BR112022023669A2 - Vlp para o tratamento de leucodistrofias - Google Patents

Vlp para o tratamento de leucodistrofias

Info

Publication number
BR112022023669A2
BR112022023669A2 BR112022023669A BR112022023669A BR112022023669A2 BR 112022023669 A2 BR112022023669 A2 BR 112022023669A2 BR 112022023669 A BR112022023669 A BR 112022023669A BR 112022023669 A BR112022023669 A BR 112022023669A BR 112022023669 A2 BR112022023669 A2 BR 112022023669A2
Authority
BR
Brazil
Prior art keywords
enzyme
leukodystrophies
vlp
treatment
expression vector
Prior art date
Application number
BR112022023669A
Other languages
English (en)
Inventor
Demina Victoria
Ernst Oliver
Hermann Anke
Stapf Markus
Original Assignee
Neuway Pharma Gmbh
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 Neuway Pharma Gmbh filed Critical Neuway Pharma Gmbh
Publication of BR112022023669A2 publication Critical patent/BR112022023669A2/pt

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Classifications

    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • 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)
    • C12Y302/01046—Galactosylceramidase (3.2.1.46)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
    • C12Y305/01015—Aspartoacylase (3.5.1.15)
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/22011—Polyomaviridae, e.g. polyoma, SV40, JC
    • C12N2710/22023—Virus like particles [VLP]
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/22011—Polyomaviridae, e.g. polyoma, SV40, JC
    • C12N2710/22041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/22042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

VLP PARA O TRATAMENTO DE LEUCODISTROFIAS. A presente invenção refere-se a partículas semelhantes a vírus (VLP) associadas a uma enzima expressa anormalmente em leucodistrofias particulares ou um vetor de expressão que codifica a enzima ou um mRNA que codifica a enzima ou uma combinação dos mesmos que são usados em um método para o tratamento de leucodistrofias específicas em um indivíduo que precisa do mesmo, de preferência um ser humano. A invenção refere-se também a uma composição farmacêutica para uso em um método para o tratamento de leucodistrofias particulares, a um vetor de expressão que codifica a enzima expressa anormalmente e a um método de associação de uma VLP com a enzima, um vetor de expressão que codifica a enzima ou um mRNA que codifica a enzima ou uma combinação dos mesmos.
BR112022023669A 2020-05-22 2021-05-19 Vlp para o tratamento de leucodistrofias BR112022023669A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP2020064324 2020-05-22
PCT/EP2021/063375 WO2021234046A1 (en) 2020-05-22 2021-05-19 Vlp for the treatment of leukodystrophies

Publications (1)

Publication Number Publication Date
BR112022023669A2 true BR112022023669A2 (pt) 2023-01-31

Family

ID=71096660

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022023669A BR112022023669A2 (pt) 2020-05-22 2021-05-19 Vlp para o tratamento de leucodistrofias

Country Status (7)

Country Link
US (1) US20230310652A1 (pt)
EP (1) EP4153756A1 (pt)
JP (1) JP2023526528A (pt)
CN (1) CN115968403A (pt)
BR (1) BR112022023669A2 (pt)
CA (1) CA3184307A1 (pt)
WO (1) WO2021234046A1 (pt)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19543553B4 (de) 1995-11-22 2009-04-09 Deutsches Primatenzentrum Gmbh VP-Antigene des JC-Virus
WO2008134123A1 (en) * 2007-04-30 2008-11-06 The Uab Research Foundation Ul97 for treatment of protein aggregate disorders
FR2920158B1 (fr) 2007-08-24 2010-03-12 Centre Nat Rech Scient Production de plasmides et expression de proteines recombinantes dans des cellules cultivees sans antibiotiques
IN2014DN10490A (pt) 2012-06-15 2015-08-21 Taisho Pharmaceutical Co Ltd
EA030305B1 (ru) 2012-08-07 2018-07-31 КЬЕЗИ ФАРМАЧЕУТИЧИ С.п.А. Животная модель болезни краббе
US20230242938A1 (en) * 2018-02-28 2023-08-03 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
EP3688147B8 (en) * 2018-12-18 2022-10-26 NEUWAY Pharma GmbH A novel drug delivery system and methods providing thereof

Also Published As

Publication number Publication date
CA3184307A1 (en) 2021-11-25
US20230310652A1 (en) 2023-10-05
EP4153756A1 (en) 2023-03-29
JP2023526528A (ja) 2023-06-21
WO2021234046A1 (en) 2021-11-25
CN115968403A (zh) 2023-04-14

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Legal Events

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B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing