DK3356524T3 - Forøget genlevering til naturlige dræberceller, hæmatopoietiske stamceller og makrofager - Google Patents

Forøget genlevering til naturlige dræberceller, hæmatopoietiske stamceller og makrofager Download PDF

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Publication number
DK3356524T3
DK3356524T3 DK16852663.0T DK16852663T DK3356524T3 DK 3356524 T3 DK3356524 T3 DK 3356524T3 DK 16852663 T DK16852663 T DK 16852663T DK 3356524 T3 DK3356524 T3 DK 3356524T3
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Denmark
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macrophage
hematopoietic stem
natural killer
cells
stem cells
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DK16852663.0T
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English (en)
Inventor
Evren Alici
Tolga Sutlu
Adil Duru
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Vycellix Inc
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Publication of DK3356524T3 publication Critical patent/DK3356524T3/da

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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/425—Thiazoles
    • A61K31/427—Thiazoles not condensed and containing further heterocyclic rings
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5365—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
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    • A61K40/00—Cellular immunotherapy
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    • A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/17—Monocytes; Macrophages
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/24—Antigen-presenting cells [APC]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K40/46—Viral antigens
    • 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
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P35/02—Antineoplastic agents specific for leukemia
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    • 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
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    • 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
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    • C12N5/0602—Vertebrate cells
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    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0646—Natural killers cells [NK], NKT cells
    • A—HUMAN NECESSITIES
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    • C12N2740/00—Reverse transcribing RNA viruses
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biochemistry (AREA)
  • Microbiology (AREA)
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  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Oncology (AREA)
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DK16852663.0T 2015-09-30 2016-09-30 Forøget genlevering til naturlige dræberceller, hæmatopoietiske stamceller og makrofager DK3356524T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562235427P 2015-09-30 2015-09-30
PCT/US2016/054618 WO2017059177A2 (en) 2015-09-30 2016-09-30 Enhanced gene delivery to natural killer cells, hematopoietic stem cells and macrophages

Publications (1)

Publication Number Publication Date
DK3356524T3 true DK3356524T3 (da) 2021-11-15

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DK16852663.0T DK3356524T3 (da) 2015-09-30 2016-09-30 Forøget genlevering til naturlige dræberceller, hæmatopoietiske stamceller og makrofager

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Country Link
US (1) US12533338B2 (da)
EP (1) EP3356524B1 (da)
JP (1) JP7034065B2 (da)
KR (1) KR102907580B1 (da)
CN (1) CN108291221B (da)
AU (2) AU2016331082B2 (da)
CA (1) CA2999891A1 (da)
DK (1) DK3356524T3 (da)
ES (1) ES2897599T3 (da)
HU (1) HUE056374T2 (da)
IL (1) IL258342B2 (da)
PT (1) PT3356524T (da)
WO (1) WO2017059177A2 (da)

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EP3215623A4 (en) * 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
WO2016196256A2 (en) 2015-06-04 2016-12-08 University Of North Carolina At Greensboro Non-aromatic difluoro analogues of resorcylic acid lactones
JP7086928B2 (ja) 2016-07-25 2022-06-20 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 改変されたナチュラルキラー細胞を生成する方法および使用方法
US12516292B2 (en) 2016-07-25 2026-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methods of producing modified natural killer cells and methods of use
CN109722437B (zh) * 2018-12-29 2020-01-07 广州百暨基因科技有限公司 一种通用型car-t细胞及其制备方法和用途
US20230272431A1 (en) * 2020-07-03 2023-08-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Methods and compositions for editing the b2m locus in b cells
CN114045265A (zh) * 2021-11-17 2022-02-15 深圳知因细胞生物科技有限公司 一种慢病毒体外转染人造血干细胞的试剂和试剂盒
CN115975922B (zh) * 2022-12-15 2024-06-28 杭州师范大学 Bx795在制备nk-92细胞激活剂上的应用

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JP2002522513A (ja) 1998-08-11 2002-07-23 アメリカ合衆国 哺乳動物細胞を形質導入する方法およびそれに関連する産物
JP2003509374A (ja) 1999-09-14 2003-03-11 チルドレンズ メディカル センター コーポレーション 骨髄細胞を用いる筋ジストロフィーの治療方法
MX2007010008A (es) * 2005-02-16 2008-01-18 Lentigen Corp Vectores lentivirales y su uso.
WO2008018517A1 (en) 2006-08-09 2008-02-14 Zeria Pharmaceutical Co., Ltd. Therapeutic and/or preventive agent for disease accompanied by cell overgrowth, and polynucleotide useful as the active ingredient
US20130071414A1 (en) * 2011-04-27 2013-03-21 Gianpietro Dotti Engineered cd19-specific t lymphocytes that coexpress il-15 and an inducible caspase-9 based suicide gene for the treatment of b-cell malignancies
US20140243403A1 (en) * 2011-06-03 2014-08-28 The General Hospital Corporation Treating colorectal, pancreatic, and lung cancer
WO2014127026A1 (en) 2013-02-12 2014-08-21 National Jewish Health Methods to identify and treat subjects having corticosteroid-resistant inflammatory diseases
CA2905352A1 (en) 2013-03-14 2014-09-25 Bellicum Pharmaceuticals, Inc. Methods for controlling t cell proliferation
US20160082015A1 (en) 2013-04-18 2016-03-24 President And Fellows Of Harvard College Methods, compositions and kits for promoting motor neuron survival and treating and diagnosing neurodegenerative disorders
CN105722508A (zh) 2013-10-07 2016-06-29 莫尔研究应用有限公司 增生性玻璃体视网膜病变的治疗

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EP3356524B1 (en) 2021-08-18
AU2023203314B2 (en) 2025-09-11
AU2016331082B2 (en) 2023-12-07
US12533338B2 (en) 2026-01-27
EP3356524A4 (en) 2019-07-31
JP2018535661A (ja) 2018-12-06
KR20180053757A (ko) 2018-05-23
AU2016331082A1 (en) 2018-04-19
WO2017059177A3 (en) 2017-07-20
EP3356524A2 (en) 2018-08-08
PT3356524T (pt) 2021-11-17
BR112018006026A2 (pt) 2018-11-06
ES2897599T3 (es) 2022-03-01
AU2023203314A1 (en) 2023-06-22
CN108291221A (zh) 2018-07-17
IL258342A (en) 2018-05-31
WO2017059177A2 (en) 2017-04-06
JP7034065B2 (ja) 2022-03-11
CA2999891A1 (en) 2017-04-06
CN108291221B (zh) 2023-01-24
IL258342B2 (en) 2023-05-01
US20190262309A1 (en) 2019-08-29
HUE056374T2 (hu) 2022-02-28
IL258342B1 (en) 2023-01-01
KR102907580B1 (ko) 2026-01-05

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