MX2023001600A - Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia. - Google Patents

Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia.

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Publication number
MX2023001600A
MX2023001600A MX2023001600A MX2023001600A MX2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A MX 2023001600 A MX2023001600 A MX 2023001600A
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Mexico
Prior art keywords
factor viii
methods
cpg reduced
compositions
treatment
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Application number
MX2023001600A
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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 MX2023001600A publication Critical patent/MX2023001600A/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
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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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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    • 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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  • Health & Medical Sciences (AREA)
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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.
MX2023001600A 2015-10-30 2018-04-30 Variantes de factor viii reducido en cpg y composiciones para usarse en el tratamiento de trastornos de hemostasia. MX2023001600A (es)

Applications Claiming Priority (4)

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

Publications (1)

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MX2023001600A true MX2023001600A (es) 2023-03-09

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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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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) 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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KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
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TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
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MX2021002198A (es) * 2018-08-24 2021-07-15 Spark Therapeutics Inc Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos de uso.
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
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CN114929735A (zh) * 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
WO2023036054A2 (en) * 2021-09-08 2023-03-16 Inspirar Limited Composition and method for treating hemophilia
WO2023211316A1 (en) * 2022-04-28 2023-11-02 Joint Stock Company "Biocad" Codon-optimized nucleic acid encoding the fviii-bdd
WO2024050039A2 (en) * 2022-08-31 2024-03-07 Hydrogene Therapeutics, Inc. Methods and compositions for hydrodynamic gene delivery
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用

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AU2016344030A1 (en) 2018-05-10
BR112018008766A2 (pt) 2018-10-30
EP3368560A1 (en) 2018-09-05
RU2745506C2 (ru) 2021-03-25
JP2022137029A (ja) 2022-09-21
RU2018119710A (ru) 2019-12-06
US11168124B2 (en) 2021-11-09
CL2018001107A1 (es) 2018-10-05
PE20231949A1 (es) 2023-12-05
EP3368560A4 (en) 2019-03-27
JP7088831B2 (ja) 2022-06-21
PE20181168A1 (es) 2018-07-19
RU2018119710A3 (es) 2020-04-02
US20180312571A1 (en) 2018-11-01
WO2017075619A1 (en) 2017-05-04
CA3002689A1 (en) 2017-05-04
MX2018005490A (es) 2018-08-16
CO2018005306A2 (es) 2018-11-30
US20170216408A1 (en) 2017-08-03
AU2016344030B2 (en) 2021-05-06
MY190926A (en) 2022-05-20
KR20180090795A (ko) 2018-08-13
JP2019503649A (ja) 2019-02-14
CN108368164A (zh) 2018-08-03
SG11201802972UA (en) 2018-05-30
PH12018501055A1 (en) 2019-01-28
IL258959A (en) 2018-06-28
SA518391463B1 (ar) 2022-12-13

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