PL3740222T3 - Zmodyfikowane białko kapsydu raav do terapii genowej - Google Patents

Zmodyfikowane białko kapsydu raav do terapii genowej

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Publication number
PL3740222T3
PL3740222T3 PL19701080.4T PL19701080T PL3740222T3 PL 3740222 T3 PL3740222 T3 PL 3740222T3 PL 19701080 T PL19701080 T PL 19701080T PL 3740222 T3 PL3740222 T3 PL 3740222T3
Authority
PL
Poland
Prior art keywords
gene therapy
capsid protein
raav capsid
modified raav
modified
Prior art date
Application number
PL19701080.4T
Other languages
English (en)
Polish (pl)
Inventor
Jonathan Douglas FINN
Dirk Grimm
Kathleen BÖRNER
Susanne Anna Snoek
Niels Broekstra
Sabine Maria Gertrude Van Der Sanden
Original Assignee
Meiragtx Uk Ii Limited
Universität Heidelberg
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Meiragtx Uk Ii Limited, Universität Heidelberg filed Critical Meiragtx Uk Ii Limited
Publication of PL3740222T3 publication Critical patent/PL3740222T3/pl

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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
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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)
  • 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)
PL19701080.4T 2018-01-17 2019-01-17 Zmodyfikowane białko kapsydu raav do terapii genowej PL3740222T3 (pl)

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 (1)

Publication Number Publication Date
PL3740222T3 true PL3740222T3 (pl) 2023-12-04

Family

ID=61022137

Family Applications (1)

Application Number Title Priority Date Filing Date
PL19701080.4T PL3740222T3 (pl) 2018-01-17 2019-01-17 Zmodyfikowane białko kapsydu raav do terapii genowej

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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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.

Also Published As

Publication number Publication date
DK3740222T5 (da) 2024-08-26
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
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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