JP2018518196A - Ｈｉｖの治療のための遺伝子治療法およびその使用 - Google Patents

Ｈｉｖの治療のための遺伝子治療法およびその使用 Download PDF

Info

Publication number
JP2018518196A
JP2018518196A JP2018513266A JP2018513266A JP2018518196A JP 2018518196 A JP2018518196 A JP 2018518196A JP 2018513266 A JP2018513266 A JP 2018513266A JP 2018513266 A JP2018513266 A JP 2018513266A JP 2018518196 A JP2018518196 A JP 2018518196A
Authority
JP
Japan
Prior art keywords
nucleic acid
sequence
hiv
seq
expression vector
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.)
Ceased
Application number
JP2018513266A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018518196A5 (https=
Inventor
鈴木　一雄
一雄 鈴木
アンソニー ドミニク ケレハー
アンソニー ドミニク ケレハー
ジェフリー フィリップ シモンズ
ジェフリー フィリップ シモンズ
シャンテル リーザ イブリン アーレンスティール
シャンテル リーザ イブリン アーレンスティール
Original Assignee
カリミューン， インコーポレーティッド
カリミューン， インコーポレーティッド
カリミューン オーストラリア ピーティーワイ リミテッド
カリミューン オーストラリア ピーティーワイ リミテッド
セント ヴィンセンツ ホスピタル シドニー リミテッド
セント ヴィンセンツ ホスピタル シドニー リミテッド
ニューサウス イノベイションズ ピーティーワイ リミテッド
ニューサウス イノベイションズ ピーティーワイ リミテッド
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 カリミューン， インコーポレーティッド, カリミューン， インコーポレーティッド, カリミューン オーストラリア ピーティーワイ リミテッド, カリミューン オーストラリア ピーティーワイ リミテッド, セント ヴィンセンツ ホスピタル シドニー リミテッド, セント ヴィンセンツ ホスピタル シドニー リミテッド, ニューサウス イノベイションズ ピーティーワイ リミテッド, ニューサウス イノベイションズ ピーティーワイ リミテッド filed Critical カリミューン， インコーポレーティッド
Publication of JP2018518196A publication Critical patent/JP2018518196A/ja
Publication of JP2018518196A5 publication Critical patent/JP2018518196A5/ja
Ceased legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • C12N15/1138—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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
JP2018513266A 2015-05-18 2016-02-15 Ｈｉｖの治療のための遺伝子治療法およびその使用 Ceased JP2018518196A (ja)

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 (2)

Publication Number Publication Date
JP2018518196A true JP2018518196A (ja) 2018-07-12
JP2018518196A5 JP2018518196A5 (https=) 2019-03-28

Family

ID=55538591

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018513266A Ceased JP2018518196A (ja) 2015-05-18 2016-02-15 Ｈｉｖの治療のための遺伝子治療法およびその使用

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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021138721A (ja) * 2015-07-08 2021-09-16 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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012533299A (ja) * 2009-07-15 2012-12-27 カリミューン， インコーポレーティッド ヒト免疫不全ウイルス阻害のための二重ベクター

Family Cites Families (4)

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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012533299A (ja) * 2009-07-15 2012-12-27 カリミューン， インコーポレーティッド ヒト免疫不全ウイルス阻害のための二重ベクター

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
J.BIOL.CHEM., 2008, VOL. 283, NO. 34, PP. 23353-23363, JPN6019046471, ISSN: 0004163076 *
PLOS ONE, 2012, VOL. 7, NO. 12, E53492, PP. 1-14, JPN6019046473, ISSN: 0004163075 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021138721A (ja) * 2015-07-08 2021-09-16 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

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
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

Similar Documents

Publication Publication Date Title
US20210032627A1 (en) Gene therapeutic for the treatment of hiv and uses thereof
JP6529141B2 (ja) ヒト免疫不全ウイルス阻害のための二重ベクター
AU2017219605B2 (en) Excision of retroviral nucleic acid sequences
Nielsen et al. Molecular strategies to inhibit HIV-1 replication
US20160289681A1 (en) Rna-based hiv inhibitors
Reinhard et al. Vpx rescue of HIV-1 from the antiviral state in mature dendritic cells is independent of the intracellular deoxynucleotide concentration
WO2010117974A2 (en) Hiv-resistant stem cells and uses thereof
Richter et al. Strategies for inhibiting function of HIV-1 accessory proteins: a necessary route to AIDS therapy?
Zeller et al. RNA-based gene therapy for the treatment and prevention of HIV: from bench to bedside
US20200339994A1 (en) Treatment of HIV
US20140010839A1 (en) Method of screening antiretroviral compounds and vaccine
WO2012159120A2 (en) Gene therapy based strategy for treating hiv
Reyes-Darias et al. Inhibition of HIV-1 replication by RNA-based strategies
Poluri et al. Genetic therapy for HIV/AIDS
WO2022256516A9 (en) Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
Ran Roles of HIV virion-associated envelope glycoprotein in modulating various cellular pathways and facilitating viral replication and its pathogenicity
Turner Transcriptional Gene Silencing of HIV-1 by Non-Coding RNAs: Progress towards a TGS-based genetic therapy
Neff Evaluation of novel therapeutics for HIV prevention and treatment in a humanized mouse model
Scarborough Targeting HIV-1 RNA with ribozymes and small interfering RNAs for therapeutic applications
Ledger Use of Short-Hairpin RNA to CCR5, and maC46 Entry Inhibitor Gene-Therapies against HIV infection
Lorgeoux Multiple roles of DDX17 in human immunodeficiency virus type 1 replication
KR20130094915A (ko) 인간 면역 결핍 바이러스의 엔씨와 텟 단백질의 상호작용 및 바이러스 잠복상태 치료제의 표적으로서의 용도
HK1169806A (en) Hiv-resistant stem cells and uses thereof

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190213

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190213

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20191122

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20191128

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200226

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200424

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200522

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200916

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20201215

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210226

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20210315

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210413

A045 Written measure of dismissal of application [lapsed due to lack of payment]

Free format text: JAPANESE INTERMEDIATE CODE: A045

Effective date: 20210726