BR112022023669A2 - Vlp para o tratamento de leucodistrofias - Google Patents
Vlp para o tratamento de leucodistrofiasInfo
- 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
Links
- 208000036546 leukodystrophy Diseases 0.000 title abstract 5
- 102000004190 Enzymes Human genes 0.000 abstract 7
- 108090000790 Enzymes Proteins 0.000 abstract 7
- 239000013604 expression vector Substances 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 3
- 108020004999 messenger RNA Proteins 0.000 abstract 2
- 239000002245 particle Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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- 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
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- 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
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- 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)
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- 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)
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- 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)
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- 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)
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- 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]
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- 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
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- 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)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (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.
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)
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 |
ME02606B (me) | 2012-06-15 | 2017-06-20 | Taisho Pharmaceutical Co Ltd | Jedinjenja 1,3 - oksazolidina ili 1,3- oksazinana kao antagonisti oreksinskog receptora |
EP2882284B1 (en) | 2012-08-07 | 2017-11-15 | Chiesi Farmaceutici S.p.A. | Animal model of krabbe's disease |
AU2019227896A1 (en) * | 2018-02-28 | 2020-08-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
BR112019014209A2 (pt) * | 2018-12-18 | 2021-09-28 | Neuway Pharma Gmbh | Sistema de liberação de fármaco, método para provimento do mesmo, partículas semelhantes a vírus (vlp), composição e uso do sistema de liberação de fármaco |
-
2021
- 2021-05-19 CN CN202180046442.8A patent/CN115968403A/zh active Pending
- 2021-05-19 CA CA3184307A patent/CA3184307A1/en active Pending
- 2021-05-19 BR BR112022023669A patent/BR112022023669A2/pt not_active Application Discontinuation
- 2021-05-19 EP EP21726656.8A patent/EP4153756A1/en active Pending
- 2021-05-19 WO PCT/EP2021/063375 patent/WO2021234046A1/en unknown
- 2021-05-19 JP JP2022571290A patent/JP2023526528A/ja active Pending
- 2021-05-19 US US17/926,678 patent/US20230310652A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CA3184307A1 (en) | 2021-11-25 |
WO2021234046A1 (en) | 2021-11-25 |
CN115968403A (zh) | 2023-04-14 |
US20230310652A1 (en) | 2023-10-05 |
JP2023526528A (ja) | 2023-06-21 |
EP4153756A1 (en) | 2023-03-29 |
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Legal Events
Date | Code | Title | Description |
---|---|---|---|
B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing |