KR102919991B1 - 유전자 치료를 위한 변형된 rAAV 캡시드 단백질 - Google Patents

유전자 치료를 위한 변형된 rAAV 캡시드 단백질

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Publication number
KR102919991B1
KR102919991B1 KR1020207023174A KR20207023174A KR102919991B1 KR 102919991 B1 KR102919991 B1 KR 102919991B1 KR 1020207023174 A KR1020207023174 A KR 1020207023174A KR 20207023174 A KR20207023174 A KR 20207023174A KR 102919991 B1 KR102919991 B1 KR 102919991B1
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KR20200135768A (ko
Inventor
조나단 더글라스 핀
더크 그림
캐슬린 뵈르너
수잔느 안나 스노크
닐스 브록스트라
사빈 마리아 제르트루다 반 더 샌딘
Original Assignee
메이라지티엑스 진 레귤레이션 리미티드
우니페르시테트 하이델베르크
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    • 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
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2750/14145—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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020207023174A 2018-01-17 2019-01-17 유전자 치료를 위한 변형된 rAAV 캡시드 단백질 Active KR102919991B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18152133 2018-01-17
EP18152133.7 2018-01-17
PCT/EP2019/051128 WO2019141765A1 (en) 2018-01-17 2019-01-17 A modified raav capsid protein for gene therapy

Publications (2)

Publication Number Publication Date
KR20200135768A KR20200135768A (ko) 2020-12-03
KR102919991B1 true KR102919991B1 (ko) 2026-01-29

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Country Status (27)

Country Link
US (1) US12480138B2 (enExample)
EP (1) EP3740222B1 (enExample)
JP (1) JP7389040B2 (enExample)
KR (1) KR102919991B1 (enExample)
CN (1) CN112004544B (enExample)
AU (1) AU2019209595B2 (enExample)
BR (1) BR112020014625A2 (enExample)
CA (1) CA3087910C (enExample)
DK (1) DK3740222T5 (enExample)
EA (1) EA202091712A1 (enExample)
ES (1) ES2957622T3 (enExample)
FI (1) FI3740222T3 (enExample)
HR (1) HRP20231126T1 (enExample)
HU (1) HUE062774T2 (enExample)
IL (1) IL275838B2 (enExample)
LT (1) LT3740222T (enExample)
MX (1) MX2020006764A (enExample)
MY (1) MY202252A (enExample)
PH (1) PH12020551096A1 (enExample)
PL (1) PL3740222T3 (enExample)
PT (1) PT3740222T (enExample)
RS (1) RS64499B1 (enExample)
SG (1) SG11202006056RA (enExample)
SI (1) SI3740222T1 (enExample)
SM (1) SMT202300284T1 (enExample)
WO (1) WO2019141765A1 (enExample)
ZA (1) ZA202004980B (enExample)

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RS64499B1 (sr) 2018-01-17 2023-09-29 Meiragtx Uk Ii Ltd Modifikovani protein kapsida raav za gensku terapiju
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
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KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
CN110950934B (zh) * 2019-12-31 2022-11-04 复旦大学 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用
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MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
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WO2024213660A1 (en) 2023-04-13 2024-10-17 Meiragtx Uk Ii Limited Inflammation-inducible promoters
CN119320802A (zh) * 2023-07-17 2025-01-17 苏州吉恒基因科技有限公司 单链单极DNA重组腺相关病毒的cis包装元件、spAAV载体和应用
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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WO2013174760A1 (en) 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Optimized aav-vectors for high transduction rates in dendritic cells

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IL275838A (en) 2020-08-31
PL3740222T3 (pl) 2023-12-04
JP2021510528A (ja) 2021-04-30
HRP20231126T1 (hr) 2024-01-05
EP3740222A1 (en) 2020-11-25
PH12020551096A1 (en) 2021-06-21
AU2019209595A1 (en) 2020-09-03
SG11202006056RA (en) 2020-07-29
WO2019141765A1 (en) 2019-07-25
ZA202004980B (en) 2025-07-30
ES2957622T3 (es) 2024-01-23
FI3740222T3 (fi) 2023-07-26
EA202091712A1 (ru) 2021-02-16
PT3740222T (pt) 2023-07-11
EP3740222B1 (en) 2023-06-28
CA3087910C (en) 2025-04-08
DK3740222T5 (da) 2024-08-26
CN112004544B (zh) 2024-08-06
CA3087910A1 (en) 2019-07-25
CN112004544A (zh) 2020-11-27
US20200354744A1 (en) 2020-11-12
IL275838B1 (en) 2024-07-01
MX2020006764A (es) 2021-01-08
RS64499B1 (sr) 2023-09-29
IL275838B2 (en) 2024-11-01
DK3740222T3 (da) 2023-07-31
SMT202300284T1 (it) 2023-11-13
NZ767106A (en) 2023-10-27
US12480138B2 (en) 2025-11-25
HUE062774T2 (hu) 2023-12-28
BR112020014625A2 (pt) 2020-12-08
KR20200135768A (ko) 2020-12-03
SI3740222T1 (sl) 2023-11-30
LT3740222T (lt) 2023-09-11
MY202252A (en) 2024-04-19
JP7389040B2 (ja) 2023-11-29
AU2019209595B2 (en) 2023-04-27

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