AU2021300616A1 - Vector - Google Patents

Vector Download PDF

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Publication number
AU2021300616A1
AU2021300616A1 AU2021300616A AU2021300616A AU2021300616A1 AU 2021300616 A1 AU2021300616 A1 AU 2021300616A1 AU 2021300616 A AU2021300616 A AU 2021300616A AU 2021300616 A AU2021300616 A AU 2021300616A AU 2021300616 A1 AU2021300616 A1 AU 2021300616A1
Authority
AU
Australia
Prior art keywords
promoter
viral vector
sequence identity
seq
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
AU2021300616A
Other languages
English (en)
Inventor
Alan William GRIFFITH
Chris HOLLOWOOD
Valeryia KUZMUK
Moin Ahson SALEEM-UDDIN
Dominic SCHMIDT
Lukas STANCZUK
Gavin Iain Welsh
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.)
Syncona Ip Holdco 3 Ltd
University of Bristol
Original Assignee
Syncona Ip Holdco 3 Ltd
University of Bristol
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 Syncona Ip Holdco 3 Ltd, University of Bristol filed Critical Syncona Ip Holdco 3 Ltd
Publication of AU2021300616A1 publication Critical patent/AU2021300616A1/en
Assigned to THE UNIVERSITY OF BRISTOL, SYNCONA IP HOLDCO (3) LIMITED reassignment THE UNIVERSITY OF BRISTOL Request for Assignment Assignors: PURESPRING THERAPEUTICS LIMITED, Syncona Investment Management Limited, THE UNIVERSITY OF BRISTOL
Pending legal-status Critical Current

Links

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
    • C12N15/63—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

Landscapes

  • 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)
AU2021300616A 2020-06-30 2021-06-30 Vector Pending AU2021300616A1 (en)

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
AU2021300616A1 true AU2021300616A1 (en) 2023-01-19

Family

ID=71949697

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021300616A Pending AU2021300616A1 (en) 2020-06-30 2021-06-30 Vector

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

Family Cites Families (8)

* 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
EP2357254A1 (en) * 2005-02-14 2011-08-17 University of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
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
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
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

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
US20230175016A1 (en) 2023-06-08
BR112022026521A2 (pt) 2023-01-31
WO2022003357A1 (en) 2022-01-06
JP2023536008A (ja) 2023-08-22
CN115997025A (zh) 2023-04-21

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Legal Events

Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: THE UNIVERSITY OF BRISTOL

Free format text: FORMER APPLICANT(S): SYNCONA INVESTMENT MANAGEMENT LIMITED; THE UNIVERSITY OF BRISTOL; PURESPRING THERAPEUTICS LIMITED

Owner name: SYNCONA IP HOLDCO (3) LIMITED

Free format text: FORMER APPLICANT(S): SYNCONA INVESTMENT MANAGEMENT LIMITED; THE UNIVERSITY OF BRISTOL; PURESPRING THERAPEUTICS LIMITED