CL2020002445A1 - Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b (divisional de la solicitud no. 201903395) - Google Patents

Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b (divisional de la solicitud no. 201903395)

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Publication number
CL2020002445A1
CL2020002445A1 CL2020002445A CL2020002445A CL2020002445A1 CL 2020002445 A1 CL2020002445 A1 CL 2020002445A1 CL 2020002445 A CL2020002445 A CL 2020002445A CL 2020002445 A CL2020002445 A CL 2020002445A CL 2020002445 A1 CL2020002445 A1 CL 2020002445A1
Authority
CL
Chile
Prior art keywords
gene therapy
hemophilia
viral vectors
divisional application
vectors encoding
Prior art date
Application number
CL2020002445A
Other languages
English (en)
Inventor
Johannes Lengler
Hanspeter Rottensteiner
Friedrich Scheiflinger
Franziska Horling
Falko-Gunther Falkner
Original Assignee
Baxalta Inc
Baxalta GmbH
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 Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of CL2020002445A1 publication Critical patent/CL2020002445A1/es

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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
    • A61K38/43—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

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

Abstract

La presente descripción proporciona, entre otros aspectos, polinucleótidos modificados con codones que codifican variantes del Factor IX para expresión en células de mamífero. En algunas modalidades, la descripción también proporciona vectores de terapia génica de mamífero, uso para tratar la hemofilia B y método para producir una partícula de virus adenoasociado (AAV).
CL2020002445A 2017-05-22 2020-09-23 Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b (divisional de la solicitud no. 201903395) CL2020002445A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762509616P 2017-05-22 2017-05-22

Publications (1)

Publication Number Publication Date
CL2020002445A1 true CL2020002445A1 (es) 2020-12-28

Family

ID=62621009

Family Applications (2)

Application Number Title Priority Date Filing Date
CL2019003395A CL2019003395A1 (es) 2017-05-22 2019-11-21 Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b.
CL2020002445A CL2020002445A1 (es) 2017-05-22 2020-09-23 Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b (divisional de la solicitud no. 201903395)

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Application Number Title Priority Date Filing Date
CL2019003395A CL2019003395A1 (es) 2017-05-22 2019-11-21 Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b.

Country Status (17)

Country Link
US (2) US10842853B2 (es)
EP (1) EP3630974A1 (es)
JP (1) JP6961171B2 (es)
KR (2) KR102386890B1 (es)
CN (1) CN110945127A (es)
AR (1) AR112057A1 (es)
AU (1) AU2018272831A1 (es)
BR (1) BR112019024256A2 (es)
CA (1) CA3064730A1 (es)
CL (2) CL2019003395A1 (es)
CO (1) CO2019012885A2 (es)
IL (2) IL270567B (es)
MX (1) MX2019013836A (es)
NZ (1) NZ759034A (es)
TW (1) TWI753168B (es)
WO (1) WO2018217731A1 (es)
ZA (1) ZA201907716B (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
US11596671B2 (en) * 2019-02-01 2023-03-07 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression
AU2020242945A1 (en) * 2019-03-19 2021-10-14 CSL Innovation Pty Ltd Factor IX variants and uses thereof in therapy
WO2020257586A2 (en) 2019-06-20 2020-12-24 Baxalta Incorporated Method of treatment with viral-based gene therapy
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
TW202246505A (zh) * 2021-03-05 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途
CN117693593A (zh) * 2021-06-23 2024-03-12 英斯培瑞有限公司 用于治疗b型血友病的组合物和方法
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
CA3232470A1 (en) * 2021-10-27 2023-05-04 Leah SABIN Compositions and methods for expressing factor ix for hemophilia b therapy
WO2023202469A1 (zh) * 2022-04-19 2023-10-26 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
CA2162497A1 (en) 1993-06-10 1994-12-22 Sheila Connelly Adenoviral vectors for treatment of hemophilia
US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
CN1172435A (zh) 1994-09-23 1998-02-04 综合医院公司 使用非哺乳动物dna病毒在哺乳动物细胞中表达外源基因
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
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
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
JP4289687B2 (ja) 1997-03-14 2009-07-01 ザ チルドレンズ ホスピタル オブ フィラデルフィア 血友病の治療のための遺伝子治療で使用する方法と組成物
US6531298B2 (en) 1997-07-21 2003-03-11 The University Of North Carolina At Chapel Hill Factor IX antihemophilic factor with increased clotting activity
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
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
CN1148228C (zh) 2000-08-30 2004-05-05 夏家辉 治疗血友病b的基因药物及其制备方法
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
EP2438931B1 (en) * 2004-09-22 2013-11-13 St. Jude Children's Research Hospital Improved expression of factor IX in gene therapy vectors
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US8383388B2 (en) * 2006-06-19 2013-02-26 Catalyst Biosciences, Inc. Modified coagulation factor IX polypeptides and use thereof for treatment
JP5797551B2 (ja) 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
DK2337849T3 (en) 2008-09-15 2018-10-01 Uniqure Biopharma B V FACTOR IX POLYPEPTIME MUTANT, APPLICATIONS THEREOF AND METHOD OF PRODUCING THEREOF
US9663778B2 (en) * 2009-07-09 2017-05-30 OPKO Biologies Ltd. Long-acting coagulation factors and methods of producing same
TWI595004B (zh) 2010-11-03 2017-08-11 介控生化科技公司 經修飾之第九因子多胜肽及其用途
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
ES2923757T3 (es) * 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
WO2013123503A1 (en) 2012-02-17 2013-08-22 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
AU2013336601B2 (en) * 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
WO2014063753A1 (en) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
DK3270944T3 (da) 2015-03-17 2020-01-27 Univ Brussel Vrije Optimerede leverspecifikke ekspressionssystemer til FVIII og FIX
EP3313991A4 (en) 2015-06-23 2018-12-05 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
CN116732066A (zh) * 2016-10-14 2023-09-12 四川至善唯新生物科技有限公司 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用

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Publication number Publication date
ZA201907716B (en) 2022-06-29
AR112057A1 (es) 2019-09-18
BR112019024256A2 (pt) 2020-06-09
JP6961171B2 (ja) 2021-11-05
KR102386890B1 (ko) 2022-04-15
IL280637A (en) 2021-03-25
WO2018217731A1 (en) 2018-11-29
JP2020520662A (ja) 2020-07-16
KR20220050238A (ko) 2022-04-22
CL2019003395A1 (es) 2020-04-17
CN110945127A (zh) 2020-03-31
KR20200010443A (ko) 2020-01-30
TWI753168B (zh) 2022-01-21
MX2019013836A (es) 2020-01-30
KR102551733B1 (ko) 2023-07-06
US20180339026A1 (en) 2018-11-29
RU2019142702A (ru) 2021-06-24
NZ759034A (en) 2023-07-28
RU2019142702A3 (es) 2021-10-12
EP3630974A1 (en) 2020-04-08
TW201900184A (zh) 2019-01-01
US10842853B2 (en) 2020-11-24
IL270567B (en) 2021-03-25
AU2018272831A1 (en) 2019-11-28
CA3064730A1 (en) 2018-11-29
CO2019012885A2 (es) 2020-01-17
US20210128700A1 (en) 2021-05-06

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