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DK3368052T3 - Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation - Google Patents

Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation Download PDF

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Publication number
DK3368052T3
DK3368052T3 DK16860828.9T DK16860828T DK3368052T3 DK 3368052 T3 DK3368052 T3 DK 3368052T3 DK 16860828 T DK16860828 T DK 16860828T DK 3368052 T3 DK3368052 T3 DK 3368052T3
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DK
Denmark
Prior art keywords
loss
reduce
stem cells
cells during
gene manipulation
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Application number
DK16860828.9T
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English (en)
Inventor
Punam Malik
Marie-Dominique Filippi
Original Assignee
Childrens Hospital Med Ct
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Publication of DK3368052T3 publication Critical patent/DK3368052T3/da

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    • 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
    • A61K35/14—Blood; Artificial blood
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0271—Chimeric vertebrates, e.g. comprising exogenous cells
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
    • 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
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
    • C12N2501/72—Transferases [EC 2.]
    • C12N2501/727—Kinases (EC 2.7.)
    • 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
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Environmental Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Biophysics (AREA)
  • Animal Husbandry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
DK16860828.9T 2015-10-27 2016-10-27 Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation DK3368052T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562246964P 2015-10-27 2015-10-27
PCT/US2016/059204 WO2017075274A1 (en) 2015-10-27 2016-10-27 Use of mapk inhibitors to reduce loss of hematopoietic stem cells during ex vivo culture and/or genetic manipulation

Publications (1)

Publication Number Publication Date
DK3368052T3 true DK3368052T3 (da) 2022-03-14

Family

ID=58631986

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16860828.9T DK3368052T3 (da) 2015-10-27 2016-10-27 Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation

Country Status (7)

Country Link
US (1) US20180325947A1 (da)
EP (1) EP3368052B1 (da)
CA (1) CA3003433A1 (da)
DK (1) DK3368052T3 (da)
ES (1) ES2908041T3 (da)
HK (1) HK1258782A1 (da)
WO (1) WO2017075274A1 (da)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10845363B2 (en) * 2012-11-27 2020-11-24 DeePath Medical Diagnostics, Inc. Cell-specific signaling biomarker analysis by high parameter cytometry; sample processing, assay set-up, method, analysis
SG11201901199WA (en) * 2016-08-18 2019-03-28 Nat Univ Singapore Substituted azole derivatives for generation, proliferation and differentiation of hematopoietic stem and progenitor cells
MX2023011658A (es) * 2021-03-31 2023-12-14 Garuda Therapeutics Inc Linajes hematopoyeticos derivados de celulas madre pluripotentes.
GB202114972D0 (en) * 2021-10-19 2021-12-01 Ospedale San Raffaele Srl Gene therapy
WO2024073606A1 (en) * 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies

Family Cites Families (22)

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Publication number Priority date Publication date Assignee Title
US5254678A (en) 1987-12-15 1993-10-19 Gene Shears Pty. Limited Ribozymes
JPH10512264A (ja) 1995-01-12 1998-11-24 スミスクライン・ビーチャム・コーポレイション 新規化合物
US6608060B1 (en) 1996-12-18 2003-08-19 Vertex Pharmaceuticals Incorporated Inhibitors of p38
US6147080A (en) 1996-12-18 2000-11-14 Vertex Pharmaceuticals Incorporated Inhibitors of p38
CA2288787A1 (en) 1997-05-22 1998-11-26 G.D. Searle And Co. Pyrazole derivatives as p38 kinase inhibitors
US6093742A (en) 1997-06-27 2000-07-25 Vertex Pharmaceuticals, Inc. Inhibitors of p38
KR100378937B1 (ko) 1997-10-20 2003-05-09 에프. 호프만-라 로슈 아게 바이사이클릭 키나아제 억제제
US6316466B1 (en) 1998-05-05 2001-11-13 Syntex (U.S.A.) Llc Pyrazole derivatives P-38 MAP kinase inhibitors
MY132496A (en) 1998-05-11 2007-10-31 Vertex Pharma Inhibitors of p38
JP2002517486A (ja) 1998-06-12 2002-06-18 バーテックス ファーマシューティカルズ インコーポレイテッド ｐ３８のインヒビター
AR023659A1 (es) 1998-09-18 2002-09-04 Vertex Pharma Un compuesto inhibidor de p38, una composicion farmaceutica que lo comprende y el uso de dicha composicion en el tratamiento y prevencion de estados patologicos
EP1114039A1 (en) 1998-09-18 2001-07-11 Vertex Pharmaceuticals Incorporated INHIBITORS OF p38
US6509361B1 (en) 1999-05-12 2003-01-21 Pharmacia Corporation 1,5-Diaryl substituted pyrazoles as p38 kinase inhibitors
US6759410B1 (en) 1999-11-23 2004-07-06 Smithline Beecham Corporation 3,4-dihydro-(1H)-quinazolin-2-ones and their use as CSBP/p38 kinase inhibitors
ES2279837T3 (es) 2000-11-20 2007-09-01 Scios Inc. Inhibidores de tipo piperidina/piperazina de la quinasa p38.
EP1423378B1 (en) 2001-08-30 2005-11-09 F. Hoffmann-La Roche Ag Aminopyrrole compounds as antiinflammatory agents
MXPA04009585A (es) 2002-04-03 2005-01-11 Hoffmann La Roche Compuestos imidazo fusionados.
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
WO2012141971A2 (en) * 2011-04-05 2012-10-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for rejuvenation and expansion of stem cells
US9428732B2 (en) * 2011-12-05 2016-08-30 Primorigen Biosciences, Inc. Compositions and methods for differentiating pluripotent stem cells into primitive blood cells and uses thereof
SG10201606959PA (en) * 2012-02-24 2016-09-29 Hutchinson Fred Cancer Res Compositions and methods for the treatment of hemoglobinopathies
CA2938419A1 (en) 2014-01-30 2015-08-06 Children's Hospital Medical Center An improved fetal hemoglobin for genetic correction of sickle cell disease

Also Published As

Publication number Publication date
CA3003433A1 (en) 2017-05-04
EP3368052B1 (en) 2021-12-08
HK1258782A1 (zh) 2019-11-22
ES2908041T3 (es) 2022-04-27
WO2017075274A1 (en) 2017-05-04
EP3368052A1 (en) 2018-09-05
US20180325947A1 (en) 2018-11-15
EP3368052A4 (en) 2019-07-17

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