CO2018005306A2 - Variantes del factor viii reducido con cpg, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasia - Google Patents

Variantes del factor viii reducido con cpg, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasia

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Publication number
CO2018005306A2
CO2018005306A2 CONC2018/0005306A CO2018005306A CO2018005306A2 CO 2018005306 A2 CO2018005306 A2 CO 2018005306A2 CO 2018005306 A CO2018005306 A CO 2018005306A CO 2018005306 A2 CO2018005306 A2 CO 2018005306A2
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Colombia
Prior art keywords
fviii
variants
cpg
nucleic acids
factor viii
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Application number
CONC2018/0005306A
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English (en)
Inventor
Xavier Anguela
Sam Hsien-I Shen
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Spark Therapeutics Inc
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Publication date
Application filed by Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of CO2018005306A2 publication Critical patent/CO2018005306A2/es

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    • 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
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    • 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
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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Abstract

De acuerdo con la invención, se proporcionan dinucleótidos de citosina-guanina (CpG) que reducen las variantes de ácidos nucleicos que codifican la proteína del Factor VIII (FVIII). Dichas variantes de ácidos nucleicos reducidas con CpG son distintas del ácido nucleico de tipo natural que codifica FVIII y pueden codificar, por ejemplo, la proteína de FVIII humano, que opcionalmente carece, en todo o en parte, del dominio B de FVIII. Dichas variantes de ácidos nucleicos reducidas con CpG incluyen variantes que exhiben una expresión aumentada (por ejemplo, una expresión incrementada 1-5 veces) en comparación con ácidos nucleicos de FVIII optimizados por codones tales como FVIII-CO3 (SEC ID NO: 21), cuando se transfieren a células, dando lugar a un aumento de la secreción de proteína de FVIII y, por lo tanto, aumento de la actividad.
CONC2018/0005306A 2015-10-30 2018-05-21 Variantes del factor viii reducido con cpg, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasia CO2018005306A2 (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)

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CO2018005306A2 true CO2018005306A2 (es) 2018-11-30

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CONC2018/0005306A CO2018005306A2 (es) 2015-10-30 2018-05-21 Variantes del factor viii reducido con cpg, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasia

Country Status (19)

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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) IL258959B2 (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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PE20231949A1 (es) 2023-12-05
IL258959B1 (en) 2024-06-01
AU2016344030A1 (en) 2018-05-10
CA3002689A1 (en) 2017-05-04
RU2018119710A3 (es) 2020-04-02
IL258959B2 (en) 2024-10-01
CL2018001107A1 (es) 2018-10-05
US11168124B2 (en) 2021-11-09
IL258959A (en) 2018-06-28
RU2018119710A (ru) 2019-12-06
JP7088831B2 (ja) 2022-06-21
PE20181168A1 (es) 2018-07-19
AU2016344030B2 (en) 2021-05-06
US20170216408A1 (en) 2017-08-03
CN108368164A (zh) 2018-08-03
EP3368560A4 (en) 2019-03-27
MX2023001600A (es) 2023-03-09
JP2019503649A (ja) 2019-02-14
MY190926A (en) 2022-05-20
BR112018008766A2 (pt) 2018-10-30
JP2022137029A (ja) 2022-09-21
SG11201802972UA (en) 2018-05-30
WO2017075619A1 (en) 2017-05-04
RU2745506C2 (ru) 2021-03-25
EP3368560A1 (en) 2018-09-05
US20180312571A1 (en) 2018-11-01
MX2018005490A (es) 2018-08-16
SA518391463B1 (ar) 2022-12-13
PH12018501055A1 (en) 2019-01-28
KR20180090795A (ko) 2018-08-13

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