JP2024514956A - 組織標的化された改変ａａｖカプシドおよびその使用方法 - Google Patents

組織標的化された改変ａａｖカプシドおよびその使用方法 Download PDF

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JP2024514956A
JP2024514956A JP2023565273A JP2023565273A JP2024514956A JP 2024514956 A JP2024514956 A JP 2024514956A JP 2023565273 A JP2023565273 A JP 2023565273A JP 2023565273 A JP2023565273 A JP 2023565273A JP 2024514956 A JP2024514956 A JP 2024514956A
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aav
capsid protein
amino acid
seq
protein
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Japanese (ja)
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JP2024514956A5 (https=
JPWO2022226374A5 (https=
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グレッグ ナクトラブ，
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ロックアネイビオ， インコーポレイテッド
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Publication of JP2024514956A5 publication Critical patent/JP2024514956A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023565273A 2021-04-23 2022-04-22 組織標的化された改変ａａｖカプシドおよびその使用方法 Pending JP2024514956A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163178965P 2021-04-23 2021-04-23
US63/178,965 2021-04-23
US202263299697P 2022-01-14 2022-01-14
US63/299,697 2022-01-14
PCT/US2022/026048 WO2022226374A1 (en) 2021-04-23 2022-04-22 Tissue-targeted modified aav capsids and methods of use thereof

Publications (3)

Publication Number Publication Date
JP2024514956A true JP2024514956A (ja) 2024-04-03
JP2024514956A5 JP2024514956A5 (https=) 2026-03-17
JPWO2022226374A5 JPWO2022226374A5 (https=) 2026-03-17

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JP2023565273A Pending JP2024514956A (ja) 2021-04-23 2022-04-22 組織標的化された改変ａａｖカプシドおよびその使用方法

Country Status (5)

Country Link
US (1) US20240197918A1 (https=)
EP (1) EP4326868A1 (https=)
JP (1) JP2024514956A (https=)
CA (1) CA3216419A1 (https=)
WO (1) WO2022226374A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025230000A1 (ja) * 2024-05-01 2025-11-06 Ｊｃｒファーマ株式会社 修飾されたａａｖ粒子、その製造方法、関連する医薬及びその製造

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US20230159949A1 (en) * 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
EP4353737A1 (en) * 2022-10-10 2024-04-17 Universitätsklinikum Hamburg-Eppendorf Fusion proteins comprising a cell surface marker specific vhh
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
CN118955652A (zh) * 2023-05-15 2024-11-15 杭州新昶基因技术有限公司 腺相关病毒衣壳蛋白、包含其的腺相关病毒及应用
WO2024238807A2 (en) * 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
WO2025137378A1 (en) * 2023-12-20 2025-06-26 Aavantibio, Inc. Aav capsid engineering for cardiac and/or musculoskeletal gene therapy
WO2025133247A1 (en) * 2023-12-21 2025-06-26 Genethon Methods for aav vector re-administration
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025185625A1 (zh) * 2024-03-04 2025-09-12 苏州新芽基因生物技术有限公司 特异性靶向人组织的多肽序列及其在构建递送载体中的应用
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026069251A1 (en) * 2024-09-30 2026-04-02 Sarepta Therapeutics, Inc. Adeno-associated virus capsid antibodies and fragments thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
CN101895633A (zh) 2010-07-14 2010-11-24 中兴通讯股份有限公司 一种移动终端及其解锁方法
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2012109570A1 (en) * 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US10330674B2 (en) 2015-01-13 2019-06-25 Massachusetts Institute Of Technology Pumilio domain-based modular protein architecture for RNA binding
AU2020368539A1 (en) * 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
AU2021264061A1 (en) * 2020-05-01 2022-12-01 Massachusetts Institute Of Technology Engineered central nervous system compositions
TW202217003A (zh) * 2020-07-22 2022-05-01 美商博得學院股份有限公司 經改造之肌肉靶向組合物

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025230000A1 (ja) * 2024-05-01 2025-11-06 Ｊｃｒファーマ株式会社 修飾されたａａｖ粒子、その製造方法、関連する医薬及びその製造

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WO2022226374A1 (en) 2022-10-27
EP4326868A1 (en) 2024-02-28
CA3216419A1 (en) 2022-10-27
US20240197918A1 (en) 2024-06-20

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