JP7677887B2 - Ａａｖウイルスベクター及びその使用 - Google Patents

Ａａｖウイルスベクター及びその使用 Download PDF

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JP7677887B2
JP7677887B2 JP2021530078A JP2021530078A JP7677887B2 JP 7677887 B2 JP7677887 B2 JP 7677887B2 JP 2021530078 A JP2021530078 A JP 2021530078A JP 2021530078 A JP2021530078 A JP 2021530078A JP 7677887 B2 JP7677887 B2 JP 7677887B2
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JP2022511776A (ja
JPWO2020113034A5 (https=
JP2022511776A5 (https=
Inventor
マイケル ハットフィールド，ジェームス
エミル ホッジ，ロバート
フェルター，ダグラス
バレイディア，ジョゼフ
メリッジョーリ，マシュー
ケー． カスパー，ブライアン
アルマン カスパー，アラン
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ノバルティス アーゲー
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2021530078A 2018-11-30 2019-11-27 Ａａｖウイルスベクター及びその使用 Active JP7677887B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024213225A JP7808674B2 (ja) 2018-11-30 2024-12-06 Ａａｖウイルスベクター及びその使用

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862773894P 2018-11-30 2018-11-30
US62/773,894 2018-11-30
US201962835242P 2019-04-17 2019-04-17
US62/835,242 2019-04-17
PCT/US2019/063649 WO2020113034A1 (en) 2018-11-30 2019-11-27 Aav viral vectors and uses thereof

Related Child Applications (1)

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JP2024213225A Division JP7808674B2 (ja) 2018-11-30 2024-12-06 Ａａｖウイルスベクター及びその使用

Publications (4)

Publication Number Publication Date
JP2022511776A JP2022511776A (ja) 2022-02-01
JPWO2020113034A5 JPWO2020113034A5 (https=) 2022-12-01
JP2022511776A5 JP2022511776A5 (https=) 2022-12-01
JP7677887B2 true JP7677887B2 (ja) 2025-05-15

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JP2024213225A Active JP7808674B2 (ja) 2018-11-30 2024-12-06 Ａａｖウイルスベクター及びその使用

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Country Link
US (1) US20220001028A1 (https=)
EP (1) EP3886919A1 (https=)
JP (2) JP7677887B2 (https=)
KR (1) KR20210099025A (https=)
CN (2) CN120860258A (https=)
AU (1) AU2019389047A1 (https=)
BR (1) BR112021009739A2 (https=)
CA (1) CA3116630A1 (https=)
IL (1) IL282885A (https=)
MX (1) MX2021006359A (https=)
TW (2) TWI897418B (https=)
WO (1) WO2020113034A1 (https=)

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TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
US20240084323A1 (en) * 2021-01-13 2024-03-14 Dignity Health Modulation of chitinase protein expression
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4380602A4 (en) * 2021-08-03 2025-06-11 The Regents Of The University Of California ADENO-ASSOCIATED VIRAL (AAV)-MEDIATED EXPRESSION OF SPHINGOSINE 1-PHOSPHATE LYASE (SPL) FOR THE TREATMENT OF PULMONARY FIBROSIS
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
KR20230152503A (ko) * 2022-04-27 2023-11-03 주식회사 헬릭스미스 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2023246734A1 (en) 2022-06-21 2023-12-28 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of sma disease
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024238958A1 (en) * 2023-05-18 2024-11-21 Ultragenyx Pharmaceutical Inc. Formulations comprising recombinant aav and methods of administering the same
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

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Also Published As

Publication number Publication date
WO2020113034A1 (en) 2020-06-04
TW202509229A (zh) 2025-03-01
US20220001028A1 (en) 2022-01-06
CN113226380A (zh) 2021-08-06
AU2019389047A1 (en) 2021-05-20
MX2021006359A (es) 2021-08-11
CN120860258A (zh) 2025-10-31
EP3886919A1 (en) 2021-10-06
JP2025060641A (ja) 2025-04-10
BR112021009739A2 (pt) 2021-10-19
JP2022511776A (ja) 2022-02-01
TW202039859A (zh) 2020-11-01
TWI844587B (zh) 2024-06-11
CA3116630A1 (en) 2020-06-04
JP7808674B2 (ja) 2026-01-29
KR20210099025A (ko) 2021-08-11
TWI897418B (zh) 2025-09-11
IL282885A (en) 2021-06-30

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