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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Pending
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JP2023523681A
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English (en)
Japanese (ja)
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JP2023536008A5 (enExample
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
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)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)
  • Image Analysis (AREA)
  • Devices For Executing Special Programs (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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
GBGB2010009.5A GB202010009D0 (en) 2020-06-30 2020-06-30 Vector
GB2010009.5 2020-06-30
PCT/GB2021/051668 WO2022003357A1 (en) 2020-06-30 2021-06-30 Vector

Publications (2)

Publication Number Publication Date
JP2023536008A true JP2023536008A (ja) 2023-08-22
JP2023536008A5 JP2023536008A5 (enExample) 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 (enExample)
EP (1) EP4172345A1 (enExample)
JP (1) JP2023536008A (enExample)
KR (1) KR20230056658A (enExample)
CN (1) CN115997025A (enExample)
AU (1) AU2021300616A1 (enExample)
BR (1) BR112022026521A2 (enExample)
CA (1) CA3183830A1 (enExample)
GB (1) GB202010009D0 (enExample)
IL (1) IL299505A (enExample)
MX (1) MX2022016101A (enExample)
WO (1) WO2022003357A1 (enExample)

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
WO2021257480A2 (en) 2020-06-14 2021-12-23 Catalyst Biosciences, Inc. 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
EP4669359A1 (en) * 2023-02-21 2025-12-31 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
CN121013858A (zh) * 2023-04-27 2025-11-25 俄勒冈健康与科学大学 靶向肾脏的aav衣壳及其使用方法
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
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
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
AU2021300616A1 (en) 2023-01-19
EP4172345A1 (en) 2023-05-03
CA3183830A1 (en) 2022-01-06
IL299505A (en) 2023-02-01
US20230175016A1 (en) 2023-06-08
CN115997025A (zh) 2023-04-21
MX2022016101A (es) 2023-06-01
GB202010009D0 (en) 2020-08-12
WO2022003357A1 (en) 2022-01-06
BR112022026521A2 (pt) 2023-01-31
KR20230056658A (ko) 2023-04-27

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