AU745386B2 - Methods and compositions for use in gene therapy for treatment of hemophilia - Google Patents

Methods and compositions for use in gene therapy for treatment of hemophilia Download PDF

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Publication number
AU745386B2
AU745386B2 AU64586/98A AU6458698A AU745386B2 AU 745386 B2 AU745386 B2 AU 745386B2 AU 64586/98 A AU64586/98 A AU 64586/98A AU 6458698 A AU6458698 A AU 6458698A AU 745386 B2 AU745386 B2 AU 745386B2
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AU
Australia
Prior art keywords
factor
promoter
composition
vector
aav
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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.)
Expired
Application number
AU64586/98A
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English (en)
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AU6458698A (en
Inventor
Roland W. Herzog
Katherine A. High
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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Application filed by Childrens Hospital of Philadelphia CHOP filed Critical Childrens Hospital of Philadelphia CHOP
Publication of AU6458698A publication Critical patent/AU6458698A/en
Application granted granted Critical
Publication of AU745386B2 publication Critical patent/AU745386B2/en
Priority to AU48844/02A priority Critical patent/AU779514C/en
Priority to AU2005201735A priority patent/AU2005201735B2/en
Anticipated expiration legal-status Critical
Expired legal-status Critical Current

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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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)
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU64586/98A 1997-03-14 1998-03-12 Methods and compositions for use in gene therapy for treatment of hemophilia Expired AU745386B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU48844/02A AU779514C (en) 1997-03-14 2002-06-19 Methods and compositions for use in gene therapy for treatment of hemophilia
AU2005201735A AU2005201735B2 (en) 1997-03-14 2005-04-26 Methods and compositions for use in gene therapy for treatment of Hemophilia

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4071197P 1997-03-14 1997-03-14
US60/040711 1997-03-14
PCT/US1998/004790 WO1998041240A1 (en) 1997-03-14 1998-03-12 Methods and compositions for use in gene therapy for treatment of hemophilia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU48844/02A Division AU779514C (en) 1997-03-14 2002-06-19 Methods and compositions for use in gene therapy for treatment of hemophilia

Publications (2)

Publication Number Publication Date
AU6458698A AU6458698A (en) 1998-10-12
AU745386B2 true AU745386B2 (en) 2002-03-21

Family

ID=21912503

Family Applications (1)

Application Number Title Priority Date Filing Date
AU64586/98A Expired AU745386B2 (en) 1997-03-14 1998-03-12 Methods and compositions for use in gene therapy for treatment of hemophilia

Country Status (9)

Country Link
US (3) US6093392A (OSRAM)
EP (1) EP1005376B1 (OSRAM)
JP (1) JP4289687B2 (OSRAM)
AT (1) ATE486614T1 (OSRAM)
AU (1) AU745386B2 (OSRAM)
CA (1) CA2283639A1 (OSRAM)
DE (1) DE69841982D1 (OSRAM)
ES (1) ES2355588T3 (OSRAM)
WO (1) WO1998041240A1 (OSRAM)

