CN114641318A - 用于治疗囊性纤维化的组合物和方法 - Google Patents

用于治疗囊性纤维化的组合物和方法 Download PDF

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Publication number
CN114641318A
CN114641318A CN202080043595.2A CN202080043595A CN114641318A CN 114641318 A CN114641318 A CN 114641318A CN 202080043595 A CN202080043595 A CN 202080043595A CN 114641318 A CN114641318 A CN 114641318A
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seq
raav
polynucleotide
cftr
acid sequence
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CN202080043595.2A
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Chinese (zh)
Inventor
约翰·F·恩格尔哈特
颜子颖
唐应华
埃里克·阮
林深
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Spirovan Science
University of Iowa Research Foundation UIRF
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Spirovan Science
University of Iowa Research Foundation UIRF
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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
    • A61K48/0066—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
    • 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
    • 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

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  • 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)
CN202080043595.2A 2019-04-15 2020-04-15 用于治疗囊性纤维化的组合物和方法 Pending CN114641318A (zh)

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 (1)

Publication Number Publication Date
CN114641318A true CN114641318A (zh) 2022-06-17

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CN202080043595.2A Pending CN114641318A (zh) 2019-04-15 2020-04-15 用于治疗囊性纤维化的组合物和方法

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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=)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114340683A (zh) * 2019-04-15 2022-04-12 衣阿华大学研究基金会 用于转基因表达的方法和组合物
US11684679B2 (en) 2016-03-07 2023-06-27 University Of Iowa Research Foundation AAV-mediated expression using a synthetic promoter and enhancer
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11999965B2 (en) 2017-01-13 2024-06-04 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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CN114340683A (zh) * 2019-04-15 2022-04-12 衣阿华大学研究基金会 用于转基因表达的方法和组合物

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WO2005056762A2 (en) * 2003-12-05 2005-06-23 University Of Iowa Research Foundation Truncated cmv promoters and vectors containing same
CN105142676A (zh) * 2013-03-14 2015-12-09 夏尔人类遗传性治疗公司 Cftr mrna组合物以及相关方法和用途
WO2014168953A1 (en) * 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
CN105431170A (zh) * 2013-04-08 2016-03-23 爱荷华大学研究基金会 嵌合腺相关病毒/博卡病毒细小病毒载体
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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
US11684679B2 (en) 2016-03-07 2023-06-27 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
US11999965B2 (en) 2017-01-13 2024-06-04 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
CN114340683A (zh) * 2019-04-15 2022-04-12 衣阿华大学研究基金会 用于转基因表达的方法和组合物

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Publication number Publication date
AU2020260076A1 (en) 2021-12-09
JP2022529457A (ja) 2022-06-22
WO2020214668A1 (en) 2020-10-22
PH12021552610A1 (en) 2022-06-27
CL2021002702A1 (es) 2022-11-11
SG11202111334SA (en) 2021-11-29
CA3137015A1 (en) 2020-10-22
IL287260A (en) 2021-12-01
JP7731801B2 (ja) 2025-09-01
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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