MX2018005490A - Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia. - Google Patents

Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia.

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Publication number
MX2018005490A
MX2018005490A MX2018005490A MX2018005490A MX2018005490A MX 2018005490 A MX2018005490 A MX 2018005490A MX 2018005490 A MX2018005490 A MX 2018005490A MX 2018005490 A MX2018005490 A MX 2018005490A MX 2018005490 A MX2018005490 A MX 2018005490A
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MX
Mexico
Prior art keywords
factor viii
methods
cpg reduced
compositions
treatment
Prior art date
Application number
MX2018005490A
Other languages
English (en)
Inventor
Anguela Xavier
Hsien- I SHEN Sam
Original Assignee
Spark Therapeutics 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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of MX2018005490A publication Critical patent/MX2018005490A/es

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • 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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12P21/00—Preparation of peptides or proteins
    • 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
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    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
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    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Abstract

Se dan a conocer variantes de ácido nucleico reducido en CpG que codifican la proteína FVIII, y métodos de uso de las mismas. En modalidades particulares, las variantes de ácido nucleico reducido en CpG que codifican FVIII se expresan con mayor eficiencia por las células, se secretan en niveles incrementados por las células por encima de las proteínas Factor VIII de tipo silvestre, exhiben una expresión y/o actividad potenciadas sobre las proteínas Factor VIII de tipo silvestre, o se empaquetan con más eficiencia en vectores virales.
MX2018005490A 2015-10-30 2016-10-31 Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia. MX2018005490A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562249001P 2015-10-30 2015-10-30
US201662331872P 2016-05-04 2016-05-04
US201662349532P 2016-06-13 2016-06-13
US201662357874P 2016-07-01 2016-07-01
PCT/US2016/059793 WO2017075619A1 (en) 2015-10-30 2016-10-31 Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

Publications (1)

Publication Number Publication Date
MX2018005490A true MX2018005490A (es) 2018-08-16

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ID=58630834

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MX2018005490A MX2018005490A (es) 2015-10-30 2016-10-31 Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia.
MX2023001600A MX2023001600A (es) 2015-10-30 2018-04-30 Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia.

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MX2023001600A MX2023001600A (es) 2015-10-30 2018-04-30 Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia.

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US (2) US20180312571A1 (es)
EP (1) EP3368560A4 (es)
JP (2) JP7088831B2 (es)
KR (1) KR20180090795A (es)
CN (1) CN108368164A (es)
AU (1) AU2016344030B2 (es)
BR (1) BR112018008766A2 (es)
CA (1) CA3002689A1 (es)
CL (1) CL2018001107A1 (es)
CO (1) CO2018005306A2 (es)
IL (1) IL258959A (es)
MX (2) MX2018005490A (es)
MY (1) MY190926A (es)
PE (2) PE20231949A1 (es)
PH (1) PH12018501055A1 (es)
RU (1) RU2745506C2 (es)
SA (1) SA518391463B1 (es)
SG (1) SG11201802972UA (es)
WO (1) WO2017075619A1 (es)

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RU2745506C2 (ru) 2021-03-25
PH12018501055A1 (en) 2019-01-28
IL258959A (en) 2018-06-28
JP2022137029A (ja) 2022-09-21
US20180312571A1 (en) 2018-11-01
RU2018119710A3 (es) 2020-04-02
KR20180090795A (ko) 2018-08-13
BR112018008766A2 (pt) 2018-10-30
US11168124B2 (en) 2021-11-09
CN108368164A (zh) 2018-08-03
JP2019503649A (ja) 2019-02-14
SA518391463B1 (ar) 2022-12-13
CA3002689A1 (en) 2017-05-04
MX2023001600A (es) 2023-03-09
MY190926A (en) 2022-05-20
CL2018001107A1 (es) 2018-10-05
PE20181168A1 (es) 2018-07-19
EP3368560A1 (en) 2018-09-05
JP7088831B2 (ja) 2022-06-21
PE20231949A1 (es) 2023-12-05
US20170216408A1 (en) 2017-08-03
RU2018119710A (ru) 2019-12-06
AU2016344030B2 (en) 2021-05-06
EP3368560A4 (en) 2019-03-27
WO2017075619A1 (en) 2017-05-04
AU2016344030A1 (en) 2018-05-10
CO2018005306A2 (es) 2018-11-30
SG11201802972UA (en) 2018-05-30

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