BR112021026866A2 - Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico - Google Patents

Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico

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Publication number
BR112021026866A2
BR112021026866A2 BR112021026866A BR112021026866A BR112021026866A2 BR 112021026866 A2 BR112021026866 A2 BR 112021026866A2 BR 112021026866 A BR112021026866 A BR 112021026866A BR 112021026866 A BR112021026866 A BR 112021026866A BR 112021026866 A2 BR112021026866 A2 BR 112021026866A2
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Brazil
Prior art keywords
methods
lysosomal storage
compositions
vector
storage disorders
Prior art date
Application number
BR112021026866A
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English (en)
Inventor
Cuong Do
Lin Liu
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M6P Therapeutics
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Publication date
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Publication of BR112021026866A2 publication Critical patent/BR112021026866A2/pt

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    • 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
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
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    • 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)
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    • 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)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • 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)
    • C12N9/2477—Hemicellulases not provided in a preceding group
    • C12N9/2488—Mannanases
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • C—CHEMISTRY; METALLURGY
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    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • 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)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • 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)
    • C12Y302/01024—Alpha-mannosidase (3.2.1.24)
    • C—CHEMISTRY; METALLURGY
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    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01046—Galactosylceramidase (3.2.1.46)
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
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    • 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
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    • C12N2800/00—Nucleic acids vectors
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01021—Beta-glucosidase (3.2.1.21)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Veterinary Medicine (AREA)
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  • Diabetes (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

composições de vetor e métodos de uso do mesmo para tratamento de distúrbios do armazamento lisossômico. são aqui fornecidos composições e métodos de uso de um vetor bicistrônico para tratar ou prevenir um distúrbio do armazena-mento lisossômico (lsd) em um indivíduo.as composições divulgadas compreendem um vetor bicistrônico compreendendo um promotor, um sítio de entrada ribossomal interno (ires), um polinucleotídeo que codifica uma enzima lisossômica e um polinucleotídeo que codifica uma glcnac-1 fosfotransferase modificada (glcnac-1 ptase). os presentes métodos compreendem a administração ao indivíduo de uma composição farmacêutica compreendendo o vetor bicistrônico conforme divulgado nesta invenção.
BR112021026866A 2019-07-02 2020-07-02 Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico BR112021026866A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962869781P 2019-07-02 2019-07-02
US201962869808P 2019-07-02 2019-07-02
PCT/US2020/040770 WO2021003442A1 (en) 2019-07-02 2020-07-02 Vector compositions and methods of using same for treatment of lysosomal storage disorders

Publications (1)

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BR112021026866A2 true BR112021026866A2 (pt) 2022-03-03

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BR112021026866A BR112021026866A2 (pt) 2019-07-02 2020-07-02 Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico

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Country Link
US (1) US20220380800A1 (pt)
EP (1) EP3994253A1 (pt)
JP (1) JP2022538497A (pt)
KR (1) KR20220069917A (pt)
CN (1) CN114616000A (pt)
AU (1) AU2020298575A1 (pt)
BR (1) BR112021026866A2 (pt)
CA (1) CA3145662A1 (pt)
TW (1) TW202122579A (pt)
WO (1) WO2021003442A1 (pt)

Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
CN113238053B (zh) * 2021-04-30 2022-05-13 四川大学华西医院 一种用于检测stat3二聚化的质粒
WO2023150051A1 (en) * 2022-02-04 2023-08-10 M6P Therapeutics, Inc. Compositions and methods of using two-promoter vector for treatment of lysosomal storage disorders
WO2023150388A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising glucocerebrocidase and methods of use thereof

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JP5054975B2 (ja) 2003-09-30 2012-10-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途
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AU2020298575A1 (en) 2022-02-24
CN114616000A (zh) 2022-06-10
WO2021003442A1 (en) 2021-01-07
JP2022538497A (ja) 2022-09-02
US20220380800A1 (en) 2022-12-01
EP3994253A1 (en) 2022-05-11
KR20220069917A (ko) 2022-05-27
CA3145662A1 (en) 2021-01-07
TW202122579A (zh) 2021-06-16

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