JP2023545384A - 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス - Google Patents

中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス Download PDF

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JP2023545384A
JP2023545384A JP2023519433A JP2023519433A JP2023545384A JP 2023545384 A JP2023545384 A JP 2023545384A JP 2023519433 A JP2023519433 A JP 2023519433A JP 2023519433 A JP2023519433 A JP 2023519433A JP 2023545384 A JP2023545384 A JP 2023545384A
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aav
capsid
raav
aav9
seq
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JP2023545384A5 (https=
Inventor
ダノス，オリビエ
ヨスト，サマンサ
マーサー，アンドリュー
リウ，イェ
ブルーダー，ジョセフ
カルムチル－メレチル，スバ
フィルンバーグ，エラド
キアン，ランドルフ
テペ，エープリル，アール．
エグレイ，ジェニファー，エム．
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リジェネックスバイオ インコーポレイテッド
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Priority claimed from PCT/US2021/054058 external-priority patent/WO2022076750A2/en
Publication of JP2023545384A publication Critical patent/JP2023545384A/ja
Publication of JP2023545384A5 publication Critical patent/JP2023545384A5/ja
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    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • C12N2750/14122—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
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2023519433A 2020-10-07 2021-10-07 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス Pending JP2023545384A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063088988P 2020-10-07 2020-10-07
US63/088,988 2020-10-07
PCT/US2021/054058 WO2022076750A2 (en) 2020-10-07 2021-10-07 Recombinant adeno-associated viruses for cns or muscle delivery

Publications (2)

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JP2023545384A true JP2023545384A (ja) 2023-10-30
JP2023545384A5 JP2023545384A5 (https=) 2024-10-17

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JP2023519433A Pending JP2023545384A (ja) 2020-10-07 2021-10-07 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス

Country Status (9)

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US (1) US20230374541A1 (https=)
EP (1) EP4225778A2 (https=)
JP (1) JP2023545384A (https=)
KR (1) KR20230082613A (https=)
CN (1) CN116783209A (https=)
AU (1) AU2021358546A1 (https=)
CA (1) CA3193685A1 (https=)
IL (1) IL301563A (https=)
MX (1) MX2023003807A (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20260055144A1 (en) * 2022-08-24 2026-02-26 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119330A2 (en) * 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
WO2020160508A1 (en) * 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119330A2 (en) * 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
WO2020160508A1 (en) * 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF VIROLOGY, vol. Vol.92, No.20, e01011-18, JPN6025053960, 2018, pages 1 - 20, ISSN: 0005765595 *

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MX2023003807A (es) 2023-04-12
EP4225778A2 (en) 2023-08-16
CA3193685A1 (en) 2022-04-14
AU2021358546A9 (en) 2024-02-08
US20230374541A1 (en) 2023-11-23
CN116783209A (zh) 2023-09-19
IL301563A (en) 2023-05-01
AU2021358546A1 (en) 2023-06-08
KR20230082613A (ko) 2023-06-08

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