MX2020006764A - Una proteína de la cápside de raav modificada para terapia génica. - Google Patents

Una proteína de la cápside de raav modificada para terapia génica.

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Publication number
MX2020006764A
MX2020006764A MX2020006764A MX2020006764A MX2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A
Authority
MX
Mexico
Prior art keywords
gene therapy
capsid protein
raav capsid
modified raav
virions
Prior art date
Application number
MX2020006764A
Other languages
English (en)
Spanish (es)
Inventor
Jonathan Douglas Finn
Dirk Grimm
Kathleen Börner
Niels Broekstra
Susanne Anna Snoek
Der Sanden Sabine Maria Gertrude Van
Original Assignee
Meiragtx Uk Ii Ltd
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 Ltd filed Critical Meiragtx Uk Ii Ltd
Publication of MX2020006764A publication Critical patent/MX2020006764A/es

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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
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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)
  • 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)
MX2020006764A 2018-01-17 2019-01-17 Una proteína de la cápside de raav modificada para terapia génica. MX2020006764A (es)

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
MX2020006764A true MX2020006764A (es) 2021-01-08

Family

ID=61022137

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Application Number Title Priority Date Filing Date
MX2020006764A MX2020006764A (es) 2018-01-17 2019-01-17 Una proteína de la cápside de raav modificada para terapia génica.

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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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AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
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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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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RS64499B1 (sr) 2018-01-17 2023-09-29 Meiragtx Uk Ii Ltd Modifikovani protein kapsida raav za gensku terapiju
KR20220081971A (ko) * 2019-07-15 2022-06-16 메이라지티엑스 유케이 Ii 리미티드 관절염 질환 치료용 변형된 aav 캡시드 단백질

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Publication number Publication date
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
KR102919991B1 (ko) 2026-01-29
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
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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