HUE056570T2 - Génterápia - Google Patents

Génterápia

Info

Publication number
HUE056570T2
HUE056570T2 HUE18719544A HUE18719544A HUE056570T2 HU E056570 T2 HUE056570 T2 HU E056570T2 HU E18719544 A HUE18719544 A HU E18719544A HU E18719544 A HUE18719544 A HU E18719544A HU E056570 T2 HUE056570 T2 HU E056570T2
Authority
HU
Hungary
Prior art keywords
gene therapy
therapy
gene
Prior art date
Application number
HUE18719544A
Other languages
English (en)
Inventor
Anna Christina Kajaste-Rudnitski
Carolina Petrillo
Bernhard Rudolf Gentner
Luigi Naldini
Pietro Genovese
Giulia Schiroli
Original Assignee
Ospedale San Raffaele Srl
Fond Telethon
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 Ospedale San Raffaele Srl, Fond Telethon filed Critical Ospedale San Raffaele Srl
Publication of HUE056570T2 publication Critical patent/HUE056570T2/hu

Links

Classifications

    • 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
    • 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
    • A61K48/0083—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)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (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)
HUE18719544A 2017-04-21 2018-04-20 Génterápia HUE056570T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1706394.2A GB201706394D0 (en) 2017-04-21 2017-04-21 Gene Therapy

Publications (1)

Publication Number Publication Date
HUE056570T2 true HUE056570T2 (hu) 2022-02-28

Family

ID=58795636

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE18719544A HUE056570T2 (hu) 2017-04-21 2018-04-20 Génterápia

Country Status (17)

Country Link
US (1) US11964027B2 (hu)
EP (2) EP3973996A1 (hu)
JP (2) JP7443063B2 (hu)
CN (1) CN110769860B (hu)
AU (1) AU2018253958B2 (hu)
CA (1) CA3060128A1 (hu)
DK (1) DK3612237T3 (hu)
ES (1) ES2900560T3 (hu)
GB (1) GB201706394D0 (hu)
HR (1) HRP20211803T1 (hu)
HU (1) HUE056570T2 (hu)
LT (1) LT3612237T (hu)
PL (1) PL3612237T3 (hu)
PT (1) PT3612237T (hu)
RS (1) RS62619B1 (hu)
SI (1) SI3612237T1 (hu)
WO (1) WO2018193118A1 (hu)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102011532B1 (ko) * 2011-09-30 2019-08-16 블루버드 바이오, 인코포레이티드. 개선된 바이러스 형질도입을 위한 화합물
WO2023077107A1 (en) * 2021-10-29 2023-05-04 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
WO2024079644A1 (en) 2022-10-11 2024-04-18 Fondazione Telethon Ets 3d cell culture methods

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5723315A (en) 1996-08-23 1998-03-03 Genetics Institute, Inc. Secreted proteins and polynucleotides encoding them
CA2263154A1 (en) 1996-08-07 1998-02-12 Darwin Discovery Limited Hydroxamic and carboxylic acid derivatives having mmp and tnf inhibitory activity
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
CN1195863C (zh) 1996-10-17 2005-04-06 牛津生物医学（英国）有限公司 逆转录病毒载体
EP1171624B1 (en) 1999-04-29 2007-07-25 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
IL160132A0 (en) 2001-08-02 2004-06-20 Inst Clayton De La Rech Methods and compositions relating to improved lentiviral vector production systems
AU2003269268A1 (en) 2002-10-09 2004-05-04 Lorantis Limited Modulation of immune function
DE602004030105D1 (de) 2003-05-05 2010-12-30 Virxsys Corp Erhöhte transduktion mit abc-transportersubstratenhemmern
EP2632491A4 (en) * 2010-10-31 2014-10-01 Tocagen Inc ENHANCED TREATMENT AND MONITORING OF CANCER USING RECOMBINANT VECTORS
WO2014109728A1 (en) 2013-01-11 2014-07-17 The Scripps Research Institute Methods and compositions for enchancing transduction efficiency of retroviral vectors
EP3613859A1 (en) 2013-10-24 2020-02-26 Ospedale San Raffaele S.r.l. Method
GB201407322D0 (en) * 2014-04-25 2014-06-11 Ospedale San Raffaele Gene therapy
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells

Also Published As

Publication number Publication date
GB201706394D0 (en) 2017-06-07
EP3612237A1 (en) 2020-02-26
HRP20211803T1 (hr) 2022-03-04
PL3612237T3 (pl) 2022-02-14
RS62619B1 (sr) 2021-12-31
US11964027B2 (en) 2024-04-23
JP2024045128A (ja) 2024-04-02
US20210121579A1 (en) 2021-04-29
WO2018193118A1 (en) 2018-10-25
CA3060128A1 (en) 2018-10-25
CN110769860A (zh) 2020-02-07
AU2018253958A1 (en) 2019-11-21
JP7443063B2 (ja) 2024-03-05
EP3973996A1 (en) 2022-03-30
CN110769860B (zh) 2024-03-08
PT3612237T (pt) 2021-12-02
DK3612237T3 (da) 2021-12-06
EP3612237B1 (en) 2021-09-01
ES2900560T3 (es) 2022-03-17
JP2020517253A (ja) 2020-06-18
AU2018253958B2 (en) 2024-03-07
LT3612237T (lt) 2022-01-25
SI3612237T1 (sl) 2022-02-28

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