HK1247963A1 - 用於hiv的治疗的基因疗法及其用途 - Google Patents

用於hiv的治疗的基因疗法及其用途 Download PDF

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Publication number
HK1247963A1
HK1247963A1 HK18107365.2A HK18107365A HK1247963A1 HK 1247963 A1 HK1247963 A1 HK 1247963A1 HK 18107365 A HK18107365 A HK 18107365A HK 1247963 A1 HK1247963 A1 HK 1247963A1
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HK
Hong Kong
Prior art keywords
nucleic acid
hiv
sequence
expression vector
silencing
Prior art date
Application number
HK18107365.2A
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English (en)
Chinese (zh)
Inventor
K.铃木
K．鈴木
A．D．凯莱赫
G．P．西蒙兹
A．D．凱萊赫
C．L．E‧阿伦斯蒂尔
C．L．E‧阿倫斯蒂爾
Original Assignee
Csl贝林基因治疗股份有限公司
卡琳繆恩股份有限公司
Csl基因治疗有限责任公司
圣文森特医院悉尼有限公司
卡琳繆恩澳大利亞私人有限公司
新南创新私人有限公司
聖文森特醫院悉尼有限公司
新南創新私人有限公司
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 Csl贝林基因治疗股份有限公司, 卡琳繆恩股份有限公司, Csl基因治疗有限责任公司, 圣文森特医院悉尼有限公司, 卡琳繆恩澳大利亞私人有限公司, 新南创新私人有限公司, 聖文森特醫院悉尼有限公司, 新南創新私人有限公司 filed Critical Csl贝林基因治疗股份有限公司
Publication of HK1247963A1 publication Critical patent/HK1247963A1/zh

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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
    • C12N15/11—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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    • 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
    • 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
    • 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
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    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • 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)
HK18107365.2A 2015-05-18 2016-02-15 用於hiv的治疗的基因疗法及其用途 HK1247963A1 (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
HK1247963A1 true HK1247963A1 (zh) 2018-10-05

Family

ID=55538591

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18107365.2A HK1247963A1 (zh) 2015-05-18 2016-02-15 用於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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US7737124B2 (en) * 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
WO2004074314A2 (en) * 2003-02-14 2004-09-02 University Of South Florida Chistosan-microparticles for ifn gene delivery
EP3659435A1 (en) * 2009-07-15 2020-06-03 Calimmune, Inc. Dual vector for inhibition of human immunodeficiency virus
WO2016029275A1 (en) * 2014-08-28 2016-03-03 Newsouth Innovations Pty Limited Treatment of hiv

Also Published As

Publication number Publication date
US20210032627A1 (en) 2021-02-04
US20180112220A1 (en) 2018-04-26
WO2016186708A1 (en) 2016-11-24
CN108026543A (zh) 2018-05-11
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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