WO2002096927A3 - Ribozyme based treatment of female reproductive diseases - Google Patents

Ribozyme based treatment of female reproductive diseases Download PDF

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Publication number
WO2002096927A3
WO2002096927A3 PCT/US2002/017674 US0217674W WO02096927A3 WO 2002096927 A3 WO2002096927 A3 WO 2002096927A3 US 0217674 W US0217674 W US 0217674W WO 02096927 A3 WO02096927 A3 WO 02096927A3
Authority
WO
WIPO (PCT)
Prior art keywords
angiogenesis
female reproductive
nucleic acid
growth factor
acid molecules
Prior art date
Application number
PCT/US2002/017674
Other languages
French (fr)
Other versions
WO2002096927A2 (en
Inventor
Jaime Escobedo
James Mcswiggen
Pamela Pavco
Dan Stinchcomb
Jennifer Sandberg
Gilad Gordon
Original Assignee
Ribozyme Pharm Inc
Chiron Corp
Jaime Escobedo
James Mcswiggen
Pamela Pavco
Dan Stinchcomb
Jennifer Sandberg
Gilad Gordon
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
Priority claimed from US10/138,674 external-priority patent/US7034009B2/en
Application filed by Ribozyme Pharm Inc, Chiron Corp, Jaime Escobedo, James Mcswiggen, Pamela Pavco, Dan Stinchcomb, Jennifer Sandberg, Gilad Gordon filed Critical Ribozyme Pharm Inc
Priority to AU2002344237A priority Critical patent/AU2002344237B8/en
Priority to CA002448320A priority patent/CA2448320A1/en
Priority to EP02752028A priority patent/EP1390385A4/en
Priority to JP2003500106A priority patent/JP2005500025A/en
Priority to US10/287,949 priority patent/US20040102389A1/en
Priority to US10/306,747 priority patent/US20030216335A1/en
Publication of WO2002096927A2 publication Critical patent/WO2002096927A2/en
Priority to PCT/US2003/005022 priority patent/WO2003070910A2/en
Priority to AU2003216323A priority patent/AU2003216323B2/en
Priority to EP03742833A priority patent/EP1521768A4/en
Priority to GB0404898A priority patent/GB2396864B/en
Priority to GB0427955A priority patent/GB2406569B/en
Priority to JP2003569803A priority patent/JP2005517436A/en
Priority to CA002456444A priority patent/CA2456444A1/en
Publication of WO2002096927A3 publication Critical patent/WO2002096927A3/en
Priority to US10/665,951 priority patent/US20040138163A1/en
Priority to US10/664,668 priority patent/US20070203333A1/en
Priority to US10/670,011 priority patent/US20040209832A1/en
Priority to US10/683,990 priority patent/US20040198682A1/en
Priority to US10/712,633 priority patent/US20040220128A1/en
Priority to US10/726,236 priority patent/US20040142895A1/en
Priority to US10/758,155 priority patent/US20050075304A1/en
Priority to US10/764,957 priority patent/US20050054596A1/en
Priority to GBGB0404461.6A priority patent/GB0404461D0/en
Priority to US10/831,620 priority patent/US20050148530A1/en
Priority to US10/844,076 priority patent/US7176304B2/en
Priority to US10/922,761 priority patent/US20050267058A1/en
Priority to JP2008148548A priority patent/JP2009000105A/en

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    • CCHEMISTRY; METALLURGY
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • A61P15/08Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • A61P15/12Drugs for genital or sexual disorders; Contraceptives for climacteric disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • A61P15/18Feminine contraceptives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/06Antipsoriatics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/02Drugs for disorders of the endocrine system of the hypothalamic hormones, e.g. TRH, GnRH, CRH, GRH, somatostatin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue

Abstract

The present invention relates to nucleic acid molecules, including dsRNA, siRNA, antisense, 2,5-A chimeras, aptamers, and enzymatic nucleic acid molecules, such as hammerhead ribozymes, DNAzymes, and allozymes, which modulate the expression of vascular endothelial growth factor receptor (VEGF) and/or vascular endothelial growth factor receptor (VEGFr) genes for the treatment and/or diagnosis of diseases and conditions associated with angiogenesis, such as cancer, tumor angiogenesis, or ocular indications such as diabetic retinopathy, or age related macular degeneration, proliferative diabetic retinopathy, hypoxia-induced angiogenesis, rheumatoid arthritis, psoriasis, wound healing, and female reproductive disorders and conditions, including but not limited to endometriosis, endometrial carcinoma, gynecologic bleeding disorders, irregular menstrual cycles, ovulation, premenstrual syndrome (PMS), and menopausal dysfunction.
PCT/US2002/017674 1995-10-26 2002-05-29 Ribozyme based treatment of female reproductive diseases WO2002096927A2 (en)

Priority Applications (26)

