JP2024521063A - Ｔｅｒｔをコードする組換えウイルスゲノム及びウイルスベクター - Google Patents

Ｔｅｒｔをコードする組換えウイルスゲノム及びウイルスベクター Download PDF

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JP2024521063A
JP2024521063A JP2023570195A JP2023570195A JP2024521063A JP 2024521063 A JP2024521063 A JP 2024521063A JP 2023570195 A JP2023570195 A JP 2023570195A JP 2023570195 A JP2023570195 A JP 2023570195A JP 2024521063 A JP2024521063 A JP 2024521063A
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aav9
seq
promoter
aav
vector
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JP2024521063A5 (enExample
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マリア ブラスコ
パウラ マルティネス
トゥベール マリア ファティマ ボッシュ
センツァーノ ヴェロニカ ヒメネス
マルティネス ミケル ガルシア
ロレンテ エステファニア カサナ
Original Assignee
フンダシオン デル セクトル パブリコ エスタタル セントロ ナショナル デ インベスティガシオネス オンコロジカス カルロス テルセーロ（エフェ．エセ．ペ． セエネイオ）
ウニベルシタ アウトノマ デ バルセロナ
テロメア セラピューティクス エセエレ
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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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2830/008—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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023570195A 2021-05-12 2022-05-12 Ｔｅｒｔをコードする組換えウイルスゲノム及びウイルスベクター Pending JP2024521063A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21382441.0A EP4089171A1 (en) 2021-05-12 2021-05-12 Recombinant tert-encoding viral genomes and vectors
EP21382441.0 2021-05-12
PCT/EP2022/062990 WO2022238557A1 (en) 2021-05-12 2022-05-12 Recombinant tert-encoding viral genomes and vectors

Publications (2)

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JP2024521063A true JP2024521063A (ja) 2024-05-28
JP2024521063A5 JP2024521063A5 (enExample) 2025-05-20

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JP2023570195A Pending JP2024521063A (ja) 2021-05-12 2022-05-12 Ｔｅｒｔをコードする組換えウイルスゲノム及びウイルスベクター

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US (2) US20250099618A1 (enExample)
EP (2) EP4089171A1 (enExample)
JP (1) JP2024521063A (enExample)
CN (1) CN117836420A (enExample)
AU (1) AU2022273863A1 (enExample)
CA (1) CA3219795A1 (enExample)
ES (1) ES3073712T3 (enExample)
MX (1) MX2023013385A (enExample)
WO (1) WO2022238557A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12668817B1 (en) * 2025-07-10 2026-06-30 Telomere Therapeutics S.L. Recombinant adeno-associated viral vectors for delivering a therapeutic gene to lungs

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20010016352A1 (en) * 1994-05-18 2001-08-23 Robert J. Bohinski Nucleic acid sequences controlling lung cell-specific gene expression
EP2402038A1 (en) * 2010-07-02 2012-01-04 Fundación Centro Nacional De Investigaciones Oncológicas Carlos III Telomerase reverse transcriptase for protection against ageing
US20140088175A1 (en) * 2004-09-08 2014-03-27 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Transduction of cells by adeno-associated virus vectors
WO2016020345A1 (en) * 2014-08-08 2016-02-11 F. Hoffmann-La Roche Ag Telomerase reverse transcriptase-based therapies

Family Cites Families (20)

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Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
EP1007637B1 (en) 1997-04-14 2004-06-30 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
US6207455B1 (en) 1997-05-01 2001-03-27 Lung-Ji Chang Lentiviral vectors
CN1247788C (zh) 1997-05-13 2006-03-29 北卡罗来纳-查佩尔山大学 基于慢病毒的基因转移载体
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6218181B1 (en) 1998-03-18 2001-04-17 The Salk Institute For Biological Studies Retroviral packaging cell line
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
DE19909769A1 (de) 1999-03-05 2000-09-07 Bundesrepublik Deutschland Let Von SIVagm abgeleitete lentivirale Vektoren, Verfahren zu ihrer Herstellung und ihre Verwendung zur Genübertragung in Säugerzellen
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US9453209B2 (en) * 2012-12-27 2016-09-27 Sierra Sciences, Llc Enhancing health in mammals using telomerase reverse transcriptase gene therapy
ES2936383T3 (es) 2014-08-08 2023-03-16 Fundacion Del Sector Publico Estatal Centro Nac De Investigaciones Oncologicas Carlos Iii F S P Cnio Terapias basadas en transcriptasa inversa de la telomerasa para el tratamiento de afecciones asociadas con el infarto de miocardio

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20010016352A1 (en) * 1994-05-18 2001-08-23 Robert J. Bohinski Nucleic acid sequences controlling lung cell-specific gene expression
US20140088175A1 (en) * 2004-09-08 2014-03-27 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Transduction of cells by adeno-associated virus vectors
EP2402038A1 (en) * 2010-07-02 2012-01-04 Fundación Centro Nacional De Investigaciones Oncológicas Carlos III Telomerase reverse transcriptase for protection against ageing
WO2016020345A1 (en) * 2014-08-08 2016-02-11 F. Hoffmann-La Roche Ag Telomerase reverse transcriptase-based therapies

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JUAN MANUEL POVEDANO; ET AL: "THERAPEUTIC EFFECTS OF TELOMERASE IN MICE WITH PULMONARY FIBROSIS INDUCED BY DAMAGE TO 以下備考", ELIFE, vol. 7, JPN5025010778, 30 January 2018 (2018-01-30), pages 31299 - 1, ISSN: 0005875103 *

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Publication number Publication date
US20260115322A1 (en) 2026-04-30
WO2022238557A1 (en) 2022-11-17
ES3073712T3 (en) 2026-07-14
EP4337780A1 (en) 2024-03-20
MX2023013385A (es) 2024-02-06
AU2022273863A1 (en) 2023-12-07
US20250099618A1 (en) 2025-03-27
EP4337780B1 (en) 2026-04-01
CN117836420A (zh) 2024-04-05
EP4089171A1 (en) 2022-11-16
EP4337780C0 (en) 2026-04-01
CA3219795A1 (en) 2022-11-17

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