JP7704998B2 - 組換えウイルスベクター及びそれの産生のための核酸 - Google Patents

組換えウイルスベクター及びそれの産生のための核酸 Download PDF

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JP7704998B2
JP7704998B2 JP2021529002A JP2021529002A JP7704998B2 JP 7704998 B2 JP7704998 B2 JP 7704998B2 JP 2021529002 A JP2021529002 A JP 2021529002A JP 2021529002 A JP2021529002 A JP 2021529002A JP 7704998 B2 JP7704998 B2 JP 7704998B2
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sequence
nucleic acid
seq
promoter
aav
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JP2022508182A (ja
JP2022508182A5 (enExample
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トーマス，ダービー
ディスミューク，デイヴィッド
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Takeda Pharmaceutical Co Ltd
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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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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
    • C12N2750/14141—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

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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)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021529002A 2018-11-21 2019-11-21 組換えウイルスベクター及びそれの産生のための核酸 Active JP7704998B2 (ja)

Priority Applications (1)

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JP2025034621A JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

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US201862770202P 2018-11-21 2018-11-21
US62/770,202 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

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JP2022508182A JP2022508182A (ja) 2022-01-19
JP2022508182A5 JP2022508182A5 (enExample) 2022-11-30
JP7704998B2 true JP7704998B2 (ja) 2025-07-09

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JP2021529002A Active JP7704998B2 (ja) 2018-11-21 2019-11-21 組換えウイルスベクター及びそれの産生のための核酸
JP2025034621A Pending JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

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US (1) US20210324418A1 (enExample)
EP (1) EP3883954A4 (enExample)
JP (2) JP7704998B2 (enExample)
KR (1) KR20210103469A (enExample)
CN (1) CN113302201A (enExample)
AR (1) AR117145A1 (enExample)
AU (1) AU2019385506A1 (enExample)
BR (1) BR112021009733A2 (enExample)
CA (1) CA3120289A1 (enExample)
CL (1) CL2021001327A1 (enExample)
CO (1) CO2021008120A2 (enExample)
EA (1) EA202191418A1 (enExample)
EC (1) ECSP21044840A (enExample)
IL (1) IL283344A (enExample)
MX (1) MX2021005997A (enExample)
PE (1) PE20211419A1 (enExample)
PH (1) PH12021551155A1 (enExample)
SG (1) SG11202105326WA (enExample)
TW (1) TW202039533A (enExample)
WO (1) WO2020106916A1 (enExample)

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AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
US12173304B2 (en) * 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
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CN116887866A (zh) 2020-12-03 2023-10-13 巴特尔纪念研究院 聚合物纳米颗粒和dna纳米结构组合物及用于非病毒递送的方法
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
JP2024516108A (ja) 2021-04-07 2024-04-12 バテル・メモリアル・インスティテュート 非ウイルス性担体を同定および使用するための迅速な設計、構築、試験、および学習技術
US20240376497A1 (en) * 2021-09-17 2024-11-14 Astellas Gene Therapies, Inc. Frataxin gene therapy
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
US12441996B2 (en) 2023-12-08 2025-10-14 Battelle Memorial Institute Use of DNA origami nanostructures for molecular information based data storage systems
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

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BR112021009733A2 (pt) 2022-01-04
EA202191418A1 (ru) 2021-08-05
JP2025102783A (ja) 2025-07-08
CN113302201A (zh) 2021-08-24
ECSP21044840A (es) 2021-09-30
SG11202105326WA (en) 2021-06-29
TW202039533A (zh) 2020-11-01
PH12021551155A1 (en) 2021-11-03
JP2022508182A (ja) 2022-01-19
AR117145A1 (es) 2021-07-14
KR20210103469A (ko) 2021-08-23
US20210324418A1 (en) 2021-10-21
CL2021001327A1 (es) 2021-12-31
WO2020106916A1 (en) 2020-05-28
EP3883954A4 (en) 2022-08-10
PE20211419A1 (es) 2021-08-03
IL283344A (en) 2021-07-29
AU2019385506A1 (en) 2021-06-03
CA3120289A1 (en) 2020-05-28
CO2021008120A2 (es) 2021-08-09
MX2021005997A (es) 2021-08-11
EP3883954A1 (en) 2021-09-29

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