CY1122004T1 - Γενετικα τροποποιημενα μεσεγχυματικα βλαστικα κυτταρα που εκφραζουν την αλφα-1 αντιθρυψινη (αττ) - Google Patents

Γενετικα τροποποιημενα μεσεγχυματικα βλαστικα κυτταρα που εκφραζουν την αλφα-1 αντιθρυψινη (αττ)

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Publication number
CY1122004T1
CY1122004T1 CY20191100553T CY191100553T CY1122004T1 CY 1122004 T1 CY1122004 T1 CY 1122004T1 CY 20191100553 T CY20191100553 T CY 20191100553T CY 191100553 T CY191100553 T CY 191100553T CY 1122004 T1 CY1122004 T1 CY 1122004T1
Authority
CY
Cyprus
Prior art keywords
antitrypsin
stem cells
genetically modified
att
cells expressing
Prior art date
Application number
CY20191100553T
Other languages
Greek (el)
English (en)
Inventor
Christine Günther
Sabine GEIGER-SCHREDELSEKER
Felix Hermann
Ralf Huss
Daria Larissa FORSTER
Original Assignee
Apceth Gmbh & Co. Kg
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 Apceth Gmbh & Co. Kg filed Critical Apceth Gmbh & Co. Kg
Publication of CY1122004T1 publication Critical patent/CY1122004T1/el

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    • 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
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0663—Bone marrow mesenchymal stem cells (BM-MSC)
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/55—Protease inhibitors
    • A61K38/57—Protease inhibitors from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • C12N2501/73—Hydrolases (EC 3.)
    • C12N2501/734—Proteases (EC 3.4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Rheumatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pulmonology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
CY20191100553T 2015-01-08 2019-05-24 Γενετικα τροποποιημενα μεσεγχυματικα βλαστικα κυτταρα που εκφραζουν την αλφα-1 αντιθρυψινη (αττ) CY1122004T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15150454 2015-01-08
EP15191934 2015-10-28
PCT/EP2016/050271 WO2016110565A1 (en) 2015-01-08 2016-01-08 Genetically modified mesenchymal stem cells expressing alpha-1 antitrypsin (aat)

Publications (1)

Publication Number Publication Date
CY1122004T1 true CY1122004T1 (el) 2020-10-14

Family

ID=55129837

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20191100553T CY1122004T1 (el) 2015-01-08 2019-05-24 Γενετικα τροποποιημενα μεσεγχυματικα βλαστικα κυτταρα που εκφραζουν την αλφα-1 αντιθρυψινη (αττ)

Country Status (18)

Country Link
US (3) US20180000969A1 (cg-RX-API-DMAC7.html)
EP (1) EP3242933B1 (cg-RX-API-DMAC7.html)
JP (4) JP2018502579A (cg-RX-API-DMAC7.html)
CN (1) CN107208063A (cg-RX-API-DMAC7.html)
AU (1) AU2016206033B2 (cg-RX-API-DMAC7.html)
CA (1) CA2970993C (cg-RX-API-DMAC7.html)
CY (1) CY1122004T1 (cg-RX-API-DMAC7.html)
DK (1) DK3242933T3 (cg-RX-API-DMAC7.html)
ES (1) ES2727820T3 (cg-RX-API-DMAC7.html)
HU (1) HUE043478T2 (cg-RX-API-DMAC7.html)
IL (1) IL253063B (cg-RX-API-DMAC7.html)
LT (1) LT3242933T (cg-RX-API-DMAC7.html)
PL (1) PL3242933T3 (cg-RX-API-DMAC7.html)
PT (1) PT3242933T (cg-RX-API-DMAC7.html)
RS (1) RS58977B1 (cg-RX-API-DMAC7.html)
SI (1) SI3242933T1 (cg-RX-API-DMAC7.html)
SM (1) SMT201900381T1 (cg-RX-API-DMAC7.html)
WO (1) WO2016110565A1 (cg-RX-API-DMAC7.html)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7372982B2 (ja) * 2018-10-29 2023-11-01 オタワ ホスピタル リサーチ インスティチュート Ａｏａｈを過剰発現する遺伝子組換え間葉系幹細胞及びその使用
CN109512893A (zh) * 2018-11-05 2019-03-26 北京世纪劲得生物技术有限公司 间充质干细胞及其注射液和组合物在高尿酸血症中的应用
WO2021020509A1 (ja) * 2019-07-31 2021-02-04 株式会社ステムリム 間葉系幹細胞の増殖促進または減少抑制のための組成物
WO2021146471A2 (en) * 2020-01-15 2021-07-22 The Regents Of The University Of California Transplanted cell protection via inhibition of polymorphonuclear cells
EP3964265A1 (en) * 2020-09-07 2022-03-09 Fundacion Instituto De Investigacion Sanitaria Fundacion Jimenez Diaz Mesenchymal stem cells co-expressing cxcr4 and il-10 and uses thereof
JP2023547728A (ja) * 2020-11-06 2023-11-13 アーテック メディカル ジーエムビーエイチ 間葉系幹細胞由来の細胞外小胞およびアルファ－１アンチトリプシンを含むウイルス感染症治療用組成物
JPWO2024005157A1 (cg-RX-API-DMAC7.html) * 2022-06-30 2024-01-04
CN116173218B (zh) * 2023-04-28 2023-08-04 北京大学口腔医学院 蛋白转运抑制剂在制备用于治疗或缓解牙周炎的药物中的用途
WO2025014986A2 (en) * 2023-07-10 2025-01-16 Precigen, Inc. Therapy for alpha-1 antitrypsin deficiency

