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 (OSRAM
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)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (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 (OSRAM) 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)

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

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
CA3052829A1 (en) * 2017-02-15 2018-08-23 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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Publication number Publication date
MX2023003807A (es) 2023-04-12
AU2021358546A9 (en) 2024-02-08
AU2021358546A1 (en) 2023-06-08
EP4225778A2 (en) 2023-08-16
US20230374541A1 (en) 2023-11-23
IL301563A (en) 2023-05-01
CN116783209A (zh) 2023-09-19
CA3193685A1 (en) 2022-04-14
KR20230082613A (ko) 2023-06-08

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