JP2023536008A - ベクター - Google Patents

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Publication number
JP2023536008A
JP2023536008A JP2023523681A JP2023523681A JP2023536008A JP 2023536008 A JP2023536008 A JP 2023536008A JP 2023523681 A JP2023523681 A JP 2023523681A JP 2023523681 A JP2023523681 A JP 2023523681A JP 2023536008 A JP2023536008 A JP 2023536008A
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JP
Japan
Prior art keywords
promoter
viral vector
sequence identity
vector
seq
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JP2023523681A
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English (en)
Japanese (ja)
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JP2023536008A5 (https=
JPWO2022003357A5 (https=
Inventor
ウィリアム グリフィス，アラン
スタンチュク，ルーカス
クズムク，ヴァレリア
ホロウウッド，クリス
シュミット，ドミニク
アーソン サレーム－ウッディン，モイン
イアン ウェルシュ，ギャビン
Original Assignee
シンコナ インヴェストメント マネージメント リミティッド
ザ ユニバーシティ オブ ブリストル
ピュアスプリング セラピューティクス リミテッド
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Application filed by シンコナ インヴェストメント マネージメント リミティッド, ザ ユニバーシティ オブ ブリストル, ピュアスプリング セラピューティクス リミテッド filed Critical シンコナ インヴェストメント マネージメント リミティッド
Publication of JP2023536008A publication Critical patent/JP2023536008A/ja
Publication of JP2023536008A5 publication Critical patent/JP2023536008A5/ja
Publication of JPWO2022003357A5 publication Critical patent/JPWO2022003357A5/ja
Pending 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)
  • Image Analysis (AREA)
  • Devices For Executing Special Programs (AREA)
JP2023523681A 2020-06-30 2021-06-30 ベクター Pending JP2023536008A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB2010009.5 2020-06-30
GBGB2010009.5A GB202010009D0 (en) 2020-06-30 2020-06-30 Vector
PCT/GB2021/051668 WO2022003357A1 (en) 2020-06-30 2021-06-30 Vector

Publications (3)

Publication Number Publication Date
JP2023536008A true JP2023536008A (ja) 2023-08-22
JP2023536008A5 JP2023536008A5 (https=) 2024-07-08
JPWO2022003357A5 JPWO2022003357A5 (https=) 2024-07-08

Family

ID=71949697

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023523681A Pending JP2023536008A (ja) 2020-06-30 2021-06-30 ベクター

Country Status (12)

Country Link
US (1) US20230175016A1 (https=)
EP (1) EP4172345A1 (https=)
JP (1) JP2023536008A (https=)
KR (1) KR20230056658A (https=)
CN (1) CN115997025A (https=)
AU (1) AU2021300616A1 (https=)
BR (1) BR112022026521A2 (https=)
CA (1) CA3183830A1 (https=)
GB (1) GB202010009D0 (https=)
IL (1) IL299505A (https=)
MX (1) MX2022016101A (https=)
WO (1) WO2022003357A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy
AU2021293174A1 (en) 2020-06-14 2023-01-19 Vertex Pharmaceuticals Incorporated Complement Factor I-related compositions and methods
GB202217332D0 (en) * 2022-11-18 2023-01-04 Univ Bristol Methods
EP4410988A1 (en) * 2023-01-31 2024-08-07 Universitätsklinikum Hamburg-Eppendorf An aav2-vector variant for targeted transfer of genes
WO2024178007A1 (en) * 2023-02-21 2024-08-29 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Mafb promoter for gene therapy for diabetes
GB202304841D0 (en) * 2023-03-31 2023-05-17 Purespring Therapeutics Ltd Promoter
AU2024262827A1 (en) * 2023-04-27 2025-10-16 Oregon Health & Science University Kidney-targeting aav capsids and methods of use thereof
GB202414499D0 (en) * 2024-10-02 2024-11-13 Purespring Therapeutics Ltd Methods

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005012351A2 (en) * 2003-08-01 2005-02-10 The Brigham And Women's Hospital, Inc. Novel functions for decay accelerating factor (daf) in inflammation
WO2013029030A1 (en) * 2011-08-24 2013-02-28 The Board Of Trustees Of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
JP2014204729A (ja) * 2005-02-14 2014-10-30 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 加齢性黄斑変性を処置および診断するための方法および試薬
JP2016104767A (ja) * 2007-11-08 2016-06-09 ザ ジェネラル ホスピタル コーポレイション 蛋白尿症の治療のための方法及び組成物
JP2018527941A (ja) * 2015-09-24 2018-09-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 補体媒介性疾患を処置するための組成物及び方法
WO2020056122A1 (en) * 2018-09-13 2020-03-19 Regeneron Pharmaceuticals, Inc. Complement factor h gene knockout rat as a model of c3 glomerulopathy

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL332875A1 (en) 1996-10-17 1999-10-25 Oxford Biomedica Ltd Retroviral vectors
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005012351A2 (en) * 2003-08-01 2005-02-10 The Brigham And Women's Hospital, Inc. Novel functions for decay accelerating factor (daf) in inflammation
JP2014204729A (ja) * 2005-02-14 2014-10-30 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 加齢性黄斑変性を処置および診断するための方法および試薬
JP2016104767A (ja) * 2007-11-08 2016-06-09 ザ ジェネラル ホスピタル コーポレイション 蛋白尿症の治療のための方法及び組成物
WO2013029030A1 (en) * 2011-08-24 2013-02-28 The Board Of Trustees Of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
JP2018527941A (ja) * 2015-09-24 2018-09-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 補体媒介性疾患を処置するための組成物及び方法
WO2020056122A1 (en) * 2018-09-13 2020-03-19 Regeneron Pharmaceuticals, Inc. Complement factor h gene knockout rat as a model of c3 glomerulopathy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DING, WEN Y. ET AL.: ""Adeno-associated virus gene therapy prevents progression of kidney disease in genetic human and mou", JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, vol. 30, JPN6025022798, November 2019 (2019-11-01), pages 94, ISSN: 0005614573 *

Also Published As

Publication number Publication date
IL299505A (en) 2023-02-01
GB202010009D0 (en) 2020-08-12
CA3183830A1 (en) 2022-01-06
KR20230056658A (ko) 2023-04-27
MX2022016101A (es) 2023-06-01
EP4172345A1 (en) 2023-05-03
AU2021300616A1 (en) 2023-01-19
US20230175016A1 (en) 2023-06-08
BR112022026521A2 (pt) 2023-01-31
WO2022003357A1 (en) 2022-01-06
CN115997025A (zh) 2023-04-21

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