PL3612237T3 - Terapia genowa - Google Patents

Terapia genowa

Info

Publication number
PL3612237T3
PL3612237T3 PL18719544T PL18719544T PL3612237T3 PL 3612237 T3 PL3612237 T3 PL 3612237T3 PL 18719544 T PL18719544 T PL 18719544T PL 18719544 T PL18719544 T PL 18719544T PL 3612237 T3 PL3612237 T3 PL 3612237T3
Authority
PL
Poland
Prior art keywords
gene therapy
therapy
gene
Prior art date
Application number
PL18719544T
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 S.R.L.
Fondazione 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 S.R.L., Fondazione Telethon filed Critical Ospedale San Raffaele S.R.L.
Publication of PL3612237T3 publication Critical patent/PL3612237T3/pl

Links

Classifications

    • 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
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/64Cyclic peptides containing only normal peptide links
    • C07K7/645Cyclosporins; Related peptides
    • 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
    • C12N15/67General methods for enhancing the expression
    • C12N15/69Increasing the copy number of the vector
    • 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
    • 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
    • C12N2740/13011Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6045RNA rev transcr viruses
    • C12N2810/6054Retroviridae
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
    • C12N2810/6081Vectors 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)
PL18719544T 2017-04-21 2018-04-20 Terapia genowa PL3612237T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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
EP18719544.1A EP3612237B1 (en) 2017-04-21 2018-04-20 Gene therapy

Publications (1)

Publication Number Publication Date
PL3612237T3 true PL3612237T3 (pl) 2022-02-14

Family

ID=58795636

Family Applications (1)

Application Number Title Priority Date Filing Date
PL18719544T PL3612237T3 (pl) 2017-04-21 2018-04-20 Terapia genowa

Country Status (18)

Country Link
US (2) US11964027B2 (pl)
EP (2) EP3973996A1 (pl)
JP (2) JP7443063B2 (pl)
CN (1) CN110769860B (pl)
AU (1) AU2018253958B2 (pl)
CA (1) CA3060128A1 (pl)
DK (1) DK3612237T3 (pl)
ES (1) ES2900560T3 (pl)
GB (1) GB201706394D0 (pl)
HR (1) HRP20211803T1 (pl)
HU (1) HUE056570T2 (pl)
IL (1) IL270032B2 (pl)
LT (1) LT3612237T (pl)
PL (1) PL3612237T3 (pl)
PT (1) PT3612237T (pl)
RS (1) RS62619B1 (pl)
SI (1) SI3612237T1 (pl)
WO (1) WO2018193118A1 (pl)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3269802B1 (en) * 2011-09-30 2019-10-23 Bluebird Bio, Inc. Compounds for improved viral transduction
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
JP2025530652A (ja) 2022-08-11 2025-09-17 フォンダジオン テレソン イーティーエス 遺伝子治療
WO2024079644A1 (en) 2022-10-11 2024-04-18 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

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
WO1998005635A1 (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
WO1998017815A1 (en) 1996-10-17 1998-04-30 Oxford Biomedica (Uk) Limited Retroviral vectors
WO2000066759A1 (en) 1999-04-29 2000-11-09 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
WO2003012054A2 (en) 2001-08-02 2003-02-13 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
US20050123514A1 (en) * 2003-05-05 2005-06-09 Brian Davis Increased transduction using ABC transporter substrates and/or inhibitors
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
CA2927712A1 (en) 2013-10-24 2015-04-30 Ospedale San Raffaele S.R.L. Method for the genetic modification of haematopoietic stem and progenitor cells (hspcs) and the use of genetically modified hspcs in gene therapy
GB201407322D0 (en) * 2014-04-25 2014-06-11 Ospedale San Raffaele Gene therapy
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells

Also Published As

Publication number Publication date
JP7443063B2 (ja) 2024-03-05
LT3612237T (lt) 2022-01-25
US12485187B2 (en) 2025-12-02
JP2024045128A (ja) 2024-04-02
CN110769860A (zh) 2020-02-07
WO2018193118A1 (en) 2018-10-25
JP2020517253A (ja) 2020-06-18
US20210121579A1 (en) 2021-04-29
SI3612237T1 (sl) 2022-02-28
US20240350661A1 (en) 2024-10-24
AU2018253958A1 (en) 2019-11-21
PT3612237T (pt) 2021-12-02
IL270032B1 (en) 2024-11-01
CN110769860B (zh) 2024-03-08
DK3612237T3 (da) 2021-12-06
US11964027B2 (en) 2024-04-23
EP3973996A1 (en) 2022-03-30
RS62619B1 (sr) 2021-12-31
HRP20211803T1 (hr) 2022-03-04
HUE056570T2 (hu) 2022-02-28
CA3060128A1 (en) 2018-10-25
EP3612237A1 (en) 2020-02-26
AU2018253958B2 (en) 2024-03-07
EP3612237B1 (en) 2021-09-01
IL270032A (pl) 2019-12-31
ES2900560T3 (es) 2022-03-17
GB201706394D0 (en) 2017-06-07
IL270032B2 (en) 2025-03-01

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