SI3242933T1 - Genetsko modificirane mezenhimatske matične celice, ki izražajo alfa-1 antitripsin (AAT) - Google Patents

Genetsko modificirane mezenhimatske matične celice, ki izražajo alfa-1 antitripsin (AAT)

Info

Publication number
SI3242933T1
SI3242933T1 SI201630262T SI201630262T SI3242933T1 SI 3242933 T1 SI3242933 T1 SI 3242933T1 SI 201630262 T SI201630262 T SI 201630262T SI 201630262 T SI201630262 T SI 201630262T SI 3242933 T1 SI3242933 T1 SI 3242933T1
Authority
SI
Slovenia
Prior art keywords
antitrypsin
aat
stem cells
cells expressing
genetically modified
Prior art date
Application number
SI201630262T
Other languages
English (en)
Inventor
Christine Gunther
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 SI3242933T1 publication Critical patent/SI3242933T1/sl

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    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2740/10041—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
    • 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
    • C12N2830/008—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)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
SI201630262T 2015-01-08 2016-01-08 Genetsko modificirane mezenhimatske matične celice, ki izražajo alfa-1 antitripsin (AAT) SI3242933T1 (sl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP15150454 2015-01-08
EP15191934 2015-10-28
EP16700404.3A EP3242933B1 (en) 2015-01-08 2016-01-08 Genetically modified mesenchymal stem cells expressing alpha-1 antitrypsin (aat)
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
SI3242933T1 true SI3242933T1 (sl) 2019-08-30

Family

ID=55129837

Family Applications (1)

Application Number Title Priority Date Filing Date
SI201630262T SI3242933T1 (sl) 2015-01-08 2016-01-08 Genetsko modificirane mezenhimatske matične celice, ki izražajo alfa-1 antitripsin (AAT)

Country Status (17)

Country Link
US (3) US20180000969A1 (sl)
EP (1) EP3242933B1 (sl)
JP (4) JP2018502579A (sl)
CN (1) CN107208063A (sl)
AU (1) AU2016206033B2 (sl)
CA (1) CA2970993A1 (sl)
CY (1) CY1122004T1 (sl)
DK (1) DK3242933T3 (sl)
ES (1) ES2727820T3 (sl)
HU (1) HUE043478T2 (sl)
IL (1) IL253063B (sl)
LT (1) LT3242933T (sl)
PL (1) PL3242933T3 (sl)
PT (1) PT3242933T (sl)
RS (1) RS58977B1 (sl)
SI (1) SI3242933T1 (sl)
WO (1) WO2016110565A1 (sl)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220010281A1 (en) * 2018-10-29 2022-01-13 Ottawa Hospital Research Institute Genetically-engineered mesenchymal stem cells overexpressing aoah and uses thereof
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
US20230058557A1 (en) * 2020-01-15 2023-02-23 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
WO2024005157A1 (ja) * 2022-06-30 2024-01-04 国立大学法人熊本大学 脂肪細胞活性化剤
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

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US5266491A (en) * 1989-03-14 1993-11-30 Mochida Pharmaceutical Co., Ltd. DNA fragment and expression plasmid containing the DNA fragment
AU2003221745A1 (en) 2002-04-19 2003-11-03 University Of Florida 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
CN104302312A (zh) * 2011-12-19 2015-01-21 Lfb美国股份有限公司 用于炎性病症治疗的重组人α-1-抗胰蛋白酶
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
EP3242933A1 (en) 2017-11-15
LT3242933T (lt) 2019-06-25
CN107208063A (zh) 2017-09-26
JP2021046416A (ja) 2021-03-25
CY1122004T1 (el) 2020-10-14
RS58977B1 (sr) 2019-08-30
AU2016206033B2 (en) 2022-06-16
JP2023002657A (ja) 2023-01-10
US20230226222A1 (en) 2023-07-20
US20200282078A1 (en) 2020-09-10
IL253063A0 (en) 2017-08-31
WO2016110565A1 (en) 2016-07-14
US20180000969A1 (en) 2018-01-04
JP2018502579A (ja) 2018-02-01
DK3242933T3 (da) 2019-06-03
ES2727820T3 (es) 2019-10-18
AU2016206033A1 (en) 2017-07-06
JP2024010006A (ja) 2024-01-23
EP3242933B1 (en) 2019-02-27
CA2970993A1 (en) 2016-07-14
PT3242933T (pt) 2019-06-05
HUE043478T2 (hu) 2019-08-28
IL253063B (en) 2019-06-30
PL3242933T3 (pl) 2019-08-30
JP7374527B2 (ja) 2023-11-07

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