SG10201912401QA - Gene therapy for neuronal ceroid lipofuscinoses - Google Patents

Gene therapy for neuronal ceroid lipofuscinoses

Info

Publication number
SG10201912401QA
SG10201912401QA SG10201912401QA SG10201912401QA SG10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA SG 10201912401Q A SG10201912401Q A SG 10201912401QA
Authority
SG
Singapore
Prior art keywords
gene therapy
neuronal ceroid
ceroid lipofuscinoses
lipofuscinoses
neuronal
Prior art date
Application number
SG10201912401QA
Inventor
James Wilson
Nathan Katz
Christian Hinderer
Juliette Hordeaux
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG10201912401QA publication Critical patent/SG10201912401QA/en

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    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • 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
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485Exopeptidases (3.4.11-3.4.19)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16111Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141Use of virus, viral particle or viral elements as a vector
    • C12N2710/16143Use 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
    • C12N2750/14143Use 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
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009Tripeptidyl-peptidase I (3.4.14.9)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dispersion Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
SG10201912401QA 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses SG10201912401QA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762504817P 2017-05-11 2017-05-11
US201762599816P 2017-12-18 2017-12-18
US201862652006P 2018-04-03 2018-04-03

Publications (1)

Publication Number Publication Date
SG10201912401QA true SG10201912401QA (en) 2020-02-27

Family

ID=64105028

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201912401QA SG10201912401QA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses
SG11201910015S SG11201910015SA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201910015S SG11201910015SA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Country Status (10)

Country Link
US (2) US11591614B2 (en)
EP (1) EP3621612A4 (en)
JP (2) JP7273730B2 (en)
KR (1) KR20200023280A (en)
AU (1) AU2018265531A1 (en)
BR (1) BR112019023303A2 (en)
CA (1) CA3061655A1 (en)
RU (1) RU2019139555A (en)
SG (2) SG10201912401QA (en)
WO (1) WO2018209205A1 (en)

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AU2017355502B2 (en) * 2016-11-04 2023-08-31 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
KR20200104852A (en) 2017-09-22 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Gene therapy for the treatment of type II mucopolysaccharides
SG11202104295UA (en) * 2018-11-14 2021-06-29 Regenxbio Inc Gene therapy for neuronal ceroid lipofuscinoses
KR20210148333A (en) * 2019-04-08 2021-12-07 더 칠드런스 호스피탈 오브 필라델피아 Treatment of ocular lysosomal storage diseases through administration of AAV expressing TPP1
US20220168449A1 (en) * 2019-04-12 2022-06-02 Encoded Therapeutics, Inc. Compositions and methods for administration of therapeutics
MX2022014258A (en) 2020-05-12 2023-02-22 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression.
MX2023008826A (en) 2021-02-01 2023-09-15 Regenxbio Inc Gene therapy for neuronal ceroid lipofuscinoses.
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression

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Also Published As

Publication number Publication date
US11591614B2 (en) 2023-02-28
RU2019139555A3 (en) 2021-10-12
AU2018265531A1 (en) 2019-11-14
EP3621612A4 (en) 2021-06-16
KR20200023280A (en) 2020-03-04
RU2019139555A (en) 2021-06-11
EP3621612A1 (en) 2020-03-18
BR112019023303A2 (en) 2020-06-16
US20230383312A1 (en) 2023-11-30
WO2018209205A9 (en) 2019-11-21
US20200239908A1 (en) 2020-07-30
SG11201910015SA (en) 2019-11-28
JP7273730B2 (en) 2023-05-15
JP2020519292A (en) 2020-07-02
JP2023099113A (en) 2023-07-11
WO2018209205A1 (en) 2018-11-15
CA3061655A1 (en) 2018-11-15

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