JP7756439B2 - 異種間適合性アデノ随伴ウイルス組成物およびその使用方法 - Google Patents

異種間適合性アデノ随伴ウイルス組成物およびその使用方法

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Publication number
JP7756439B2
JP7756439B2 JP2022567079A JP2022567079A JP7756439B2 JP 7756439 B2 JP7756439 B2 JP 7756439B2 JP 2022567079 A JP2022567079 A JP 2022567079A JP 2022567079 A JP2022567079 A JP 2022567079A JP 7756439 B2 JP7756439 B2 JP 7756439B2
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Prior art keywords
aav
capsid protein
vector
seq
amino acid
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Japanese (ja)
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JP2023524291A (ja
JP2023524291A5 (enExample
Inventor
アラビンド アソカン，
トレバー ゴンザレス，
ローレンス パトリック ハヴリク，
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Duke University
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Duke University
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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022567079A 2020-05-05 2021-05-05 異種間適合性アデノ随伴ウイルス組成物およびその使用方法 Active JP7756439B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063020062P 2020-05-05 2020-05-05
US63/020,062 2020-05-05
PCT/US2021/030937 WO2021226267A2 (en) 2020-05-05 2021-05-05 Cross-species compatible adeno-associated virus compositions and methods of use thereof

Publications (3)

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JP2023524291A JP2023524291A (ja) 2023-06-09
JP2023524291A5 JP2023524291A5 (enExample) 2024-05-15
JP7756439B2 true JP7756439B2 (ja) 2025-10-20

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Country Link
US (3) US12577586B2 (enExample)
EP (1) EP4125977A4 (enExample)
JP (1) JP7756439B2 (enExample)
KR (1) KR20230029616A (enExample)
CN (1) CN116096377B (enExample)
AR (1) AR122013A1 (enExample)
AU (1) AU2021268946A1 (enExample)
BR (1) BR112022022644A2 (enExample)
CA (1) CA3177869A1 (enExample)
IL (1) IL297577A (enExample)
MX (1) MX2022013963A (enExample)
TW (1) TWI910162B (enExample)
WO (1) WO2021226267A2 (enExample)

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TWI910162B (zh) * 2020-05-05 2026-01-01 美國公爵大學 跨物種相容腺相關病毒組合物及其使用方法
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2024044725A2 (en) * 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2024168089A2 (en) * 2023-02-07 2024-08-15 Duke University Compositions comprising aavs and methods for treating gastrointestinal diseases
US20240261438A1 (en) * 2023-02-08 2024-08-08 Ginkgo Bioworks, Inc. Gene therapy for angelman syndrome
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
EP4689139A1 (en) * 2023-03-31 2026-02-11 Duke University Compositions comprising brain-tropic aavs and methods of use thereof
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025250454A1 (en) * 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

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WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
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Also Published As

Publication number Publication date
IL297577A (en) 2022-12-01
JP2023524291A (ja) 2023-06-09
MX2022013963A (es) 2023-02-22
TW202208631A (zh) 2022-03-01
AR122013A1 (es) 2022-08-03
EP4125977A2 (en) 2023-02-08
US12146151B2 (en) 2024-11-19
US20230151389A1 (en) 2023-05-18
CN116096377A (zh) 2023-05-09
CA3177869A1 (en) 2021-11-11
KR20230029616A (ko) 2023-03-03
US12577586B2 (en) 2026-03-17
US12037598B2 (en) 2024-07-16
US20240076695A1 (en) 2024-03-07
US20240067988A1 (en) 2024-02-29
WO2021226267A3 (en) 2021-12-16
BR112022022644A2 (pt) 2023-01-17
WO2021226267A2 (en) 2021-11-11
AU2021268946A1 (en) 2022-12-01
CN116096377B (zh) 2026-01-02
EP4125977A4 (en) 2024-09-25
TWI910162B (zh) 2026-01-01

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