A2 (en) 2020-03-18
ES2753638T3 (es) 2020-04-13
ES2441167T3 (es) 2014-02-03
US20110217284A1 (en) 2011-09-08
EP3241899A1 (en) 2017-11-08
EP2390323B1 (en) 2013-11-27
EP2767587B1 (en) 2017-04-05
EP2390323A2 (en) 2011-11-30
ES2628688T3 (es) 2017-08-03
US10125357B2 (en) 2018-11-13
EP3623470A3 (en) 2020-07-22
EP2390323A3 (en) 2012-01-11

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