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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JP2022508182A5 (enExample
JP2022508182A (ja
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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (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

Applications Claiming Priority (3)

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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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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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CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
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TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
US12173304B2 (en) 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
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EP4221761A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. Neurod1 combination vector
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WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
IL303390A (en) 2020-12-03 2023-08-01 Battelle Memorial Institute Compositions of polymer nanoparticles and DNA nanostructures and methods for non-viral transport
US20240058477A1 (en) * 2021-01-04 2024-02-22 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
WO2022216977A1 (en) 2021-04-07 2022-10-13 Batelle Memorial Institute Rapid design, build, test, and learn technologies for identifying and using non-viral carriers
MX2024003241A (es) * 2021-09-17 2024-05-15 Astellas Gene Therapies Inc Terapia genica con frataxina.
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
EP4536286A1 (en) * 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE GENE EXPRESSION
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
AU2024393766A1 (en) 2023-12-08 2026-06-18 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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AR117145A1 (es) 2021-07-14
CA3120289A1 (en) 2020-05-28
EP3883954A1 (en) 2021-09-29
MX2021005997A (es) 2021-08-11
BR112021009733A2 (pt) 2022-01-04
CO2021008120A2 (es) 2021-08-09
AU2019385506A1 (en) 2021-06-03
PH12021551155A1 (en) 2021-11-03
ECSP21044840A (es) 2021-09-30
US20210324418A1 (en) 2021-10-21
CL2021001327A1 (es) 2021-12-31
CN113302201A (zh) 2021-08-24
IL283344A (en) 2021-07-29
EP3883954A4 (en) 2022-08-10
KR20210103469A (ko) 2021-08-23
WO2020106916A1 (en) 2020-05-28
EA202191418A1 (ru) 2021-08-05
TW202039533A (zh) 2020-11-01
JP2025102783A (ja) 2025-07-08
SG11202105326WA (en) 2021-06-29
PE20211419A1 (es) 2021-08-03
JP2022508182A (ja) 2022-01-19

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