LT3740222T - Modifikuotas raav kapsidės baltymas, skirtas genų terapijai - Google Patents

Modifikuotas raav kapsidės baltymas, skirtas genų terapijai

Info

Publication number
LT3740222T
LT3740222T LTEPPCT/EP2019/051128T LTEP2019051128T LT3740222T LT 3740222 T LT3740222 T LT 3740222T LT EP2019051128 T LTEP2019051128 T LT EP2019051128T LT 3740222 T LT3740222 T LT 3740222T
Authority
LT
Lithuania
Prior art keywords
gene therapy
capsid protein
raav capsid
modified raav
modified
Prior art date
Application number
LTEPPCT/EP2019/051128T
Other languages
English (en)
Lithuanian (lt)
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 LT3740222T publication Critical patent/LT3740222T/lt

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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

Landscapes

  • 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)
LTEPPCT/EP2019/051128T 2018-01-17 2019-01-17 Modifikuotas raav kapsidės baltymas, skirtas genų terapijai LT3740222T (lt)

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
LT3740222T true LT3740222T (lt) 2023-09-11

Family

ID=61022137

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/EP2019/051128T LT3740222T (lt) 2018-01-17 2019-01-17 Modifikuotas raav kapsidės baltymas, skirtas genų terapijai

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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EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
SI3740222T1 (sl) 2018-01-17 2023-11-30 Meiragtx Uk Ii Limited Modificiran kapsidni protein raav za gensko terapijo
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
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
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.
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
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
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
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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
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
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

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SI3740222T1 (sl) 2018-01-17 2023-11-30 Meiragtx Uk Ii Limited Modificiran kapsidni protein raav za gensko terapijo
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
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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