DK3740222T5 - Modificeret RAAV-capsidprotein til genterapi - Google Patents

Modificeret RAAV-capsidprotein til genterapi Download PDF

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Publication number
DK3740222T5
DK3740222T5 DK19701080.4T DK19701080T DK3740222T5 DK 3740222 T5 DK3740222 T5 DK 3740222T5 DK 19701080 T DK19701080 T DK 19701080T DK 3740222 T5 DK3740222 T5 DK 3740222T5
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Prior art keywords
gene therapy
capsid protein
raav capsid
modified raav
modified
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DK19701080.4T
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Danish (da)
English (en)
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DK3740222T3 (da
Inventor
Jonathan Douglas Finn
Dirk Grimm
Kathleen Börner
Susanne Anna Snoek
Niels Broekstra
Der Sanden Sabine Maria Gertrude Van
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Meiragtx Uk Ltd
Univ Heidelberg
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated 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)
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  • Molecular Biology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • 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)
DK19701080.4T 2018-01-17 2019-01-17 Modificeret RAAV-capsidprotein til genterapi DK3740222T5 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18152133 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
DK3740222T3 DK3740222T3 (da) 2023-07-31
DK3740222T5 true DK3740222T5 (da) 2024-08-26

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DK19701080.4T DK3740222T5 (da) 2018-01-17 2019-01-17 Modificeret RAAV-capsidprotein til genterapi

Country Status (27)

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

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JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
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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
BR112022016965A2 (pt) * 2020-02-25 2022-12-06 Childrens Medical Res Institute Polipeptídeos e vetores de capsídeo do vírus adenoassociado
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
GB202110014D0 (en) * 2021-07-12 2021-08-25 Cytiva Bioprocess R & D Ab A method for separating adeno-associated virus capsids, compositions obtained by said method and uses thereof
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
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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
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MX2022000551A (es) * 2019-07-15 2022-05-18 Meiragtx Uk Ii Ltd Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica.

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Publication number Publication date
MY202252A (en) 2024-04-19
JP7389040B2 (ja) 2023-11-29
BR112020014625A2 (pt) 2020-12-08
LT3740222T (lt) 2023-09-11
HUE062774T2 (hu) 2023-12-28
IL275838B2 (en) 2024-11-01
PL3740222T3 (pl) 2023-12-04
MX2020006764A (es) 2021-01-08
ES2957622T3 (es) 2024-01-23
EA202091712A1 (ru) 2021-02-16
HRP20231126T1 (hr) 2024-01-05
CA3087910C (en) 2025-04-08
SMT202300284T1 (it) 2023-11-13
CN112004544A (zh) 2020-11-27
SI3740222T1 (sl) 2023-11-30
US20200354744A1 (en) 2020-11-12
PH12020551096A1 (en) 2021-06-21
US12480138B2 (en) 2025-11-25
NZ767106A (en) 2023-10-27
EP3740222A1 (en) 2020-11-25
DK3740222T3 (da) 2023-07-31
RS64499B1 (sr) 2023-09-29
FI3740222T3 (fi) 2023-07-26
KR102919991B1 (ko) 2026-01-29
AU2019209595B2 (en) 2023-04-27
KR20200135768A (ko) 2020-12-03
CA3087910A1 (en) 2019-07-25
ZA202004980B (en) 2025-07-30
IL275838B1 (en) 2024-07-01
SG11202006056RA (en) 2020-07-29
EP3740222B1 (en) 2023-06-28
JP2021510528A (ja) 2021-04-30
IL275838A (en) 2020-08-31
PT3740222T (pt) 2023-07-11
AU2019209595A1 (en) 2020-09-03
CN112004544B (zh) 2024-08-06
WO2019141765A1 (en) 2019-07-25

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