LT3411484T - Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį - Google Patents

Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį

Info

Publication number
LT3411484T
LT3411484T LTEPPCT/US2017/014914T LTUS2017014914T LT3411484T LT 3411484 T LT3411484 T LT 3411484T LT US2017014914 T LTUS2017014914 T LT US2017014914T LT 3411484 T LT3411484 T LT 3411484T
Authority
LT
Lithuania
Prior art keywords
stranded
injection
self
cerebrospinal fluid
associated virus
Prior art date
Application number
LTEPPCT/US2017/014914T
Other languages
English (en)
Inventor
Anthony DONSANTE
Karen Kozarsky
Nicholas Matthew Boulis
Jonathan Patrick Riley
Original Assignee
Emory University
Regenxbio Inc.
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 Emory University, Regenxbio Inc. filed Critical Emory University
Publication of LT3411484T publication Critical patent/LT3411484T/lt

Links

Classifications

    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
LTEPPCT/US2017/014914T 2016-02-05 2017-01-25 Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį LT3411484T (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662292157P 2016-02-05 2016-02-05
PCT/US2017/014914 WO2017136202A1 (en) 2016-02-05 2017-01-25 Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

Publications (1)

Publication Number Publication Date
LT3411484T true LT3411484T (lt) 2023-11-27

Family

ID=59500060

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2017/014914T LT3411484T (lt) 2016-02-05 2017-01-25 Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį

Country Status (15)

Country Link
US (3) US12070510B2 (lt)
EP (2) EP3411484B1 (lt)
JP (4) JP2019504888A (lt)
AU (1) AU2017214283B2 (lt)
DK (1) DK3411484T3 (lt)
ES (1) ES2962860T3 (lt)
FI (1) FI3411484T3 (lt)
HR (1) HRP20231451T1 (lt)
HU (1) HUE064408T2 (lt)
IL (2) IL300254B2 (lt)
LT (1) LT3411484T (lt)
PL (1) PL3411484T3 (lt)
PT (1) PT3411484T (lt)
SI (1) SI3411484T1 (lt)
WO (1) WO2017136202A1 (lt)

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Also Published As

Publication number Publication date
EP3411484B1 (en) 2023-10-04
IL300254A (en) 2023-03-01
JP2024084797A (ja) 2024-06-25
HUE064408T2 (hu) 2024-03-28
AU2017214283B2 (en) 2023-06-29
US20190038777A1 (en) 2019-02-07
IL260954A (lt) 2018-09-20
ES2962860T3 (es) 2024-03-21
JP2019504888A (ja) 2019-02-21
HRP20231451T1 (hr) 2024-03-01
EP3411484A1 (en) 2018-12-12
US12070510B2 (en) 2024-08-27
SI3411484T1 (sl) 2023-12-29
DK3411484T3 (da) 2023-11-20
IL300254B2 (en) 2025-06-01
IL260954B1 (en) 2023-03-01
IL300254B1 (en) 2025-02-01
PL3411484T3 (pl) 2024-02-19
CA3012653A1 (en) 2017-08-10
JP2025041933A (ja) 2025-03-26
EP3411484A4 (en) 2019-08-07
JP2022101648A (ja) 2022-07-06
IL260954B2 (en) 2023-07-01
WO2017136202A1 (en) 2017-08-10
EP4299079A3 (en) 2024-05-01
EP4299079A2 (en) 2024-01-03
AU2017214283A1 (en) 2018-08-02
PT3411484T (pt) 2023-11-17
FI3411484T3 (fi) 2023-11-15
US20250135041A1 (en) 2025-05-01
US20240108761A1 (en) 2024-04-04

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