CN115997025A - 载体 - Google Patents

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Publication number
CN115997025A
CN115997025A CN202180046691.7A CN202180046691A CN115997025A CN 115997025 A CN115997025 A CN 115997025A CN 202180046691 A CN202180046691 A CN 202180046691A CN 115997025 A CN115997025 A CN 115997025A
Authority
CN
China
Prior art keywords
promoter
viral vector
seq
sequence identity
aav
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
CN202180046691.7A
Other languages
English (en)
Chinese (zh)
Inventor
A.W.格里菲思
L.斯坦楚克
V.库兹穆克
C.霍洛伍德
D.施密特
M.A.萨利姆-乌丁
G.I.韦尔什
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.)
Xinkona Intellectual Property Holdings 3 Co ltd
University of Bristol
Original Assignee
Prespring Treatment Co ltd
University of Bristol
Syncona Management LLP
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 Prespring Treatment Co ltd, University of Bristol, Syncona Management LLP filed Critical Prespring Treatment Co ltd
Publication of CN115997025A publication Critical patent/CN115997025A/zh
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)
CN202180046691.7A 2020-06-30 2021-06-30 载体 Pending CN115997025A (zh)

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 (1)

Publication Number Publication Date
CN115997025A true CN115997025A (zh) 2023-04-21

Family

ID=71949697

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180046691.7A Pending CN115997025A (zh) 2020-06-30 2021-06-30 载体

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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103920142A (zh) * 2005-02-14 2014-07-16 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
CN106062200A (zh) * 2014-02-17 2016-10-26 伦敦国王学院 腺相关病毒载体

Family Cites Families (6)

* 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
WO2005012351A2 (en) * 2003-08-01 2005-02-10 The Brigham And Women's Hospital, Inc. Novel functions for decay accelerating factor (daf) in inflammation
ES2457822T3 (es) * 2007-11-08 2014-04-29 The General Hospital Corporation Procedimientos y composiciones de tratamiento de enfermedades proteinúricas
ES2857773T5 (es) * 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
CN108291216B (zh) * 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
JP7222075B2 (ja) * 2018-09-13 2023-02-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｃ３糸球体症のモデルとしての補体因子ｈ遺伝子ノックアウトラット

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103920142A (zh) * 2005-02-14 2014-07-16 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
CN106062200A (zh) * 2014-02-17 2016-10-26 伦敦国王学院 腺相关病毒载体

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JAMIE WILLOWS ET AL.: "The role of complement in kidney disease", CLINICAL MEDICINE, 31 December 2020 (2020-12-31), pages 156 - 160 *

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
JP2023536008A (ja) 2023-08-22

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Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
TA01 Transfer of patent application right
TA01 Transfer of patent application right

Effective date of registration: 20241121

Address after: London

Applicant after: Xinkona Intellectual Property Holdings (3) Co.,Ltd.

Country or region after: Britain

Applicant after: University OF BRISTOL

Address before: London

Applicant before: SYNCONA MANAGEMENT LLP

Country or region before: Britain

Applicant before: University OF BRISTOL

Applicant before: Prespring Treatment Co.,Ltd.