CA2970993C - GENETICALLY MODIFIED MESENCHYMATOUS STEM CELLS EXPRESSING ALPHA-1-ANTITRYPSIN (AAT) - Google Patents

GENETICALLY MODIFIED MESENCHYMATOUS STEM CELLS EXPRESSING ALPHA-1-ANTITRYPSIN (AAT)

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Publication number
CA2970993C
CA2970993C CA2970993A CA2970993A CA2970993C CA 2970993 C CA2970993 C CA 2970993C CA 2970993 A CA2970993 A CA 2970993A CA 2970993 A CA2970993 A CA 2970993A CA 2970993 C CA2970993 C CA 2970993C
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Canada
Prior art keywords
genetically modified
promoter
aat
cells
modified cell
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.)
Active
Application number
CA2970993A
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English (en)
French (fr)
Other versions
CA2970993A1 (en
Inventor
Christine Gunther
Sabine Geiger-Schredelseker
Felix Hermann
Ralf Huss
Daria Larissa FORSTER
Original Assignee
Apceth GmbH and Co KG
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Publication of CA2970993A1 publication Critical patent/CA2970993A1/en
Application granted granted Critical
Publication of CA2970993C publication Critical patent/CA2970993C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • 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
    • 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
    • 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

Landscapes

  • 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)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Rheumatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Molecular Biology (AREA)
  • Pulmonology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Otolaryngology (AREA)
CA2970993A 2015-01-08 2016-01-08 GENETICALLY MODIFIED MESENCHYMATOUS STEM CELLS EXPRESSING ALPHA-1-ANTITRYPSIN (AAT) Active CA2970993C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP15150454.5 2015-01-08
EP15150454 2015-01-08
EP15191934.7 2015-10-28
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 (2)

Publication Number Publication Date
CA2970993A1 CA2970993A1 (en) 2016-07-14
CA2970993C true CA2970993C (en) 2025-02-06

Family

ID=55129837

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2970993A Active CA2970993C (en) 2015-01-08 2016-01-08 GENETICALLY MODIFIED MESENCHYMATOUS STEM CELLS EXPRESSING ALPHA-1-ANTITRYPSIN (AAT)

Country Status (18)

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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210086662A (ko) * 2018-10-29 2021-07-08 오타와 하스피털 리서치 인스티튜트 Aoah를 과발현하는 유전자조작된 중간엽 줄기 세포 및 이의 용도
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
EP4585223A3 (en) * 2020-01-15 2025-07-30 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
EP4240323A1 (en) * 2020-11-06 2023-09-13 AATEC Medical GmbH A composition comprising extra-cellular vesicles from mesenchymal stem cells and alpha-1 antitrypsin for the treatment of viral infections
JPWO2024005157A1 (https=) * 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
CN121909290A (zh) * 2023-07-10 2026-04-21 普瑞赛格恩公司 α-1抗胰蛋白酶缺乏症的治疗

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
EP2578677A1 (en) 2007-08-09 2013-04-10 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
BR112014014751A8 (pt) * 2011-12-19 2017-07-04 Lfb Usa Inc composição compreendendo alfa-1-antitripsina, células epiteliais de glândula mamária, mamífero não humano transgênico, e, método para reduzir a atividade de elastase no pulmão
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
SI3242933T1 (sl) 2019-08-30
HUE043478T2 (hu) 2019-08-28
US20200282078A1 (en) 2020-09-10
IL253063A0 (en) 2017-08-31
IL253063B (en) 2019-06-30
EP3242933A1 (en) 2017-11-15
AU2016206033B2 (en) 2022-06-16
RS58977B1 (sr) 2019-08-30
JP2024010006A (ja) 2024-01-23
US20230226222A1 (en) 2023-07-20
WO2016110565A1 (en) 2016-07-14
PT3242933T (pt) 2019-06-05
AU2016206033A1 (en) 2017-07-06
EP3242933B1 (en) 2019-02-27
JP7374527B2 (ja) 2023-11-07
LT3242933T (lt) 2019-06-25
JP2018502579A (ja) 2018-02-01
CY1122004T1 (el) 2020-10-14
ES2727820T3 (es) 2019-10-18
DK3242933T3 (da) 2019-06-03
CN107208063A (zh) 2017-09-26
PL3242933T3 (pl) 2019-08-30
JP2023002657A (ja) 2023-01-10
CA2970993A1 (en) 2016-07-14
JP2021046416A (ja) 2021-03-25
SMT201900381T1 (it) 2019-09-09
US20180000969A1 (en) 2018-01-04

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