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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Publication number
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 (https=
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)
  • 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)

Application Number Priority Date Filing Date Title
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)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

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JP2025034621A Division 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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JP2022508182A JP2022508182A (ja) 2022-01-19
JP2022508182A5 JP2022508182A5 (https=) 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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Country Link
US (1) US20210324418A1 (https=)
EP (1) EP3883954A4 (https=)
JP (2) JP7704998B2 (https=)
KR (1) KR20210103469A (https=)
CN (1) CN113302201A (https=)
AR (1) AR117145A1 (https=)
AU (1) AU2019385506A1 (https=)
BR (1) BR112021009733A2 (https=)
CA (1) CA3120289A1 (https=)
CL (1) CL2021001327A1 (https=)
CO (1) CO2021008120A2 (https=)
EA (1) EA202191418A1 (https=)
EC (1) ECSP21044840A (https=)
IL (1) IL283344A (https=)
MX (1) MX2021005997A (https=)
PE (1) PE20211419A1 (https=)
PH (1) PH12021551155A1 (https=)
SG (1) SG11202105326WA (https=)
TW (1) TW202039533A (https=)
WO (1) WO2020106916A1 (https=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
US12173304B2 (en) * 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
WO2022026410A2 (en) * 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
EP4222270A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. NEUROD1-VECTOR
JP2023543360A (ja) * 2020-09-29 2023-10-13 ニューエクセル・セラピューティクス・インコーポレイテッド Ｄｌｘ２ベクター
WO2022072309A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Ascl1 vector
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector
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
CA3203975A1 (en) 2020-12-03 2022-06-09 Battelle Memorial Institute Polymer nanoparticle and dna nanostructure compositions and methods for non-viral delivery
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
CA3216359A1 (en) 2021-04-07 2022-10-13 Battelle Memorial Institute Rapid design, build, test, and learn technologies for identifying and using non-viral carriers
IL311499A (en) * 2021-09-17 2024-05-01 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
EP4598590A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Construct for enhancing 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
AU2024353375A1 (en) 2023-09-29 2026-04-09 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
WO2025122954A1 (en) 2023-12-08 2025-06-12 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170051358A1 (en) 2014-05-06 2017-02-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using nfs1 biomarkers and modulators
US20180021364A1 (en) 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002081632A2 (en) * 2001-04-04 2002-10-17 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services Nucleic acids for transgene expression
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
US20160256571A1 (en) * 2013-09-30 2016-09-08 Sanofi Invention
US11697825B2 (en) * 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CR20170406A (es) * 2015-02-10 2017-11-14 Genzyme Corp Arni variante
US11040113B2 (en) * 2015-03-23 2021-06-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with Friedreich ataxia
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
BR112017024514A2 (pt) * 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
MX390848B (es) * 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
WO2017023708A1 (en) * 2015-07-31 2017-02-09 California Institute Of Technology Activity-dependent expression of nucleic acids
SG11201802408RA (en) * 2015-11-05 2018-05-30 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
MX2018007330A (es) * 2015-12-15 2019-03-14 Genzyme Corp Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
EP4484443A3 (en) * 2016-06-29 2025-03-26 CRISPR Therapeutics AG Materials and methods for treatment of friedreich ataxia and other related disorders
CA3045335A1 (en) * 2016-12-01 2018-06-07 Universite Laval Crispr-based treatment of friedreich ataxia
WO2018204764A1 (en) * 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
AU2018338728B2 (en) * 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170051358A1 (en) 2014-05-06 2017-02-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using nfs1 biomarkers and modulators
US20180021364A1 (en) 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Cloning vector pAAV-9(5)-CMV-EGFP-CytbAS, complete sequence，NCBI，ACCESSION KT343252, 2024年8月30日検索，2015年12月19日，<https://www.ncbi.nlm.nih.gov/nucleotide/KT343252.1?report=genbank&log$=nuclalign&blast_rank=38&RID=D2PYWFGT013＞
Synthetic construct Gluc-T2A-GFP fusion protein (fCMV-Gluc-T2A-GFP) gene, complete cds，NCBI，ACCESSION MG786372, 2024年8月30日検索，2018年03月06日，<https://www.ncbi.nlm.nih.gov/nucleotide/MG786372.1?report=genbank&log$=nuclalign&blast_rank=62&RID=D2RUSM9E016>

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

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