KR20220116459A - 아데노(adeno)-관련 바이러스 벡터 변이체 - Google Patents

아데노(adeno)-관련 바이러스 벡터 변이체 Download PDF

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KR20220116459A
KR20220116459A KR1020227021143A KR20227021143A KR20220116459A KR 20220116459 A KR20220116459 A KR 20220116459A KR 1020227021143 A KR1020227021143 A KR 1020227021143A KR 20227021143 A KR20227021143 A KR 20227021143A KR 20220116459 A KR20220116459 A KR 20220116459A
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capsid protein
modified
aav capsid
seq
targeting peptide
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KR1020227021143A
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Korean (ko)
Inventor
베버리 데이비드슨
융훙 천
폴 티. 래넘
쉐위안 류
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더 칠드런스 호스피탈 오브 필라델피아
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Publication of KR20220116459A publication Critical patent/KR20220116459A/ko
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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2750/14122—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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Peptides Or Proteins (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020227021143A 2019-11-22 2020-11-20 아데노(adeno)-관련 바이러스 벡터 변이체 Pending KR20220116459A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962939315P 2019-11-22 2019-11-22
US62/939,315 2019-11-22
US202063084709P 2020-09-29 2020-09-29
US63/084,709 2020-09-29
PCT/US2020/061464 WO2021102234A1 (en) 2019-11-22 2020-11-20 Adeno-associated viral vector variants

Publications (1)

Publication Number Publication Date
KR20220116459A true KR20220116459A (ko) 2022-08-23

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ID=75980202

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Application Number Title Priority Date Filing Date
KR1020227021143A Pending KR20220116459A (ko) 2019-11-22 2020-11-20 아데노(adeno)-관련 바이러스 벡터 변이체

Country Status (13)

Country Link
US (2) US20230346981A1 (https=)
EP (1) EP4061427A4 (https=)
JP (3) JP7712271B2 (https=)
KR (1) KR20220116459A (https=)
CN (2) CN115023242B (https=)
AU (1) AU2020388404A1 (https=)
BR (1) BR112022009864A2 (https=)
CA (1) CA3159113A1 (https=)
CO (1) CO2022008507A2 (https=)
IL (1) IL293140A (https=)
MX (1) MX2022006188A (https=)
PH (1) PH12022551247A1 (https=)
WO (1) WO2021102234A1 (https=)

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WO2024043523A1 (ko) * 2022-08-26 2024-02-29 경희대학교 산학협력단 활막 표적화 화합물 및 이의 용도

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EP4136457B1 (en) * 2020-04-17 2025-02-19 Regeneron Pharmaceuticals, Inc. Detection assays for coronavirus neutralizing antibodies
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
JP2025514794A (ja) * 2022-04-22 2025-05-09 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア ヒト上衣特異的プロモーターおよびその使用方法
CN119654334A (zh) * 2022-05-11 2025-03-18 费城儿童医院 用于靶向深层脑结构的腺相关病毒载体
AU2023267671A1 (en) * 2022-05-13 2024-12-05 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for targeting brain microvasculature
JP2025521111A (ja) 2022-05-16 2025-07-08 ジェンザイム・コーポレーション 異染性白質ジストロフィーを治療する方法
WO2024017387A1 (en) 2022-07-22 2024-01-25 Shanghai Vitalgen Biopharma Co., Ltd. Novel aav capsids for targeting nervous system and uses thereof
IL319666A (en) * 2022-09-19 2025-05-01 Childrens Hospital Philadelphia AV evolution at single-cell resolution using SPLIT-SEQ
IL322459A (en) * 2023-01-31 2025-09-01 Childrens Hospital Philadelphia Innovative gene therapy construct for cln2 disease
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN116970041B (zh) * 2023-07-21 2024-02-09 广州派真生物技术有限公司 腺相关病毒突变体及其应用
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025090598A1 (en) * 2023-10-23 2025-05-01 The Children's Hospital Of Philadelphia Aav vectors for treatment of cln2 disease
WO2025090631A1 (en) * 2023-10-23 2025-05-01 The Children's Hospital Of Philadelphia Aav vectors for delivery of apoe2
US20250152747A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease
US20250152744A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating neurodegenerative disorders
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
WO2025213097A1 (en) * 2024-04-05 2025-10-09 The Children's Hospital Of Philadelphia Novel gene therapy constructs for stxbp1 haploinsufficiency
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN121265801A (zh) * 2025-06-06 2026-01-06 昆明理工大学 一种靶向性的药物递送载体及其制备方法

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US20040031072A1 (en) * 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
PT1453547T (pt) * 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
ES2739288T3 (es) * 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
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
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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024043523A1 (ko) * 2022-08-26 2024-02-29 경희대학교 산학협력단 활막 표적화 화합물 및 이의 용도

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JP2025102825A (ja) 2025-07-08
CA3159113A1 (en) 2021-05-27
JP2023503455A (ja) 2023-01-30
PH12022551247A1 (en) 2023-11-13
EP4061427A1 (en) 2022-09-28
WO2021102234A1 (en) 2021-05-27
MX2022006188A (es) 2022-08-22
BR112022009864A2 (pt) 2022-08-02
JP7712271B2 (ja) 2025-07-23
CN119978146A (zh) 2025-05-13
JP2025148448A (ja) 2025-10-07
CN115023242A (zh) 2022-09-06
CO2022008507A2 (es) 2022-07-08
EP4061427A4 (en) 2024-01-10
IL293140A (en) 2022-07-01
US20230346981A1 (en) 2023-11-02
CN115023242B (zh) 2025-03-14
US20240100194A1 (en) 2024-03-28
AU2020388404A1 (en) 2022-06-09

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