SG11202100025QA - Methods for gene modification of hematopoietic cells - Google Patents

Methods for gene modification of hematopoietic cells

Info

Publication number
SG11202100025QA
SG11202100025QA SG11202100025QA SG11202100025QA SG11202100025QA SG 11202100025Q A SG11202100025Q A SG 11202100025QA SG 11202100025Q A SG11202100025Q A SG 11202100025QA SG 11202100025Q A SG11202100025Q A SG 11202100025QA SG 11202100025Q A SG11202100025Q A SG 11202100025QA
Authority
SG
Singapore
Prior art keywords
methods
hematopoietic cells
gene modification
gene
modification
Prior art date
Application number
SG11202100025QA
Inventor
Brian Beard
Gaurav D Shah
Roncero Juan Antonio Bueren
Sanz Jose Carlos Segovia
Galdo Paula Rio
Ordonez Susana Navarro
Novoa Elena Almarza
Bustamante Oscar Quintana
Nunez Cristina Mesa
Kenneth Law
Kinnari Patel
Original Assignee
Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas
Consorcio Centro De Investigacion Biomedica En Red M P
Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz
Rocket Pharmaceuticals 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 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas, Consorcio Centro De Investigacion Biomedica En Red M P, Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz, Rocket Pharmaceuticals Ltd filed Critical Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas
Publication of SG11202100025QA publication Critical patent/SG11202100025QA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2500/00Specific components of cell culture medium
    • C12N2500/50Soluble polymers, e.g. polyethyleneglycol [PEG]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Hematology (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11202100025QA 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells SG11202100025QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862712146P 2018-07-30 2018-07-30
PCT/US2019/044237 WO2020028430A1 (en) 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells

Publications (1)

Publication Number Publication Date
SG11202100025QA true SG11202100025QA (en) 2021-01-28

Family

ID=69232048

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202100025QA SG11202100025QA (en) 2018-07-30 2019-07-30 Methods for gene modification of hematopoietic cells

Country Status (12)

Country Link
US (1) US20210290685A1 (en)
EP (1) EP3830248A4 (en)
JP (1) JP2021532776A (en)
KR (1) KR20210046004A (en)
CN (1) CN112888777A (en)
AU (1) AU2019315438A1 (en)
BR (1) BR112021001757A2 (en)
CA (1) CA3106241A1 (en)
IL (1) IL280465A (en)
MX (1) MX2021001292A (en)
SG (1) SG11202100025QA (en)
WO (1) WO2020028430A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112022021862A2 (en) * 2020-05-27 2023-02-28 Univ Zuerich INNOVATIVE TRANSDUCTION IMPROVEERS AND THEIR USES
CN116194154A (en) 2020-08-07 2023-05-30 太空飞船七有限责任公司 PLAKOPHILIN-2 (PKP 2) gene therapy using AAV vectors
CN114763563B (en) * 2021-01-12 2024-04-30 深圳华大临床检验中心 Method for improving slow virus infection efficiency

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2744603C2 (en) * 2016-02-12 2021-03-11 Блубёрд Био, Инк. Compositions increasing the number of copies of the vector (vcn), and methods of using
US20190284533A1 (en) * 2016-02-12 2019-09-19 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same
WO2017218519A1 (en) * 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
US20200071721A1 (en) * 2016-12-06 2020-03-05 Bluebird Bio, Inc. Gene therapy for mucopolysaccharidosis, type ii
CN110214182A (en) * 2016-12-06 2019-09-06 蓝鸟生物公司 Gene therapy for I type mucopolysaccharidosis

Also Published As

Publication number Publication date
IL280465A (en) 2021-03-25
AU2019315438A1 (en) 2021-01-28
EP3830248A4 (en) 2022-05-04
EP3830248A1 (en) 2021-06-09
BR112021001757A2 (en) 2021-05-11
WO2020028430A1 (en) 2020-02-06
MX2021001292A (en) 2021-07-15
CN112888777A (en) 2021-06-01
CA3106241A1 (en) 2020-02-06
US20210290685A1 (en) 2021-09-23
KR20210046004A (en) 2021-04-27
JP2021532776A (en) 2021-12-02

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