JP7731801B2 - 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス - Google Patents

嚢胞性線維症の治療のための組換えアデノ随伴ウイルス

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Publication number
JP7731801B2
JP7731801B2 JP2021561742A JP2021561742A JP7731801B2 JP 7731801 B2 JP7731801 B2 JP 7731801B2 JP 2021561742 A JP2021561742 A JP 2021561742A JP 2021561742 A JP2021561742 A JP 2021561742A JP 7731801 B2 JP7731801 B2 JP 7731801B2
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sequence
seq
polynucleotide
raav
aav
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JP2021561742A
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English (en)
Japanese (ja)
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JP2022529457A (ja
JPWO2020214668A5 (https=
JP2022529457A5 (https=
Inventor
エフ． エンゲルハート、ジョン
ヤン、ジーイン
タン、インファ
ユン、エリック
リン、シェン
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University of Iowa Research Foundation UIRF
Spirovant Sciences Inc
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University of Iowa Research Foundation UIRF
Spirovant Sciences Inc
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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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021561742A 2019-04-15 2020-04-15 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス Active JP7731801B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962833972P 2019-04-15 2019-04-15
US62/833,972 2019-04-15
US201962926308P 2019-10-25 2019-10-25
US62/926,308 2019-10-25
US202062967214P 2020-01-29 2020-01-29
US62/967,214 2020-01-29
PCT/US2020/028264 WO2020214668A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Publications (4)

Publication Number Publication Date
JP2022529457A JP2022529457A (ja) 2022-06-22
JPWO2020214668A5 JPWO2020214668A5 (https=) 2023-04-18
JP2022529457A5 JP2022529457A5 (https=) 2023-04-18
JP7731801B2 true JP7731801B2 (ja) 2025-09-01

Family

ID=70847479

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021561742A Active JP7731801B2 (ja) 2019-04-15 2020-04-15 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス

Country Status (14)

Country Link
US (1) US20220241436A1 (https=)
EP (1) EP3955971A1 (https=)
JP (1) JP7731801B2 (https=)
KR (1) KR20220044899A (https=)
CN (1) CN114641318A (https=)
AU (1) AU2020260076A1 (https=)
BR (1) BR112021020708A2 (https=)
CA (1) CA3137015A1 (https=)
CL (1) CL2021002702A1 (https=)
IL (1) IL287260A (https=)
MX (1) MX2021012681A (https=)
PH (1) PH12021552610A1 (https=)
SG (1) SG11202111334SA (https=)
WO (1) WO2020214668A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3198936A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
EP3955970A1 (en) * 2019-04-15 2022-02-23 University Of Iowa Research Foundation Methods and compositions for transgene expression
US20230242941A1 (en) * 2020-06-30 2023-08-03 Eric Yuen Methods and compositions for administering recombinant viral vectors
US20240115738A1 (en) 2021-04-15 2024-04-11 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
WO2026072721A1 (en) * 2024-09-25 2026-04-02 Carbon Biosciences, Inc. Parvovirus compositions and methods for gene therapy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer

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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
AU5445998A (en) 1996-11-19 1998-06-10 Surgx Corporation A transient voltage protection device and method of making same
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
JP2007524379A (ja) 2003-03-31 2007-08-30 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン 上皮ナトリウムチャンネル関連障害のＰｈａｒｍｉｃｏ−遺伝子治療
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005056762A2 (en) * 2003-12-05 2005-06-23 University Of Iowa Research Foundation Truncated cmv promoters and vectors containing same
KR20210122917A (ko) * 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
AU2014251099B2 (en) * 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
US11000597B2 (en) * 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
EP3955970A1 (en) * 2019-04-15 2022-02-23 University Of Iowa Research Foundation Methods and compositions for transgene expression

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer

Non-Patent Citations (2)

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PNAS，2009年，Vol.106, No.10，p.3865-3870, Supporting Information
UniProtKB/TrEMBL [online]，2018年12月05日，Accesion No. P13569, entry version 243，https://rest.uniprot.org/unisave/P13569?format=txt&versions=243，［検索日 2022.04.08］

Also Published As

Publication number Publication date
AU2020260076A1 (en) 2021-12-09
JP2022529457A (ja) 2022-06-22
WO2020214668A1 (en) 2020-10-22
PH12021552610A1 (en) 2022-06-27
CN114641318A (zh) 2022-06-17
CL2021002702A1 (es) 2022-11-11
SG11202111334SA (en) 2021-11-29
CA3137015A1 (en) 2020-10-22
IL287260A (en) 2021-12-01
KR20220044899A (ko) 2022-04-12
US20220241436A1 (en) 2022-08-04
BR112021020708A2 (pt) 2022-03-15
MX2021012681A (es) 2022-03-25
EP3955971A1 (en) 2022-02-23

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