WO2013093760A3 - Compositions, procédés et trousses pour la préparation de protéines recombinantes sialylées - Google Patents

Compositions, procédés et trousses pour la préparation de protéines recombinantes sialylées Download PDF

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Publication number
WO2013093760A3
WO2013093760A3 PCT/IB2012/057392 IB2012057392W WO2013093760A3 WO 2013093760 A3 WO2013093760 A3 WO 2013093760A3 IB 2012057392 W IB2012057392 W IB 2012057392W WO 2013093760 A3 WO2013093760 A3 WO 2013093760A3
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WO
WIPO (PCT)
Prior art keywords
recombinant proteins
kits
compositions
preparing
methods
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.)
Ceased
Application number
PCT/IB2012/057392
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English (en)
Other versions
WO2013093760A2 (fr
Inventor
Thomas Barnett
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.)
Grifols SA
Original Assignee
Grifols SA
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Filing date
Publication date
Application filed by Grifols SA filed Critical Grifols SA
Priority to AU2012340501A priority Critical patent/AU2012340501A1/en
Publication of WO2013093760A2 publication Critical patent/WO2013093760A2/fr
Publication of WO2013093760A3 publication Critical patent/WO2013093760A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • 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
    • A61K38/45—Transferases (2)
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1081—Glycosyltransferases (2.4) transferring other glycosyl groups (2.4.99)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/99—Glycosyltransferases (2.4) transferring other glycosyl groups (2.4.99)
    • C12Y204/99004—Beta-galactoside alpha-2,3-sialyltransferase (2.4.99.4)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Hematology (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

L'invention concerne un procédé de modification d'un motif de sialylation d'une ou plusieurs protéines recombinantes exprimées dans une cellule, la cellule étant génétiquement modifiée pour exprimer de façon recombinante une α2,3-sialyltransférase pour sialyler la protéine. Ce système d'expression augmente la sialylation de la protéine qui est exprimée, en particulier la quantité d'un acide sialique lié en α-2,3, fixé à la protéine.
PCT/IB2012/057392 2011-12-19 2012-12-17 Compositions, procédés et trousses pour la préparation de protéines recombinantes sialylées Ceased WO2013093760A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2012340501A AU2012340501A1 (en) 2011-12-19 2012-12-17 Compositions, methods, and kits for preparing sialylated recombinant proteins

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161577291P 2011-12-19 2011-12-19
US61/577,291 2011-12-19

Publications (2)

Publication Number Publication Date
WO2013093760A2 WO2013093760A2 (fr) 2013-06-27
WO2013093760A3 true WO2013093760A3 (fr) 2013-11-14

Family

ID=47681979

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2012/057392 Ceased WO2013093760A2 (fr) 2011-12-19 2012-12-17 Compositions, procédés et trousses pour la préparation de protéines recombinantes sialylées

Country Status (4)

Country Link
AR (1) AR089324A1 (fr)
AU (1) AU2012340501A1 (fr)
UY (1) UY34527A (fr)
WO (1) WO2013093760A2 (fr)

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WO2014127215A1 (fr) * 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Gène du facteur viii optimisé
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
US20160130324A1 (en) 2014-10-31 2016-05-12 Shire Human Genetic Therapies, Inc. C1 Inhibitor Fusion Proteins and Uses Thereof
EP3042952A1 (fr) 2015-01-07 2016-07-13 CEVEC Pharmaceuticals GmbH Glycoprotéines recombinantes sialylées o-glycanes et lignées cellulaires de production de celles-ci
JP6651548B2 (ja) 2015-05-22 2020-02-19 ツェー・エス・エル・ベーリング・レングナウ・アクチエンゲゼルシャフト 改変フォン・ヴィルブランド因子を製造するための方法
EP3297656B1 (fr) 2015-05-22 2020-01-08 CSL Behring Lengnau AG Polypeptides du facteur von willebrand tronqué pour traiter l'hémophilie
CN108463243B (zh) 2015-11-19 2022-06-14 夏尔人类遗传性治疗公司 重组人c1酯酶抑制剂及其用途
DK3400002T3 (da) * 2016-01-07 2022-04-11 CSL Behring Lengnau AG Muteret, trunkeret von willebrand faktor
CA3012695A1 (fr) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Genes du facteur viii optimises
US11814421B2 (en) 2016-11-11 2023-11-14 CSL Behring Lengnau AG Truncated von Willebrand Factor polypeptides for treating hemophilia
SG11201903954WA (en) 2016-11-11 2019-05-30 CSL Behring Lengnau AG Truncated von willebrand factor polypeptides for extravascular administration in the treatment or prophylaxis of a blood coagulation disorder
EP3382014A1 (fr) * 2017-03-29 2018-10-03 CEVEC Pharmaceuticals GmbH Glycoprotéines recombinées avec une fucosylation antennulaire réduite
ES2966835T3 (es) 2017-06-22 2024-04-24 CSL Behring Lengnau AG Modulación de la inmunogenicidad del FVIII por VWF truncado
EP3441471A1 (fr) 2017-08-08 2019-02-13 CEVEC Pharmaceuticals GmbH Utilisation de variants du récepteur du facteur de croissance constitutivement actifs comme marqueurs de sélection pour générer des cellules de production stabiles
SG11202109850SA (en) * 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
WO2021001522A1 (fr) 2019-07-04 2021-01-07 CSL Behring Lengnau AG Facteur de von willebrand (vwf) tronqué pour augmenter la stabilité in vitro du facteur viii de coagulation
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
US20220380785A1 (en) * 2019-11-01 2022-12-01 The University Of British Columbia Compositions and methods for sialylated mucin-type o-glycosylation of therapeutic proteins
US20220348637A1 (en) 2019-11-11 2022-11-03 CSL Behring Lengnau AG Polypeptides for inducing tolerance to factor viii
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.

Citations (4)

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Publication number Priority date Publication date Assignee Title
US20060099685A1 (en) * 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
WO2006070011A1 (fr) * 2004-12-30 2006-07-06 Crucell Holland B.V. Procedes d'obtention de proteines recombinantes a sialylation accrue a partir de cellules qui expriment une proteine adenovirale e1a et proteines obtenues de cette maniere
WO2009127826A1 (fr) * 2008-04-16 2009-10-22 Ferring International Center Sa Fsh recombinante comprenant la sialylation alpha 2,3 et alpha 2,6
WO2011042688A1 (fr) * 2009-10-05 2011-04-14 Ferring International Center Sa Préparation pharmaceutique comprenant une hcg recombinante

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WO2006070011A1 (fr) * 2004-12-30 2006-07-06 Crucell Holland B.V. Procedes d'obtention de proteines recombinantes a sialylation accrue a partir de cellules qui expriment une proteine adenovirale e1a et proteines obtenues de cette maniere
WO2009127826A1 (fr) * 2008-04-16 2009-10-22 Ferring International Center Sa Fsh recombinante comprenant la sialylation alpha 2,3 et alpha 2,6
WO2011042688A1 (fr) * 2009-10-05 2011-04-14 Ferring International Center Sa Préparation pharmaceutique comprenant une hcg recombinante

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Also Published As

Publication number Publication date
AR089324A1 (es) 2014-08-13
AU2012340501A1 (en) 2013-07-11
UY34527A (es) 2013-07-31
WO2013093760A2 (fr) 2013-06-27

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