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5266491A (en) * 1989-03-14 1993-11-30 Mochida Pharmaceutical Co., Ltd. DNA fragment and expression plasmid containing the DNA fragment
US20070003518A1 (en) * 2002-04-19 2007-01-04 Atkinson Mark A Raav vector-based compositions and methods for the prevention and treatment of mammalian diseases
US20080095749A1 (en) * 2004-03-22 2008-04-24 Sudeepta Aggarwal Mesenchymal stem cells and uses therefor
US9457070B2 (en) * 2005-06-07 2016-10-04 The Regents Of The University Of Colorado, A Body Corporate Compositions, methods and uses of alpha 1-antitrypsin for early intervention in bone marrow transplantation and treatment of graft versus host disease
EP2581441A1 (en) * 2007-08-09 2013-04-17 Genzyme Corporation Method of treating autoimmune disease with mesenchymal stem cells
US8758761B2 (en) * 2007-09-30 2014-06-24 University Of Florida Research Foundation, Inc. Combination therapies for treating type 1 diabetes
EP2626369A1 (en) * 2011-11-24 2013-08-14 Fundació Institut d'Investigació Biomèdica de Bellvitge (IDIBELL) Engineered mesenchymal stem cells and their therapeutic use
AU2012323992A1 (en) * 2011-12-19 2013-07-04 Lfb Usa, Inc. Recombinant human alpha-1-antitrypsin for the treatment of inflammatory disorders
CN102920735A (zh) * 2012-11-14 2013-02-13 青岛奥克生物开发有限公司 一种间充质干细胞注射液及其制备方法和在制备治疗糖尿病药物中的应用
CN105456293A (zh) * 2014-09-05 2016-04-06 深圳市北科生物科技有限公司 一种用于治疗糖尿病的干细胞制剂及其制备方法

Also Published As

Publication number Publication date
CN107208063A (zh) 2017-09-26
LT3242933T (lt) 2019-06-25
CA2970993A1 (en) 2016-07-14
IL253063A0 (en) 2017-08-31
SI3242933T1 (sl) 2019-08-30
ES2727820T3 (es) 2019-10-18
AU2016206033A1 (en) 2017-07-06
JP2024010006A (ja) 2024-01-23
JP2018502579A (ja) 2018-02-01
JP7374527B2 (ja) 2023-11-07
EP3242933A1 (en) 2017-11-15
SMT201900381T1 (it) 2019-09-09
RS58977B1 (sr) 2019-08-30
US20180000969A1 (en) 2018-01-04
JP2023002657A (ja) 2023-01-10
US20200282078A1 (en) 2020-09-10
EP3242933B1 (en) 2019-02-27
WO2016110565A1 (en) 2016-07-14
US20230226222A1 (en) 2023-07-20
JP2021046416A (ja) 2021-03-25
PT3242933T (pt) 2019-06-05
AU2016206033B2 (en) 2022-06-16
PL3242933T3 (pl) 2019-08-30
DK3242933T3 (da) 2019-06-03
CA2970993C (en) 2025-02-06
IL253063B (en) 2019-06-30
HUE043478T2 (hu) 2019-08-28

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