CA3183830A1 - Vector - Google Patents

Vector

Info

Publication number
CA3183830A1
CA3183830A1 CA3183830A CA3183830A CA3183830A1 CA 3183830 A1 CA3183830 A1 CA 3183830A1 CA 3183830 A CA3183830 A CA 3183830A CA 3183830 A CA3183830 A CA 3183830A CA 3183830 A1 CA3183830 A1 CA 3183830A1
Authority
CA
Canada
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
CA3183830A
Other languages
English (en)
French (fr)
Inventor
Alan William GRIFFITH
Lukas STANCZUK
Valeryia KUZMUK
Chris HOLLOWOOD
Dominic SCHMIDT
Moin Ahson SALEEM-UDDIN
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
Original Assignee
University of Bristol
Syncona Investment Management Ltd
Purespring Therapeutics Ltd
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 University of Bristol, Syncona Investment Management Ltd, Purespring Therapeutics Ltd filed Critical University of Bristol
Publication of CA3183830A1 publication Critical patent/CA3183830A1/en
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)
  • 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)
CA3183830A 2020-06-30 2021-06-30 Vector Pending CA3183830A1 (en)

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 (1)

Publication Number Publication Date
CA3183830A1 true CA3183830A1 (en) 2022-01-06

Family

ID=71949697

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3183830A Pending CA3183830A1 (en) 2020-06-30 2021-06-30 Vector

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

Family Cites Families (8)

* 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
US20050169914A1 (en) * 2003-08-01 2005-08-04 Colgan Sean P. Novel functions for decay accelerating factor (DAF) in inflammation
US7745389B2 (en) * 2005-02-14 2010-06-29 University Of Iowa Research Foundation Methods for treatment of age-related macular degeneration
EP2730282A1 (en) * 2007-11-08 2014-05-14 The General Hospital Corporation Methods and compositions for the treatment of proteinuric diseases
EP3795581A3 (en) * 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
EP3359663A4 (en) * 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
US12653168B2 (en) * 2018-09-13 2026-06-16 Regeneron Pharmaceuticals, Inc. Complement factor H gene knockout rat as a model of C3 glomerulopathy

Also Published As

Publication number Publication date
AU2021300616A1 (en) 2023-01-19
EP4172345A1 (en) 2023-05-03
IL299505A (en) 2023-02-01
US20230175016A1 (en) 2023-06-08
JP2023536008A (ja) 2023-08-22
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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