Application Number Priority Date Filing Date Title
AU2002344237A AU2002344237B8 (en) 2001-05-29 2002-05-29 Nucleic Acid Based Modulation of Female Reproductive Diseases and Conditions
CA002448320A CA2448320A1 (en) 2001-05-29 2002-05-29 Ribozyme based treatment of female reproductive diseases
EP02752028A EP1390385A4 (en) 2001-05-29 2002-05-29 Nucleic acid based modulation of female reproductive diseases and conditions
JP2003500106A JP2005500025A (en) 2001-05-29 2002-05-29 Nucleic acid-based regulation of female reproductive diseases and conditions
US10/287,949 US20040102389A1 (en) 1995-10-26 2002-11-04 Nucleic acid-mediated treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US10/306,747 US20030216335A1 (en) 2001-11-30 2002-11-27 Method and reagent for the modulation of female reproductive diseases and conditions
CA002456444A CA2456444A1 (en) 2002-02-20 2003-02-20 Rna interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (sina)
PCT/US2003/005022 WO2003070910A2 (en) 2002-02-20 2003-02-20 INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP2003569803A JP2005517436A (en) 2002-02-20 2003-02-20 RNA interference-mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acids (siNA)
AU2003216323A AU2003216323B2 (en) 2002-02-20 2003-02-20 Inhibition of vascular endothelial growth factor (vegf) and vegf receptor gene expression using short interfereing nucleic acid (sina)
EP03742833A EP1521768A4 (en) 2002-02-20 2003-02-20 RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR AND VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
GB0404898A GB2396864B (en) 2002-02-20 2003-02-20 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression
GB0427955A GB2406569B (en) 2002-02-20 2003-02-20 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfe
US10/665,951 US20040138163A1 (en) 2002-05-29 2003-09-18 RNA interference mediated inhibition of vascular edothelial growth factor and vascular edothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/664,668 US20070203333A1 (en) 2001-11-30 2003-09-18 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/670,011 US20040209832A1 (en) 2001-11-30 2003-09-23 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/683,990 US20040198682A1 (en) 2001-11-30 2003-10-10 RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (siNA)
US10/712,633 US20040220128A1 (en) 1995-10-26 2003-11-13 Nucleic acid based modulation of female reproductive diseases and conditions
US10/726,236 US20040142895A1 (en) 1995-10-26 2003-12-02 Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US10/758,155 US20050075304A1 (en) 2001-11-30 2004-01-12 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/764,957 US20050054596A1 (en) 2001-11-30 2004-01-26 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
GBGB0404461.6A GB0404461D0 (en) 2001-05-29 2004-02-27 Ribozyme based treatment of female reproductive diseases
US10/831,620 US20050148530A1 (en) 2002-02-20 2004-04-23 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/844,076 US7176304B2 (en) 2002-02-20 2004-05-11 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/922,761 US20050267058A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (sINA)
JP2008148548A JP2009000105A (en) 2002-02-20 2008-06-05 RNA INTERFERENCE-MEDIATED INHIBITION OF VASCULAR ENDOTHELIUM GROWTH FACTOR AND VASCULAR ENDOTHELIUM GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID(siNA)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US87016101A 2001-05-29 2001-05-29
US09/870,161 2001-05-29
US33446101P 2001-11-30 2001-11-30
US60/334,461 2001-11-30
US10/138,674 2002-05-03
US10/138,674 US7034009B2 (en) 1995-10-26 2002-05-03 Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
US10/138,674 Continuation-In-Part US7034009B2 (en) 1995-10-26 2002-05-03 Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)

Related Child Applications (6)

Application Number Title Priority Date Filing Date
US10/287,949 Continuation-In-Part US20040102389A1 (en) 1995-10-26 2002-11-04 Nucleic acid-mediated treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US10/664,668 Continuation-In-Part US20070203333A1 (en) 2001-11-30 2003-09-18 RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US10/683,990 Continuation-In-Part US20040198682A1 (en) 2001-05-18 2003-10-10 RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (siNA)
US10/712,633 Continuation US20040220128A1 (en) 1995-10-26 2003-11-13 Nucleic acid based modulation of female reproductive diseases and conditions
US10/712,633 Continuation-In-Part US20040220128A1 (en) 1995-10-26 2003-11-13 Nucleic acid based modulation of female reproductive diseases and conditions
US10/726,236 Continuation-In-Part US20040142895A1 (en) 1995-10-26 2003-12-02 Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway

Publications (2)

Publication Number Publication Date
WO2002096927A2 WO2002096927A2 (en) 2002-12-05
WO2002096927A3 true WO2002096927A3 (en) 2003-02-20

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2002/017674 WO2002096927A2 (en) 1995-10-26 2002-05-29 Ribozyme based treatment of female reproductive diseases

Country Status (6)

Country Link
EP (1) EP1390385A4 (en)
JP (2) JP2005500025A (en)
AU (1) AU2002344237B8 (en)
CA (1) CA2448320A1 (en)
GB (1) GB0404461D0 (en)
WO (1) WO2002096927A2 (en)

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US9233113B2 (en) 2009-06-22 2016-01-12 Ampio Pharmaceuticals, Inc. Method for treatment of diseases
US9351979B2 (en) 2012-12-19 2016-05-31 Ampio Pharmaceuticals, Inc. Methods of treatment of diseases
US9394330B2 (en) 2012-03-21 2016-07-19 Alios Biopharma, Inc. Solid forms of a thiophosphoramidate nucleotide prodrug

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US7833992B2 (en) * 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
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US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20050014172A1 (en) 2002-02-20 2005-01-20 Ivan Richards RNA interference mediated inhibition of muscarinic cholinergic receptor gene expression using short interfering nucleic acid (siNA)
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US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
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EP1432724A4 (en) * 2002-02-20 2006-02-01 Sirna Therapeutics Inc Rna interference mediated inhibition of map kinase genes
EP1495121A2 (en) 2002-04-18 2005-01-12 Lynkeus Biotech GmbH Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
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JP2005500025A (en) 2005-01-06
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EP1390385A2 (en) 2004-02-25
GB0404461D0 (en) 2004-03-31
WO2002096927A2 (en) 2002-12-05
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AU2002344237B8 (en) 2008-11-06
AU2002344237B2 (en) 2008-10-23

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