GB201706394D0 - Gene Therapy - Google Patents

Gene Therapy

Info

Publication number
GB201706394D0
GB201706394D0 GBGB1706394.2A GB201706394A GB201706394D0 GB 201706394 D0 GB201706394 D0 GB 201706394D0 GB 201706394 A GB201706394 A GB 201706394A GB 201706394 D0 GB201706394 D0 GB 201706394D0
Authority
GB
United Kingdom
Prior art keywords
gene therapy
therapy
gene
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.)
Ceased
Application number
GBGB1706394.2A
Other languages
English (en)
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
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 Fondazione Telethon, Ospedale San Raffaele SRL filed Critical Fondazione Telethon
Priority to GBGB1706394.2A priority Critical patent/GB201706394D0/en
Publication of GB201706394D0 publication Critical patent/GB201706394D0/en
Priority to JP2019556865A priority patent/JP7443063B2/ja
Priority to EP18719544.1A priority patent/EP3612237B1/en
Priority to PCT/EP2018/060237 priority patent/WO2018193118A1/en
Priority to HRP20211803TT priority patent/HRP20211803T1/hr
Priority to RS20211471A priority patent/RS62619B1/sr
Priority to US16/606,633 priority patent/US11964027B2/en
Priority to PL18719544T priority patent/PL3612237T3/pl
Priority to ES18719544T priority patent/ES2900560T3/es
Priority to CA3060128A priority patent/CA3060128A1/en
Priority to CN201880038804.7A priority patent/CN110769860B/zh
Priority to SI201830485T priority patent/SI3612237T1/sl
Priority to DK18719544.1T priority patent/DK3612237T3/da
Priority to HUE18719544A priority patent/HUE056570T2/hu
Priority to EP21194123.2A priority patent/EP3973996A1/en
Priority to PT187195441T priority patent/PT3612237T/pt
Priority to IL270032A priority patent/IL270032B2/en
Priority to LTEPPCT/EP2018/060237T priority patent/LT3612237T/lt
Priority to AU2018253958A priority patent/AU2018253958B2/en
Priority to JP2023219262A priority patent/JP2024045128A/ja
Priority to US18/597,488 priority patent/US12485187B2/en
Ceased 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (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)
GBGB1706394.2A 2017-04-21 2017-04-21 Gene Therapy Ceased GB201706394D0 (en)

Priority Applications (21)

Application Number Priority Date Filing Date Title
GBGB1706394.2A GB201706394D0 (en) 2017-04-21 2017-04-21 Gene Therapy
AU2018253958A AU2018253958B2 (en) 2017-04-21 2018-04-20 Gene therapy
CN201880038804.7A CN110769860B (zh) 2017-04-21 2018-04-20 基因疗法
DK18719544.1T DK3612237T3 (da) 2017-04-21 2018-04-20 Genterapi
PCT/EP2018/060237 WO2018193118A1 (en) 2017-04-21 2018-04-20 Gene therapy
HRP20211803TT HRP20211803T1 (hr) 2017-04-21 2018-04-20 Genska terapija
RS20211471A RS62619B1 (sr) 2017-04-21 2018-04-20 Genska terapija
US16/606,633 US11964027B2 (en) 2017-04-21 2018-04-20 Method for improving retroviral transduction and gene editing in hematopoietic stem cells using cyclosporine h (CsH)
PL18719544T PL3612237T3 (pl) 2017-04-21 2018-04-20 Terapia genowa
ES18719544T ES2900560T3 (es) 2017-04-21 2018-04-20 Terapia génica.
CA3060128A CA3060128A1 (en) 2017-04-21 2018-04-20 Cyclosporin h for increasing efficiency of transduction and gene editing of cells by a viral vector
JP2019556865A JP7443063B2 (ja) 2017-04-21 2018-04-20 遺伝子治療
SI201830485T SI3612237T1 (sl) 2017-04-21 2018-04-20 Genska terapija
EP18719544.1A EP3612237B1 (en) 2017-04-21 2018-04-20 Gene therapy
HUE18719544A HUE056570T2 (hu) 2017-04-21 2018-04-20 Génterápia
EP21194123.2A EP3973996A1 (en) 2017-04-21 2018-04-20 Gene therapy
PT187195441T PT3612237T (pt) 2017-04-21 2018-04-20 Terapia genética
IL270032A IL270032B2 (en) 2017-04-21 2018-04-20 Garden care
LTEPPCT/EP2018/060237T LT3612237T (lt) 2017-04-21 2018-04-20 Genų terapija
JP2023219262A JP2024045128A (ja) 2017-04-21 2023-12-26 遺伝子治療
US18/597,488 US12485187B2 (en) 2017-04-21 2024-03-06 Method for improving retroviral transduction and gene editing in hematopoietic stem cells using clyclosporin H and UM171

