JP2024526938A - 複数のベクターを共送達することによる、大きなタンパク質の産生方法 - Google Patents

複数のベクターを共送達することによる、大きなタンパク質の産生方法 Download PDF

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JP2024526938A
JP2024526938A JP2024503838A JP2024503838A JP2024526938A JP 2024526938 A JP2024526938 A JP 2024526938A JP 2024503838 A JP2024503838 A JP 2024503838A JP 2024503838 A JP2024503838 A JP 2024503838A JP 2024526938 A JP2024526938 A JP 2024526938A
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polypeptide
split intein
dystrophin
foreign
intein
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JP2024503838A
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Japanese (ja)
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JP2024526938A5 (cg-RX-API-DMAC7.html
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ジェフリー エス． チェンバレン
ヒシャム タスファウト
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University of Washington
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University of Washington
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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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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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)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024503838A 2021-07-23 2022-07-22 複数のベクターを共送達することによる、大きなタンパク質の産生方法 Pending JP2024526938A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163225212P 2021-07-23 2021-07-23
US63/225,212 2021-07-23
US202163256819P 2021-10-18 2021-10-18
US63/256,819 2021-10-18
PCT/US2022/038032 WO2023004125A2 (en) 2021-07-23 2022-07-22 Generation of large proteins by co-delivery of multiple vectors

Publications (2)

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JP2024526938A true JP2024526938A (ja) 2024-07-19
JP2024526938A5 JP2024526938A5 (cg-RX-API-DMAC7.html) 2025-07-29

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JP2024503838A Pending JP2024526938A (ja) 2021-07-23 2022-07-22 複数のベクターを共送達することによる、大きなタンパク質の産生方法

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US (1) US20250099616A1 (cg-RX-API-DMAC7.html)
EP (1) EP4373949A4 (cg-RX-API-DMAC7.html)
JP (1) JP2024526938A (cg-RX-API-DMAC7.html)
WO (1) WO2023004125A2 (cg-RX-API-DMAC7.html)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025015036A1 (en) * 2023-07-10 2025-01-16 Kate Therapeutics, Inc. Aav gene therapy approach for treating lgmd2b/dysferlinopathy
WO2025017169A1 (en) 2023-07-20 2025-01-23 Genethon Novel mididystrophins
WO2025160497A1 (en) * 2024-01-26 2025-07-31 The Trustees Of Indiana University Aav delivery system for full-length dystrophin
WO2025217117A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal muscle-specific targeting motifs and uses thereof
WO2026109723A1 (en) * 2024-11-22 2026-05-28 Katholieke Universiteit Leuven Efficient intein splicing in dual aav gene delivery

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001029243A1 (en) * 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
JP2014528720A (ja) * 2011-09-28 2014-10-30 エラ、ビオテック、ソシエダッド、アノニマＥｒａ Ｂｉｏｔｅｃｈ， Ｓ．Ａ． スプリットインテインおよびその使用
JP2020523006A (ja) * 2017-06-05 2020-08-06 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 増強された改変ウイルスカプシドタンパク質

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3116606A1 (en) * 2018-10-15 2020-04-23 Fondazione Telethon Intein proteins and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001029243A1 (en) * 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
JP2014528720A (ja) * 2011-09-28 2014-10-30 エラ、ビオテック、ソシエダッド、アノニマＥｒａ Ｂｉｏｔｅｃｈ， Ｓ．Ａ． スプリットインテインおよびその使用
JP2020523006A (ja) * 2017-06-05 2020-08-06 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 増強された改変ウイルスカプシドタンパク質

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
HUMAN GENE THERAPY, vol. 19, JPN7026002275, 2008, pages 958 - 964, ISSN: 0005871836 *
HUMAN GENE THERAPY, vol. 25, JPN7026002274, 2014, pages 98 - 108, ISSN: 0005871837 *
J. MOL. BIOL., vol. 426, JPN6026020941, 2014, pages 4018 - 4029, ISSN: 0005871835 *

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EP4373949A2 (en) 2024-05-29
WO2023004125A2 (en) 2023-01-26
EP4373949A4 (en) 2025-09-17
US20250099616A1 (en) 2025-03-27
WO2023004125A3 (en) 2023-03-09

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