CN108026543A - 用于hiv的治疗的基因疗法及其用途 - Google Patents

用于hiv的治疗的基因疗法及其用途 Download PDF

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Publication number
CN108026543A
CN108026543A CN201680042303.7A CN201680042303A CN108026543A CN 108026543 A CN108026543 A CN 108026543A CN 201680042303 A CN201680042303 A CN 201680042303A CN 108026543 A CN108026543 A CN 108026543A
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China
Prior art keywords
nucleic acid
hiv
sequence
expression vector
silencing
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.)
Pending
Application number
CN201680042303.7A
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English (en)
Chinese (zh)
Inventor
K·铃木
A·D·凯莱赫
G·P·西蒙兹
C·L·E·阿伦斯蒂尔
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.)
NewSouth Innovations Pty Ltd
St Vincents Hospital Sydney Ltd
CSL Gene Therapy Pty Ltd
CSL Behring Gene Therapy Inc
Original Assignee
NewSouth Innovations Pty Ltd
St Vincents Hospital Sydney Ltd
Calimmune Australia Pty Ltd
Calimmune Inc
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Filing date
Publication date
Application filed by NewSouth Innovations Pty Ltd, St Vincents Hospital Sydney Ltd, Calimmune Australia Pty Ltd, Calimmune Inc filed Critical NewSouth Innovations Pty Ltd
Publication of CN108026543A publication Critical patent/CN108026543A/zh
Pending legal-status Critical Current

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    • 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
    • C12N15/113—Non-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
    • C12N15/1131—Non-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 viruses
    • C12N15/1132—Non-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 viruses against retroviridae, e.g. HIV
    • 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
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/113—Non-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
    • C12N15/1138—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • AIDS & HIV (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201680042303.7A 2015-05-18 2016-02-15 用于hiv的治疗的基因疗法及其用途 Pending CN108026543A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562163332P 2015-05-18 2015-05-18
US62/163,332 2015-05-18
PCT/US2016/017931 WO2016186708A1 (en) 2015-05-18 2016-02-15 Gene therapeutic for the treatment of hiv and uses thereof

Publications (1)

Publication Number Publication Date
CN108026543A true CN108026543A (zh) 2018-05-11

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Application Number Title Priority Date Filing Date
CN201680042303.7A Pending CN108026543A (zh) 2015-05-18 2016-02-15 用于hiv的治疗的基因疗法及其用途

Country Status (9)

Country Link
US (2) US20180112220A1 (https=)
EP (1) EP3294895B1 (https=)
JP (1) JP2018518196A (https=)
CN (1) CN108026543A (https=)
AU (1) AU2016265003A1 (https=)
CA (1) CA2986469A1 (https=)
ES (1) ES2833449T3 (https=)
HK (1) HK1247963A1 (https=)
WO (1) WO2016186708A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6890831B2 (ja) * 2015-07-08 2021-06-18 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Ｈｉｖ予備免疫化および免疫療法
US10888613B2 (en) 2016-02-08 2021-01-12 American Gene Technologies International Inc. Method of producing cells resistant to HIV infection
IL284348B2 (en) 2016-07-08 2025-06-01 American Gene Tech Int Inc Hiv pre-immunization and immunotherapy
US20200061117A1 (en) * 2017-05-08 2020-02-27 The Regents Of The University Of California Protective Chimeric Antigen Receptor Stem Cell Gene Therapy for Viral Infection
WO2019018383A1 (en) 2017-07-18 2019-01-24 Calimmune, Inc. COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
JP2022514955A (ja) 2018-12-23 2022-02-16 シーエスエル・ベーリング・エルエルシー ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療
BR112021012318A2 (pt) 2018-12-23 2022-01-18 Csl Behring Llc Células t doadoras com interruptor de eliminação
WO2021263070A1 (en) 2020-06-26 2021-12-30 Csl Behring Llc Donor t-cells with kill switch

Citations (3)

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US20070116767A1 (en) * 2003-02-14 2007-05-24 Mohapatra Shyam S Chitosan-microparticles for ifn gene delivery
US20080003682A1 (en) * 2001-09-13 2008-01-03 Carlos Lois-Caballe Method for expression of small antiviral rna molecules with reduced cytotoxicity within a cell
WO2011008348A2 (en) * 2009-07-15 2011-01-20 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus

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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
WO2016029275A1 (en) * 2014-08-28 2016-03-03 Newsouth Innovations Pty Limited Treatment of hiv

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
US20080003682A1 (en) * 2001-09-13 2008-01-03 Carlos Lois-Caballe Method for expression of small antiviral rna molecules with reduced cytotoxicity within a cell
US20070116767A1 (en) * 2003-02-14 2007-05-24 Mohapatra Shyam S Chitosan-microparticles for ifn gene delivery
WO2011008348A2 (en) * 2009-07-15 2011-01-20 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus

Non-Patent Citations (9)

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Title
CHANTELLE AHLENSTIEL ET AL.: "Novel RNA Duplex Locks HIV-1 in a Latent State via Chromatin-mediated Transcriptional Silencing", 《MOLECULAR THERAPY—NUCLEIC ACIDS》 *
GENE-ERROL E. RINGPIS ET AL.: "Engineering HIV-1-Resistant T-Cells from Short-Hairpin RNA-Expressing Hematopoietic Stem/Progenitor Cells in Humanized BLT Mice", 《PLOS ONE》 *
JON E. WALKER ET AL.: "Generation of an HIV-1-Resistant Immune System with CD34+ Hematopoietic Stem Cells Transduced with a Triple-Combination Anti-HIV Lentiviral Vector", 《JOURNAL OF VIROLOGY》 *
JOSEPH S ANDERSON ET AL.: "Preintegration HIV-1 Inhibition by a Combination Lentiviral Vector Containing a Chimeric TRIM5α Protein, a CCR5 shRNA, and a TAR Decoy", 《MOLECULAR THERAPY》 *
KAZUO SUZUKI ET AL.: "Promoter Targeting shRNA Suppresses HIV-1 Infection In vivo Through Transcriptional Gene Silencing", 《MOLECULAR THERAPY—NUCLEIC ACIDS》 *
KAZUO SUZUKI ET AL.: "Transcriptional gene silencing of HIV-1 through promoter targeted RNA is highly specific", 《RNA BIOLOGY》 *
ORIT WOLSTEIN ET AL.: "Preclinical safety and efficacy of an anti–HIV-1 lentiviral vector containing a short hairpin RNAto CCR5 and the C46 fusion inhibitor", 《MOLECULAR THERAPY — METHODS & CLINICAL DEVELOPMENT》 *
YE LIU ET AL.: "A Novel Approach to Block HIV-1 Coreceptor CXCR4 in Non-toxic Manner", 《MOL BIOTECHNOL》 *
郝彦哲等: "慢病毒介导双shRNA靶向于HIV的抑制作用研究", 《病毒学报》 *

Also Published As

Publication number Publication date
US20210032627A1 (en) 2021-02-04
US20180112220A1 (en) 2018-04-26
WO2016186708A1 (en) 2016-11-24
HK1247963A1 (zh) 2018-10-05
CA2986469A1 (en) 2016-11-24
AU2016265003A1 (en) 2017-12-07
EP3294895A1 (en) 2018-03-21
EP3294895B1 (en) 2020-09-09
ES2833449T3 (es) 2021-06-15
JP2018518196A (ja) 2018-07-12

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