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
GB201706394D0 true GB201706394D0 (en) 2017-06-07

Family

ID=58795636

Family Applications (1)

Application Number Title Priority Date Filing Date
GBGB1706394.2A Ceased GB201706394D0 (en) 2017-04-21 2017-04-21 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
ES2769270T3 (es) * 2011-09-30 2020-06-25 Bluebird Bio Inc Compuestos para transducción viral mejorada
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
US20260048148A1 (en) 2022-08-11 2026-02-19 Fondazione Telethon Ets (50%) 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

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
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
ATE528406T1 (de) 1999-04-29 2011-10-15 Gbp Ip Llc Verfahren und mittel zur herstellung von sicheren,rekombinanten lentivirusvektoren mit hohem titer
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
EP1549347A1 (en) * 2002-10-09 2005-07-06 Lorantis Limited Modulation of immune function
WO2004098531A2 (en) * 2003-05-05 2004-11-18 Virxsys Corporation Increased transduction using abc transporter substrates and/or inhibitors
WO2012058673A2 (en) * 2010-10-31 2012-05-03 Tocagen Inc. Enhanced cancer treatment and monitoring using recombinant vectors
EP2943203A4 (en) 2013-01-11 2016-08-24 Scripps Research Inst METHOD AND COMPOSITIONS FOR INCREASING THE TRANSDUCTION EFFICIENCY OF RETROVIRAL VECTORS
EP3060670B1 (en) 2013-10-24 2019-07-10 Ospedale San Raffaele S.r.l. Method
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
CN110769860A (zh) 2020-02-07
US20210121579A1 (en) 2021-04-29
EP3612237A1 (en) 2020-02-26
WO2018193118A1 (en) 2018-10-25
JP7443063B2 (ja) 2024-03-05
IL270032A (https=) 2019-12-31
DK3612237T3 (da) 2021-12-06
LT3612237T (lt) 2022-01-25
PT3612237T (pt) 2021-12-02
EP3612237B1 (en) 2021-09-01
SI3612237T1 (sl) 2022-02-28
RS62619B1 (sr) 2021-12-31
CA3060128A1 (en) 2018-10-25
ES2900560T3 (es) 2022-03-17
CN110769860B (zh) 2024-03-08
IL270032B1 (en) 2024-11-01
US11964027B2 (en) 2024-04-23
IL270032B2 (en) 2025-03-01
PL3612237T3 (pl) 2022-02-14
AU2018253958A1 (en) 2019-11-21
US12485187B2 (en) 2025-12-02
AU2018253958B2 (en) 2024-03-07
HRP20211803T1 (hr) 2022-03-04
HUE056570T2 (hu) 2022-02-28
JP2024045128A (ja) 2024-04-02
EP3973996A1 (en) 2022-03-30
US20240350661A1 (en) 2024-10-24
JP2020517253A (ja) 2020-06-18

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Legal Events

Date Code Title Description
AT Applications terminated before publication under section 16(1)