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BR112015021036A2 - composições e métodos para tratar mps1 - Google Patents

composições e métodos para tratar mps1

Info

Publication number
BR112015021036A2
BR112015021036A2 BR112015021036A BR112015021036A BR112015021036A2 BR 112015021036 A2 BR112015021036 A2 BR 112015021036A2 BR 112015021036 A BR112015021036 A BR 112015021036A BR 112015021036 A BR112015021036 A BR 112015021036A BR 112015021036 A2 BR112015021036 A2 BR 112015021036A2
Authority
BR
Brazil
Prior art keywords
methods
compositions
treating
sequence
vector
Prior art date
Application number
BR112015021036A
Other languages
English (en)
Inventor
L Gurda Brittney
M Wilson James
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 BR112015021036A2 publication Critical patent/BR112015021036A2/pt

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
    • 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
    • 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
    • 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)

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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

resumo "composições e métodos para tratar mps1." trata-se de um vetor que tem um cassete de expressão que contém um gene de hidua que tem uma sequência de seq id no:1 ou uma sequência de pelo menos cerca de 95% idêntica à mesma que codifica uma alfa-l-iduronidase humana funcional. o vetor pode ser um vetor de produção ou um raav8. também são fornecidas composições que contêm esses vetores e métodos para tratar mpsi e os sintomas associados às síndromes de hurler, hurler-scheie e scheie.
BR112015021036A 2013-03-15 2014-03-13 composições e métodos para tratar mps1 BR112015021036A2 (pt)

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
BR112015021036A2 true BR112015021036A2 (pt) 2017-10-10

Family

ID=51580934

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015021036A BR112015021036A2 (pt) 2013-03-15 2014-03-13 composições e métodos para tratar mps1

Country Status (22)

Country Link
US (3) US10137176B2 (pt)
EP (2) EP3747998B1 (pt)
JP (4) JP6591956B2 (pt)
KR (2) KR102346455B1 (pt)
CN (1) CN105026554B (pt)
AU (2) AU2014235096B2 (pt)
BR (1) BR112015021036A2 (pt)
CA (1) CA2901328C (pt)
CY (1) CY1123169T1 (pt)
DK (1) DK2984166T3 (pt)
ES (1) ES2805355T3 (pt)
HK (1) HK1221735A1 (pt)
HR (1) HRP20201063T1 (pt)
HU (1) HUE051373T2 (pt)
LT (1) LT2984166T (pt)
MX (2) MX2015012739A (pt)
PL (1) PL2984166T3 (pt)
PT (1) PT2984166T (pt)
RU (1) RU2708318C2 (pt)
SG (2) SG11201507507PA (pt)
SI (1) SI2984166T1 (pt)
WO (1) WO2014151341A1 (pt)

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CA2901328A1 (en) 2014-09-25
CY1123169T1 (el) 2021-10-29
US10792343B2 (en) 2020-10-06
KR102581652B1 (ko) 2023-09-22
RU2015144234A (ru) 2017-04-24
JP2016512683A (ja) 2016-05-09
EP2984166B1 (en) 2020-04-22
HUE051373T2 (hu) 2021-03-01
AU2014235096A1 (en) 2015-09-10
AU2020203826A1 (en) 2020-07-02
ES2805355T3 (es) 2021-02-11
PT2984166T (pt) 2020-07-16
PL2984166T3 (pl) 2021-01-11
KR102346455B1 (ko) 2022-01-04
CN105026554A (zh) 2015-11-04
RU2708318C2 (ru) 2019-12-05
CA2901328C (en) 2023-08-01
JP2020202863A (ja) 2020-12-24
MX2015012739A (es) 2016-02-19
CN105026554B (zh) 2020-08-11
US20160000887A1 (en) 2016-01-07
US20200397872A1 (en) 2020-12-24
EP3747998B1 (en) 2024-07-24
JP7553527B2 (ja) 2024-09-18
MX2021010918A (es) 2021-10-01
HK1221735A1 (zh) 2017-06-09
AU2020203826B2 (en) 2022-09-15
US10137176B2 (en) 2018-11-27
JP7168621B2 (ja) 2022-11-09
US20190111116A1 (en) 2019-04-18
KR20150129678A (ko) 2015-11-20
DK2984166T3 (da) 2020-07-20
EP2984166A1 (en) 2016-02-17
AU2014235096B2 (en) 2020-03-19
SG10201707319UA (en) 2017-10-30
EP3747998A9 (en) 2021-08-04
SI2984166T1 (sl) 2020-09-30
KR20220005599A (ko) 2022-01-13
HRP20201063T1 (hr) 2020-11-27
EP2984166A4 (en) 2016-09-07
SG11201507507PA (en) 2015-10-29
EP3747998A1 (en) 2020-12-09
LT2984166T (lt) 2020-08-10
JP6591956B2 (ja) 2019-10-16
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JP2019134717A (ja) 2019-08-15
WO2014151341A1 (en) 2014-09-25

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