LT2984166T - Kompozicijos, skirtos mpsi gydymui - Google Patents

Kompozicijos, skirtos mpsi gydymui

Info

Publication number
LT2984166T
LT2984166T LTEP14770186.6T LT14770186T LT2984166T LT 2984166 T LT2984166 T LT 2984166T LT 14770186 T LT14770186 T LT 14770186T LT 2984166 T LT2984166 T LT 2984166T
Authority
LT
Lithuania
Prior art keywords
compositions
mpsi
treating
treating mpsi
Prior art date
Application number
LTEP14770186.6T
Other languages
English (en)
Inventor
James Wilson
Brittney GURDA
Original Assignee
The Trustees Of The University Of 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 The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of LT2984166T publication Critical patent/LT2984166T/lt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
LTEP14770186.6T 2013-03-15 2014-03-13 Kompozicijos, skirtos mpsi gydymui LT2984166T (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361788724P 2013-03-15 2013-03-15
PCT/US2014/025509 WO2014151341A1 (en) 2013-03-15 2014-03-13 Compositions and methods for treating mpsi

Publications (1)

Publication Number Publication Date
LT2984166T true LT2984166T (lt) 2020-08-10

Family

ID=51580934

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP14770186.6T LT2984166T (lt) 2013-03-15 2014-03-13 Kompozicijos, skirtos mpsi gydymui

Country Status (22)

Country Link
US (3) US10137176B2 (lt)
EP (2) EP3747998A1 (lt)
JP (4) JP6591956B2 (lt)
KR (2) KR102346455B1 (lt)
CN (1) CN105026554B (lt)
AU (2) AU2014235096B2 (lt)
BR (1) BR112015021036A2 (lt)
CA (1) CA2901328C (lt)
CY (1) CY1123169T1 (lt)
DK (1) DK2984166T3 (lt)
ES (1) ES2805355T3 (lt)
HK (1) HK1221735A1 (lt)
HR (1) HRP20201063T1 (lt)
HU (1) HUE051373T2 (lt)
LT (1) LT2984166T (lt)
MX (2) MX2015012739A (lt)
PL (1) PL2984166T3 (lt)
PT (1) PT2984166T (lt)
RU (1) RU2708318C2 (lt)
SG (2) SG10201707319UA (lt)
SI (1) SI2984166T1 (lt)
WO (1) WO2014151341A1 (lt)

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BR112018015751A2 (pt) * 2016-02-03 2019-02-05 Univ Pennsylvania terapia gênica para tratamento de mucopolissacaridose tipo i
WO2017136533A1 (en) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses
AU2017223465A1 (en) * 2016-02-22 2018-08-09 The University Of North Carolina At Chapel Hill AAV-IDUA vector for treatment of MPS I-associated blindness
MA44873A (fr) * 2016-04-15 2019-03-13 Univ Pennsylvania Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative
BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
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EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
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IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
CA3193833A1 (en) 2020-10-09 2022-04-14 Juliette HORDEAUX Compositions and methods for treatment of fabry disease
KR102590276B1 (ko) * 2021-01-04 2023-10-16 부산대학교 산학협력단 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도
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CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i

Also Published As

Publication number Publication date
PL2984166T3 (pl) 2021-01-11
BR112015021036A2 (pt) 2017-10-10
EP3747998A9 (en) 2021-08-04
US20160000887A1 (en) 2016-01-07
CA2901328A1 (en) 2014-09-25
EP2984166A4 (en) 2016-09-07
CN105026554B (zh) 2020-08-11
HUE051373T2 (hu) 2021-03-01
DK2984166T3 (da) 2020-07-20
JP2023011752A (ja) 2023-01-24
MX2021010918A (es) 2021-10-01
AU2020203826A1 (en) 2020-07-02
ES2805355T3 (es) 2021-02-11
SG11201507507PA (en) 2015-10-29
MX2015012739A (es) 2016-02-19
SG10201707319UA (en) 2017-10-30
KR102346455B1 (ko) 2022-01-04
AU2014235096B2 (en) 2020-03-19
SI2984166T1 (sl) 2020-09-30
JP2020202863A (ja) 2020-12-24
US10792343B2 (en) 2020-10-06
EP2984166B1 (en) 2020-04-22
JP2016512683A (ja) 2016-05-09
PT2984166T (pt) 2020-07-16
EP3747998A1 (en) 2020-12-09
KR102581652B1 (ko) 2023-09-22
CY1123169T1 (el) 2021-10-29
JP6591956B2 (ja) 2019-10-16
US20190111116A1 (en) 2019-04-18
KR20150129678A (ko) 2015-11-20
CN105026554A (zh) 2015-11-04
AU2014235096A1 (en) 2015-09-10
US20200397872A1 (en) 2020-12-24
RU2708318C2 (ru) 2019-12-05
EP2984166A1 (en) 2016-02-17
HRP20201063T1 (hr) 2020-11-27
JP2019134717A (ja) 2019-08-15
CA2901328C (en) 2023-08-01
AU2020203826B2 (en) 2022-09-15
US10137176B2 (en) 2018-11-27
RU2015144234A (ru) 2017-04-24
JP7168621B2 (ja) 2022-11-09
KR20220005599A (ko) 2022-01-13
HK1221735A1 (zh) 2017-06-09
WO2014151341A1 (en) 2014-09-25

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