BR112021017062A2 - Variantes do fator ix e usos das mesmas em terapia - Google Patents

Variantes do fator ix e usos das mesmas em terapia

Info

Publication number
BR112021017062A2
BR112021017062A2 BR112021017062A BR112021017062A BR112021017062A2 BR 112021017062 A2 BR112021017062 A2 BR 112021017062A2 BR 112021017062 A BR112021017062 A BR 112021017062A BR 112021017062 A BR112021017062 A BR 112021017062A BR 112021017062 A2 BR112021017062 A2 BR 112021017062A2
Authority
BR
Brazil
Prior art keywords
variants
factor
therapy
nucleic acids
acids encoding
Prior art date
Application number
BR112021017062A
Other languages
English (en)
Inventor
Holger Lind
Marco Hofmann
Philipp Claar
Thomas Weimer
Walid Azar
Original Assignee
CSL Behring Lengnau AG
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 CSL Behring Lengnau AG filed Critical CSL Behring Lengnau AG
Publication of BR112021017062A2 publication Critical patent/BR112021017062A2/pt

Links

Classifications

    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

variantes do fator ix e usos das mesmas em terapia. a presente invenção refere-se ao fornecimento de variantes do fator ix, moléculas compreendendo as variantes, ácidos nucleicos codificando as variantes, composições compreendendo as variantes, ou os ácidos nucleicos codificando as variantes, e seus usos em métodos para a modulação de hemostasia, por exemplo, na profilaxia ou tratamento de hemofilia b. as variantes do fator ix melhoraram as propriedades biológicas relativas às outras variantes do fator ix, e/ou relativas ao fator ix do tipo selvagem.
BR112021017062A 2019-03-19 2020-03-18 Variantes do fator ix e usos das mesmas em terapia BR112021017062A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP19163619 2019-03-19
PCT/EP2020/057400 WO2020187969A1 (en) 2019-03-19 2020-03-18 Factor ix variants and uses thereof in therapy

Publications (1)

Publication Number Publication Date
BR112021017062A2 true BR112021017062A2 (pt) 2021-11-16

Family

ID=65818428

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021017062A BR112021017062A2 (pt) 2019-03-19 2020-03-18 Variantes do fator ix e usos das mesmas em terapia

Country Status (9)

Country Link
US (1) US20220154161A1 (pt)
EP (1) EP3941509A1 (pt)
JP (1) JP2022525660A (pt)
KR (1) KR20210141608A (pt)
CN (2) CN113573726A (pt)
AU (1) AU2020242945A1 (pt)
BR (1) BR112021017062A2 (pt)
CA (1) CA3131648A1 (pt)
WO (1) WO2020187969A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023275024A1 (en) 2021-07-01 2023-01-05 CSL Behring Lengnau AG Factor ix subcutaneous administration with enhanced safety
WO2023077012A1 (en) * 2021-10-27 2023-05-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI353991B (en) 2003-05-06 2011-12-11 Syntonix Pharmaceuticals Inc Immunoglobulin chimeric monomer-dimer hybrids
WO2005024044A2 (en) 2003-09-05 2005-03-17 Gtc Biotherapeutics, Inc. Method for the production of fusion proteins in transgenic mammal milk
EP1694315A4 (en) 2003-12-03 2009-10-28 Novo Nordisk As GLYCOPEGYLATED FACTOR IX
EP2975135A1 (en) 2005-05-25 2016-01-20 Novo Nordisk A/S Glycopegylated factor IX
US8476234B2 (en) 2006-02-03 2013-07-02 Prolor Biotech Inc. Long-acting coagulation factors and methods of producing same
EP2032607B2 (en) 2006-06-14 2017-02-22 CSL Behring GmbH Proteolytically cleavable fusion protein comprising a blood coagulation factor
US8383388B2 (en) * 2006-06-19 2013-02-26 Catalyst Biosciences, Inc. Modified coagulation factor IX polypeptides and use thereof for treatment
EP2149603A1 (en) * 2008-07-28 2010-02-03 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and their use for treating bleeding disorders
US9670475B2 (en) 2010-07-09 2017-06-06 Bioverativ Therapeutics Inc. Factor IX polypeptides and methods of use thereof
AU2013204511B2 (en) * 2010-11-03 2016-03-17 Gc Biopharma Corp. Modified factor ix polypeptides and uses thereof
TWI557135B (zh) * 2010-11-03 2016-11-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
CN117106095A (zh) 2014-01-10 2023-11-24 比奥贝拉蒂治疗公司 因子viii嵌合蛋白及其用途
US10745680B2 (en) 2015-08-03 2020-08-18 Bioverativ Therapeutics Inc. Factor IX fusion proteins and methods of making and using same
US11708570B2 (en) * 2016-07-27 2023-07-25 The Children's Hospital Of Philadelphia Compositions and methods for modulating Factor IX function
AU2017358861B2 (en) * 2016-11-11 2022-02-17 CSL Behring Lengnau AG Truncated von Willebrand Factor polypeptides for treating hemophilia
KR102386890B1 (ko) * 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
FR3069540B1 (fr) * 2017-07-28 2019-09-13 Universite Claude Bernard Lyon 1 Proteine modifiee avec demi-vie amelioree

Also Published As

Publication number Publication date
US20220154161A1 (en) 2022-05-19
CN117603949A (zh) 2024-02-27
AU2020242945A1 (en) 2021-10-14
KR20210141608A (ko) 2021-11-23
CN113573726A (zh) 2021-10-29
CA3131648A1 (en) 2020-09-24
JP2022525660A (ja) 2022-05-18
WO2020187969A1 (en) 2020-09-24
EP3941509A1 (en) 2022-01-26

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Legal Events

Date Code Title Description
B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]