MX2020012170A - Nuevos vectores virales adeno-asociados dirigidos al higado. - Google Patents

Nuevos vectores virales adeno-asociados dirigidos al higado.

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Publication number
MX2020012170A
MX2020012170A MX2020012170A MX2020012170A MX2020012170A MX 2020012170 A MX2020012170 A MX 2020012170A MX 2020012170 A MX2020012170 A MX 2020012170A MX 2020012170 A MX2020012170 A MX 2020012170A MX 2020012170 A MX2020012170 A MX 2020012170A
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MX
Mexico
Prior art keywords
viral vectors
novel
associated viral
liver targeting
novel liver
Prior art date
Application number
MX2020012170A
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English (en)
Inventor
Peter Cameron Colosi
Silvia Ramirez
Original Assignee
Biomarin Pharm Inc
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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of MX2020012170A publication Critical patent/MX2020012170A/es

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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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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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    • 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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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Abstract

La invención se refiere a nuevas proteínas de cápside de virus adenoasociado (AAV), partículas de AAV que comprenden una nueva proteína de cápside, polinucleótidos que codifican para estas proteínas de cápside y vectores de AAV que expresan estas proteínas de cápside. La invención también se refiere a métodos para preparar los vectores de AAV descritos en el presente documento que expresan las nuevas proteínas de cápside de la invención y a los usos terapéuticos asociados de las mismas.
MX2020012170A 2018-05-14 2019-05-14 Nuevos vectores virales adeno-asociados dirigidos al higado. MX2020012170A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862671265P 2018-05-14 2018-05-14
PCT/US2019/032097 WO2019222136A2 (en) 2018-05-14 2019-05-14 Novel liver targeting adeno-associated viral vectors

Publications (1)

Publication Number Publication Date
MX2020012170A true MX2020012170A (es) 2021-01-29

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MX2020012170A MX2020012170A (es) 2018-05-14 2019-05-14 Nuevos vectores virales adeno-asociados dirigidos al higado.

Country Status (16)

Country Link
US (1) US11821008B2 (es)
EP (1) EP3794016A2 (es)
JP (1) JP2021522842A (es)
KR (1) KR20210010496A (es)
CN (1) CN112424217A (es)
AR (1) AR115097A1 (es)
AU (1) AU2019268302A1 (es)
BR (1) BR112020023292A2 (es)
CA (1) CA3100001A1 (es)
CL (2) CL2020002960A1 (es)
MX (1) MX2020012170A (es)
PE (1) PE20210113A1 (es)
PH (1) PH12020551938A1 (es)
SG (1) SG11202011145UA (es)
TW (1) TW202005978A (es)
WO (1) WO2019222136A2 (es)

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