Families Citing this family (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5885971A (en) * 1995-03-24 1999-03-23 The Regents Of The University Of California Gene therapy by secretory gland expression
US6531455B1 (en) * 1995-03-24 2003-03-11 The Regents Of The University Of California Delivery of polynucleotides by secretory gland expression
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6797505B2 (en) * 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV5 vector and uses thereof
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
CA2364417C (en) * 1999-02-19 2013-11-05 Matthew John During Peroral gene therapy of diabetes and obesity
AU5470900A (en) * 1999-06-08 2000-12-28 Children's Hospital Of Philadelphia, The Methods for preventing formation of inhibitory antibodies in the setting of genetherapy
US6610906B1 (en) * 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
WO2001049118A1 (en) * 2000-01-03 2001-07-12 Panagenic International, Inc. Compositions comprising genome segments and methods of using the same
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
CA2410901C (en) * 2000-06-01 2012-03-20 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
AU2001268373A1 (en) * 2000-06-13 2001-12-24 The Children's Hospital Of Philadelphia Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
US7125841B2 (en) * 2000-11-14 2006-10-24 The University Of Texas Systems Mutant human factor IX with an increased resistance to inhibition by heparin
US6794376B2 (en) * 2000-12-06 2004-09-21 William Beaumont Hospital Methods and compositions for enhancing diffusion of therapeutic agents through tissue
US20020159978A1 (en) * 2001-02-06 2002-10-31 James Allen Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions
EP1418810A4 (en) 2001-08-03 2006-08-02 Us Gov Health & Human Serv TREATMENT OF HEMOPHILIA ORAL
ES2388170T3 (es) 2001-08-24 2012-10-09 Uvic Industry Partnerships Inc. Proaerolisina que contiene secuencias de activación de proteasa y métodos de uso para el tratamiento del cáncer de próstata
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
US20060078542A1 (en) * 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
US20060148742A1 (en) * 2004-02-26 2006-07-06 Kaye David M Polynucleotide delivery to cardiac tissue
US7722596B2 (en) * 2004-02-26 2010-05-25 Osprey Medical, Inc. Regional cardiac tissue treatment
DK1804839T3 (da) 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US8152786B2 (en) * 2006-11-07 2012-04-10 Osprey Medical, Inc. Collection catheter and kit
US9050400B2 (en) * 2008-08-12 2015-06-09 Osprey Medical, Inc. Remote sensing catheter system and methods
US20100041984A1 (en) * 2008-08-12 2010-02-18 James Edward Shapland Impedance sensing device and catheter system
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
FR2952374B1 (fr) * 2009-11-06 2012-11-16 Univ Claude Bernard Lyon Production de facteur ix recombinant dans une lignee cellulaire hepatocytaire humaine
WO2011057243A2 (en) * 2009-11-09 2011-05-12 University Of Central Florida Research Foundation, Inc. Administration of plant expressed oral tolerance agents
IN2012DN04875A (OSRAM) * 2009-12-08 2015-09-25 Health Research Inc
US9295816B2 (en) * 2009-12-09 2016-03-29 Osprey Medical, Inc. Catheter with distal and proximal ports
ES2680915T3 (es) * 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
KR101930711B1 (ko) 2010-10-12 2018-12-19 더 칠드런스 호스피탈 오브 필라델피아 혈우병 ｂ의 치료를 위한 방법 및 조성물
PT2717893T (pt) * 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
WO2012177778A1 (en) 2011-06-20 2012-12-27 Mount Sinai School Of Medicine Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders
WO2013023126A2 (en) 2011-08-11 2013-02-14 Osprey Medical Inc. Systems and methods for limb treatment
WO2017180807A1 (en) 2016-04-15 2017-10-19 Baxalta Incorporated Method and apparatus for providing a pharmacokinetic drug dosing regiment
AU2014287005C1 (en) * 2013-07-12 2021-06-03 The Children's Hospital Of Philadelphia AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies
MX392639B (es) 2014-03-21 2025-03-24 Univ Leland Stanford Junior Vectores virales recombinantes para la integración de transgenes
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2017100676A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
US10896749B2 (en) 2017-01-27 2021-01-19 Shire Human Genetic Therapies, Inc. Drug monitoring tool
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
WO2019083171A2 (ko) 2017-10-26 2019-05-02 주식회사 큐로진생명과학 솔루블 vegfr-1 변이체 cdna를 함유하는 raav를 포함하는 황반변성 치료용 조성물
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
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0316558A1 (en) * 1982-08-04 1989-05-24 Btg International Limited Factor IX DNA
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
NZ244306A (en) * 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5834266A (en) * 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US6342390B1 (en) * 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
US5843742A (en) * 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US5834306A (en) * 1994-12-23 1998-11-10 Sri International Tissue specific hypoxia regulated therapeutic constructs
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5817784A (en) * 1996-08-09 1998-10-06 Amgen Inc. Neurogene
AU723497C (en) * 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
JP2001501819A (ja) * 1996-09-25 2001-02-13 セル・ジェネシス・インコーポレイテッド アデノ随伴ウィルスベクターの非侵襲性投与

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CHEN L. ET AL (1997) HUM GENE THER 8(2):125-35 *
KOEBERL D. D. ET AL (1997) PROC NATL ACAD SCI USA 94:1426-31 *

Also Published As

Publication number Publication date
ES2355588T3 (es) 2011-03-29
US6093392A (en) 2000-07-25
AU6458698A (en) 1998-10-12
ATE486614T1 (de) 2010-11-15
EP1005376A1 (en) 2000-06-07
JP4289687B2 (ja) 2009-07-01
US7998734B2 (en) 2011-08-16
DE69841982D1 (de) 2010-12-16
EP1005376B1 (en) 2010-11-03
CA2283639A1 (en) 1998-09-24
US20080213222A1 (en) 2008-09-04
US20110263690A1 (en) 2011-10-27
EP1005376A4 (en) 2004-11-24
WO1998041240A1 (en) 1998-09-24
JP2001517221A (ja) 2001-10-02

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