AU2002223402A1 - A novel gene therapy agent for haemophilia b and its preparation method - Google Patents

A novel gene therapy agent for haemophilia b and its preparation method

Info

Publication number
AU2002223402A1
AU2002223402A1 AU2002223402A AU2340202A AU2002223402A1 AU 2002223402 A1 AU2002223402 A1 AU 2002223402A1 AU 2002223402 A AU2002223402 A AU 2002223402A AU 2340202 A AU2340202 A AU 2340202A AU 2002223402 A1 AU2002223402 A1 AU 2002223402A1
Authority
AU
Australia
Prior art keywords
gene
preparation
therapy
haemophilia
gene therapy
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.)
Abandoned
Application number
AU2002223402A
Inventor
Jiahui Xia
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.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of AU2002223402A1 publication Critical patent/AU2002223402A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

The invention deals with the gene drugs for therapy Hemophilia B and its method of preparation. It contains human source gene vector-FIX recombinant constructed using DNA sequence as leading sequence of therapy gene without important physiological function-related gene on the short arms of human group D,G chromosomes or to which DNA sequence is homologous. This invention also provides the method of preparing gene drug. The therapy gene of gene drug for hemophilia B has a high stability of expression, safety, high expression efficiency and no immunogenecity.
AU2002223402A 2000-08-30 2001-08-29 A novel gene therapy agent for haemophilia b and its preparation method Abandoned AU2002223402A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CN00113652 2000-08-30
CN00113652 2000-08-30
CNB011028300A CN1148228C (en) 2000-08-30 2001-01-19 Gene medicine for treating hemophilia B and its preparing process
CN01102830 2001-01-19
PCT/CN2001/001291 WO2002034296A1 (en) 2000-08-30 2001-08-29 A novel gene therapy agent for haemophilia b and its preparation method

Publications (1)

Publication Number Publication Date
AU2002223402A1 true AU2002223402A1 (en) 2002-05-06

Family

ID=25739400

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2002223402A Abandoned AU2002223402A1 (en) 2000-08-30 2001-08-29 A novel gene therapy agent for haemophilia b and its preparation method

Country Status (8)

Country Link
US (1) US7361639B2 (en)
EP (1) EP1316319B1 (en)
JP (1) JP2004512313A (en)
CN (1) CN1148228C (en)
AT (1) ATE453401T1 (en)
AU (1) AU2002223402A1 (en)
DE (1) DE60140950D1 (en)
WO (1) WO2002034296A1 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002020802A1 (en) * 2000-09-04 2002-03-14 Hunan Royal Biotech A cell line expressing mutant human tissue-type plasminogen activator, constructing strategy and method of expressed protein preparation
CN1880458B (en) * 2005-06-15 2010-04-28 中国医学科学院基础医学研究所 Human source chromosome targeting vector of human beta-globin gene cluster and application therefor
US8980382B2 (en) * 2009-12-02 2015-03-17 Applied Materials, Inc. Oxygen-doping for non-carbon radical-component CVD films
KR20220100728A (en) 2014-03-21 2022-07-15 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Genome editing without nucleases
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN110945127A (en) 2017-05-22 2020-03-31 百深公司 Viral vectors encoding recombinant FIX with increased expression for gene therapy of hemophilia B
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08511423A (en) * 1993-06-10 1996-12-03 ジェネティック セラピー，インコーポレイテッド Adenovirus vector for treatment of hemophilia
AU6468199A (en) * 1998-09-23 2000-04-10 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Method to treat haemophilia by (in vivo) gene therapy with retroviral vectors

Also Published As

Publication number Publication date
WO2002034296A1 (en) 2002-05-02
EP1316319B1 (en) 2009-12-30
US7361639B2 (en) 2008-04-22
EP1316319A4 (en) 2008-08-06
US20040038919A1 (en) 2004-02-26
ATE453401T1 (en) 2010-01-15
DE60140950D1 (en) 2010-02-11
CN1148228C (en) 2004-05-05
CN1302664A (en) 2001-07-11
JP2004512313A (en) 2004-04-22
EP1316319A1 (en) 2003-06-04

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