MA43968A - Thérapie génique pour traiter la mucopolysaccharidose de type i - Google Patents

Thérapie génique pour traiter la mucopolysaccharidose de type i

Info

Publication number
MA43968A
MA43968A MA043968A MA43968A MA43968A MA 43968 A MA43968 A MA 43968A MA 043968 A MA043968 A MA 043968A MA 43968 A MA43968 A MA 43968A MA 43968 A MA43968 A MA 43968A
Authority
MA
Morocco
Prior art keywords
gene therapy
mucopolysaccharidosis type
treat mucopolysaccharidosis
treat
type
Prior art date
Application number
MA043968A
Other languages
English (en)
Inventor
Christian Hinderer
James M Wilson
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 MA43968A publication Critical patent/MA43968A/fr

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
    • A61K48/005Medicinal 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
    • 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
    • A61K9/0012Galenical 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
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • 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)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031Beta-glucuronidase (3.2.1.31)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/15Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Dispersion Chemistry (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
MA043968A 2016-02-03 2017-02-02 Thérapie génique pour traiter la mucopolysaccharidose de type i MA43968A (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662290547P 2016-02-03 2016-02-03
US201662323271P 2016-04-15 2016-04-15
US201662337178P 2016-05-16 2016-05-16
US201662367798P 2016-07-28 2016-07-28
US201762452560P 2017-01-31 2017-01-31

Publications (1)

Publication Number Publication Date
MA43968A true MA43968A (fr) 2018-12-12

Family

ID=58108727

Family Applications (1)

Application Number Title Priority Date Filing Date
MA043968A MA43968A (fr) 2016-02-03 2017-02-02 Thérapie génique pour traiter la mucopolysaccharidose de type i

Country Status (11)

Country Link
US (3) US10973929B2 (fr)
EP (1) EP3411488A1 (fr)
JP (2) JP7360241B2 (fr)
KR (1) KR20180121899A (fr)
AU (2) AU2017215211C1 (fr)
BR (1) BR112018015751A2 (fr)
CA (1) CA3012195A1 (fr)
IL (1) IL260959B1 (fr)
MA (1) MA43968A (fr)
SG (2) SG11201806270XA (fr)
WO (1) WO2017136500A1 (fr)

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MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018209205A1 (fr) 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Thérapie génique de céroïdes-lipofuscinoses neuronales
CA3068328A1 (fr) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Therapie genique mediee par aav9 pour traiter la mucopolysaccharidose de type i
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EP3717652A4 (fr) 2017-11-30 2021-11-24 The Trustees of the University of Pennsylvania Thérapie génique pour mucopolysaccharidose de type iiib
SG11202100423YA (en) * 2018-07-18 2021-02-25 Regenxbio Inc Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus)
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AU2020369570A1 (en) * 2019-10-23 2022-05-12 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
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EP4096721A1 (fr) * 2020-02-02 2022-12-07 The Trustees of The University of Pennsylvania Compositions utiles pour traiter la gm1 gan[[d]]gliosidose
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
BR112023002904A2 (pt) * 2020-08-26 2023-04-25 Univ Pennsylvania Vírus adenoassociado recombinante para tratar neurodegeneração com início na idade adulta associada a grn
US20230365955A1 (en) 2020-10-09 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
AU2021368725A1 (en) * 2020-10-30 2023-05-25 Immusoft Corporation Methods of administering genetically modified b cells for in vivo delivery of therapeutic agents
CA3209779A1 (fr) 2021-02-01 2022-08-04 Regenxbio Inc. Therapie genique de ceroides-lipofuscinoses neuronales
WO2023087019A2 (fr) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions pour la réduction spécifique de drg de l'expression de transgènes
WO2023102517A1 (fr) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement de la maladie de fabry
WO2023172975A1 (fr) * 2022-03-08 2023-09-14 University Of Massachusetts Méthodes d'encapsulation de vaar
WO2023196893A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement d'un cancer du sein métastatique her2 positif et d'autres cancers
WO2023196892A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Immunisation passive avec des anticorps neutralisants anti-aav pour empêcher la transduction hors cible de vecteurs aav administrés par voie intrathécale

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US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
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MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
CA3068328A1 (fr) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Therapie genique mediee par aav9 pour traiter la mucopolysaccharidose de type i

Also Published As

Publication number Publication date
IL260959A (en) 2018-09-20
AU2017215211B2 (en) 2023-05-18
US20240024510A1 (en) 2024-01-25
SG10201912758TA (en) 2020-02-27
AU2017215211A1 (en) 2018-08-09
EP3411488A1 (fr) 2018-12-12
WO2017136500A1 (fr) 2017-08-10
AU2023202680A1 (en) 2023-07-13
JP7360241B2 (ja) 2023-10-12
US10973929B2 (en) 2021-04-13
SG11201806270XA (en) 2018-08-30
CA3012195A1 (fr) 2017-08-10
JP2023062088A (ja) 2023-05-02
KR20180121899A (ko) 2018-11-09
BR112018015751A2 (pt) 2019-02-05
JP2019512005A (ja) 2019-05-09
AU2017215211C1 (en) 2023-11-16
IL260959B1 (en) 2024-05-01
US20210228740A1 (en) 2021-07-29
US20190038772A1 (en) 2019-02-07

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