JP2023537625A - 新規ａａｖカプシド及びそれを含む組成物 - Google Patents

新規ａａｖカプシド及びそれを含む組成物 Download PDF

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JP2023537625A
JP2023537625A JP2023511556A JP2023511556A JP2023537625A JP 2023537625 A JP2023537625 A JP 2023537625A JP 2023511556 A JP2023511556 A JP 2023511556A JP 2023511556 A JP2023511556 A JP 2023511556A JP 2023537625 A JP2023537625 A JP 2023537625A
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seq
acid sequence
aav
capsid
amino acid
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JP2023537625A5 (https=
Inventor
ウイルソン，ジェームス・エム
ナンビアー，カリヤニ
ワン，チャン
シドレーン，ジェニー・エイ
マルティーノ，リチャード・アレキサンダー
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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Publication of JP2023537625A publication Critical patent/JP2023537625A/ja
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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
    • A61K48/0008—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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
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  • Veterinary Medicine (AREA)
  • Public Health (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023511556A 2020-08-14 2021-08-13 新規ａａｖカプシド及びそれを含む組成物 Pending JP2023537625A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063065616P 2020-08-14 2020-08-14
US63/065,616 2020-08-14
US202063109734P 2020-11-04 2020-11-04
US63/109,734 2020-11-04
PCT/US2021/045945 WO2022036220A1 (en) 2020-08-14 2021-08-13 Novel aav capsids and compositions containing same

Publications (2)

Publication Number Publication Date
JP2023537625A true JP2023537625A (ja) 2023-09-04
JP2023537625A5 JP2023537625A5 (https=) 2024-08-13

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JP2023511556A Pending JP2023537625A (ja) 2020-08-14 2021-08-13 新規ａａｖカプシド及びそれを含む組成物

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US (1) US20230287451A1 (https=)
EP (1) EP4196170A1 (https=)
JP (1) JP2023537625A (https=)
KR (1) KR20230051208A (https=)
CN (1) CN116209769A (https=)
AU (1) AU2021325954A1 (https=)
CA (1) CA3188956A1 (https=)
IL (1) IL300410A (https=)
MX (1) MX2023001863A (https=)
TW (1) TW202221127A (https=)
WO (1) WO2022036220A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
CN119173269A (zh) * 2022-03-03 2024-12-20 宾夕法尼亚州大学信托人 用于递送glp-1受体激动剂融合物的aav载体
US20260021205A1 (en) * 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
CN117801116A (zh) * 2022-09-30 2024-04-02 上海玮美基因科技有限责任公司 一种融合型新型腺相关病毒及其应用
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
CN120677245A (zh) * 2024-01-25 2025-09-19 鼐济医药科技(杭州)有限公司 Aav载体、载体组合物、嵌合载体和治疗或预防sma的方法

Citations (1)

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WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids

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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
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WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids

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Publication number Publication date
US20230287451A1 (en) 2023-09-14
KR20230051208A (ko) 2023-04-17
EP4196170A1 (en) 2023-06-21
CA3188956A1 (en) 2022-02-17
CN116209769A (zh) 2023-06-02
WO2022036220A1 (en) 2022-02-17
MX2023001863A (es) 2023-06-13
TW202221127A (zh) 2022-06-01
IL300410A (en) 2023-04-01
AU2021325954A1 (en) 2023-03-02

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