JP7623703B2 - 硝子体内送達のためのバリアントａａｖキャプシド - Google Patents

硝子体内送達のためのバリアントａａｖキャプシド Download PDF

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JP7623703B2
JP7623703B2 JP2021561683A JP2021561683A JP7623703B2 JP 7623703 B2 JP7623703 B2 JP 7623703B2 JP 2021561683 A JP2021561683 A JP 2021561683A JP 2021561683 A JP2021561683 A JP 2021561683A JP 7623703 B2 JP7623703 B2 JP 7623703B2
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aav
capsid protein
retinal
cells
gene product
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JP2022529775A (ja
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アナヒタ ケラヴァラ，
ダイアナ セペダ，
メヒディ ガスミ，
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アドヴェラム バイオテクノロジーズ， インコーポレイテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021561683A 2019-04-26 2020-04-24 硝子体内送達のためのバリアントａａｖキャプシド Active JP7623703B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962839548P 2019-04-26 2019-04-26
US62/839,548 2019-04-26
US201962923924P 2019-10-21 2019-10-21
US62/923,924 2019-10-21
PCT/US2020/029895 WO2020219933A1 (en) 2019-04-26 2020-04-24 Variant aav capsids for intravitreal delivery

Publications (4)

Publication Number Publication Date
JP2022529775A JP2022529775A (ja) 2022-06-24
JP2022529775A5 JP2022529775A5 (https=) 2023-05-11
JPWO2020219933A5 JPWO2020219933A5 (https=) 2023-05-11
JP7623703B2 true JP7623703B2 (ja) 2025-01-29

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JP2021561683A Active JP7623703B2 (ja) 2019-04-26 2020-04-24 硝子体内送達のためのバリアントａａｖキャプシド

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US (1) US11510950B2 (https=)
EP (1) EP3958911A1 (https=)
JP (1) JP7623703B2 (https=)
KR (1) KR20220004114A (https=)
CN (2) CN113766934B (https=)
AU (1) AU2020262948A1 (https=)
BR (1) BR112021020957A2 (https=)
CA (1) CA3130217A1 (https=)
IL (1) IL287438A (https=)
MX (2) MX2021012311A (https=)
SG (1) SG11202108928XA (https=)
TW (2) TW202532428A (https=)
WO (1) WO2020219933A1 (https=)
ZA (1) ZA202105975B (https=)

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CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
CN115044614B (zh) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
CN113648432B (zh) * 2021-03-31 2024-01-19 中山大学中山眼科中心 rAAV2/Retro作为针对视网膜感光细胞的递送系统及其在制备治疗视网膜疾病药物中的应用
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
AU2024242380A1 (en) * 2023-03-31 2025-11-06 Exegenesis Bio Singapore Pte. Ltd. Variant aav9 capsid proteins and uses thereof
WO2024211831A1 (en) 2023-04-05 2024-10-10 Adverum Biotechnologies, Inc. Immune landscape signatures for ocular inflammation
AU2024297654A1 (en) 2023-07-13 2026-01-22 Kriya Therapeutics, Inc. Complement pathway inhibitor fusions and methods of using the same
CN117247434B (zh) * 2023-11-10 2024-02-02 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
CN118955654B (zh) * 2024-10-15 2025-05-16 朗信启昇(苏州)生物制药有限公司 具有变异衣壳的腺相关病毒病毒体及其用途
WO2026084101A1 (ko) 2024-10-18 2026-04-23 주식회사 아바타테라퓨틱스 아데노연관바이러스 변이체

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JP2014518614A (ja) 2011-04-22 2014-08-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
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WO2018022905A2 (en) 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
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JP2022529775A (ja) 2022-06-24
US20200338146A1 (en) 2020-10-29
TW202106699A (zh) 2021-02-16
CA3130217A1 (en) 2020-10-29
IL287438A (en) 2021-12-01
WO2020219933A1 (en) 2020-10-29
MX2025015023A (es) 2026-02-03
US11510950B2 (en) 2022-11-29
TW202532428A (zh) 2025-08-16
AU2020262948A1 (en) 2021-09-30
BR112021020957A2 (pt) 2021-12-14
ZA202105975B (en) 2024-12-18
CN113766934B (zh) 2024-11-08
CN119708162A (zh) 2025-03-28
KR20220004114A (ko) 2022-01-11
SG11202108928XA (en) 2021-09-29
TWI862583B (zh) 2024-11-21
MX2021012311A (es) 2021-11-12
EP3958911A1 (en) 2022-03-02
CN113766934A (zh) 2021-12-07

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