AU2018253958B2 - Gene therapy - Google Patents

Gene therapy Download PDF

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Publication number
AU2018253958B2
AU2018253958B2 AU2018253958A AU2018253958A AU2018253958B2 AU 2018253958 B2 AU2018253958 B2 AU 2018253958B2 AU 2018253958 A AU2018253958 A AU 2018253958A AU 2018253958 A AU2018253958 A AU 2018253958A AU 2018253958 B2 AU2018253958 B2 AU 2018253958B2
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AU
Australia
Prior art keywords
cells
csh
moi
csa
vector
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Active
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AU2018253958A
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English (en)
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AU2018253958A1 (en
Inventor
Pietro Genovese
Bernhard Rudolf Gentner
Anna Christina KAJASTE-RUDNITSKI
Luigi Naldini
Carolina PETRILLO
Giulia SCHIROLI
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.)
Fondazione Telethon
Ospedale San Raffaele SRL
Original Assignee
Fondazione Telethon
Ospedale San Raffaele SRL
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Publication of AU2018253958A1 publication Critical patent/AU2018253958A1/en
Assigned to OSPEDALE SAN RAFFAELE S.R.L, FONDAZIONE TELETHON ETS reassignment OSPEDALE SAN RAFFAELE S.R.L Amend patent request/document other than specification (104) Assignors: FONDAZIONE TELETHON, OSPEDALE SAN RAFFAELE S.R.L
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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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0083—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 administration regime
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/64—Cyclic peptides containing only normal peptide links
    • C07K7/645—Cyclosporins; Related peptides
    • 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
    • C12N15/67—General methods for enhancing the expression
    • C12N15/69—Increasing the copy number of the 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6045—RNA rev transcr viruses
    • C12N2810/6054—Retroviridae
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
    • C12N2810/6081—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses rhabdoviridae, e.g. VSV

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2018253958A 2017-04-21 2018-04-20 Gene therapy Active AU2018253958B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1706394.2 2017-04-21
GBGB1706394.2A GB201706394D0 (en) 2017-04-21 2017-04-21 Gene Therapy
PCT/EP2018/060237 WO2018193118A1 (en) 2017-04-21 2018-04-20 Gene therapy

Publications (2)

Publication Number Publication Date
AU2018253958A1 AU2018253958A1 (en) 2019-11-21
AU2018253958B2 true AU2018253958B2 (en) 2024-03-07

Family

ID=58795636

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018253958A Active AU2018253958B2 (en) 2017-04-21 2018-04-20 Gene therapy

Country Status (18)

Country Link
US (2) US11964027B2 (https=)
EP (2) EP3612237B1 (https=)
JP (2) JP7443063B2 (https=)
CN (1) CN110769860B (https=)
AU (1) AU2018253958B2 (https=)
CA (1) CA3060128A1 (https=)
DK (1) DK3612237T3 (https=)
ES (1) ES2900560T3 (https=)
GB (1) GB201706394D0 (https=)
HR (1) HRP20211803T1 (https=)
HU (1) HUE056570T2 (https=)
IL (1) IL270032B2 (https=)
LT (1) LT3612237T (https=)
PL (1) PL3612237T3 (https=)
PT (1) PT3612237T (https=)
RS (1) RS62619B1 (https=)
SI (1) SI3612237T1 (https=)
WO (1) WO2018193118A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2760994T (pt) * 2011-09-30 2017-08-24 Bluebird Bio Inc Compostos para melhor transdução viral
US20250230511A1 (en) * 2021-10-29 2025-07-17 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
GB202208550D0 (en) * 2022-06-10 2022-07-27 Ospedale San Raffaele Srl Cyclosporin h and uses thereof
GB202209098D0 (en) * 2022-06-21 2022-08-10 Ucl Business Ltd Cyclosporine analogues
WO2024033802A2 (en) 2022-08-11 2024-02-15 Fondazione Telethon Ets Gene therapy
EP4602153A1 (en) 2022-10-11 2025-08-20 Fondazione Telethon ETS 3d cell culture methods
IT202300011790A1 (it) 2023-06-08 2024-12-08 Fond Telethon Ets Protocolli di manipolazione genica in cellule immunitarie
IT202300022191A1 (it) 2023-10-24 2025-04-24 Fond Telethon Ets Mezzi e metodi per la modifica genetica di uba1
EP4640830A1 (en) 2024-04-23 2025-10-29 Fondazione Telethon ETS Means and methods for safe and efficient gene editing in cells

Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2015162594A2 (en) * 2014-04-25 2015-10-29 Ospedale San Raffaele S.R.L. Gene therapy

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US5723315A (en) 1996-08-23 1998-03-03 Genetics Institute, Inc. Secreted proteins and polynucleotides encoding them
DE69729007T2 (de) 1996-08-07 2005-04-07 Darwin Discovery Ltd., Slough Hydroxamsäure- und carbonsäure-derivate mit mmp und tnf hemmender wirkung
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
PL332875A1 (en) 1996-10-17 1999-10-25 Oxford Biomedica Ltd Retroviral vectors
EP1849873B1 (en) 1999-04-29 2011-10-12 Gbp Ip, Llc Method and means for producing high titer, safe, recombinant lentivirus vectors
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
WO2004032969A1 (en) 2002-10-09 2004-04-22 Lorantis Limited Modulation of immune function
ATE488252T1 (de) * 2003-05-05 2010-12-15 Virxsys Corp Erhöhte transduktion mit abc- transportersubstratenhemmern
WO2012058673A2 (en) 2010-10-31 2012-05-03 Tocagen Inc. Enhanced cancer treatment and monitoring using recombinant vectors
US20150352228A1 (en) 2013-01-11 2015-12-10 Bruce TORBET Methods and compositions for enhancing transduction efficiency of retroviral vectors
ES2747951T3 (es) 2013-10-24 2020-03-12 Ospedale San Raffaele Srl Método
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells

Patent Citations (1)

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Non-Patent Citations (3)

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Bouchard, M. J., et al., July 2003, Activation and inhibition of cellular calcium and tyrosine kinase signaling pathways identify targets of the HBx protein involved in hepatitis B virus replication, J. Virol. 77(14):7713-7719. *
PETRILLO, C. ET AL: "Cyclosporin A and Rapamycin Relieve Distinct Lentiviral Restriction Blocks in Hematopoietic Stem and Progenitor Cells", MOLECULAR THERAPY. 2014, vol. 23, no. 2, pages 352 - 362 *
ZONARI, E. ET AL: "Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications", Blood. 2 December 2016, Vol. 128, No. 22: 4707, 801. Gene Therapy and Transfer: Poster III *

Also Published As

Publication number Publication date
DK3612237T3 (da) 2021-12-06
JP2020517253A (ja) 2020-06-18
IL270032A (https=) 2019-12-31
CN110769860A (zh) 2020-02-07
RS62619B1 (sr) 2021-12-31
US20210121579A1 (en) 2021-04-29
JP7443063B2 (ja) 2024-03-05
CA3060128A1 (en) 2018-10-25
US12485187B2 (en) 2025-12-02
EP3612237A1 (en) 2020-02-26
EP3612237B1 (en) 2021-09-01
US20240350661A1 (en) 2024-10-24
PT3612237T (pt) 2021-12-02
IL270032B2 (en) 2025-03-01
ES2900560T3 (es) 2022-03-17
WO2018193118A1 (en) 2018-10-25
PL3612237T3 (pl) 2022-02-14
AU2018253958A1 (en) 2019-11-21
JP2024045128A (ja) 2024-04-02
US11964027B2 (en) 2024-04-23
LT3612237T (lt) 2022-01-25
SI3612237T1 (sl) 2022-02-28
IL270032B1 (en) 2024-11-01
HRP20211803T1 (hr) 2022-03-04
HUE056570T2 (hu) 2022-02-28
CN110769860B (zh) 2024-03-08
EP3973996A1 (en) 2022-03-30
GB201706394D0 (en) 2017-06-07

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